{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,3]],"date-time":"2026-11-03T17:22:57Z","timestamp":1793726577647,"version":"4.6.1"},"reference-count":375,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,10,3]],"date-time":"2025-10-03T00:00:00Z","timestamp":1759449600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100000265","name":"UK Research and Innovation Medical Research Council","doi-asserted-by":"publisher","award":["MR\/S009434\/1"],"award-info":[{"award-number":["MR\/S009434\/1"]}],"id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000265","name":"UK Research and Innovation Medical Research Council","doi-asserted-by":"publisher","award":["MR\/S036784\/1"],"award-info":[{"award-number":["MR\/S036784\/1"]}],"id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000265","name":"UK Research and Innovation Medical Research Council","doi-asserted-by":"publisher","award":["MR\/R025134\/1"],"award-info":[{"award-number":["MR\/R025134\/1"]}],"id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000265","name":"UK Research and Innovation Medical Research Council","doi-asserted-by":"publisher","award":["MR\/T044853\/1"],"award-info":[{"award-number":["MR\/T044853\/1"]}],"id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000265","name":"UK Research and Innovation Medical Research Council","doi-asserted-by":"publisher","award":["MR\/V034758\/1"],"award-info":[{"award-number":["MR\/V034758\/1"]}],"id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000265","name":"UK Research and Innovation Medical Research Council","doi-asserted-by":"publisher","award":["MR\/Y008405\/1"],"award-info":[{"award-number":["MR\/Y008405\/1"]}],"id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000265","name":"UK Research and Innovation Medical Research Council","doi-asserted-by":"publisher","award":["MR\/X008029\/1"],"award-info":[{"award-number":["MR\/X008029\/1"]}],"id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000265","name":"UK Research and Innovation Medical Research Council","doi-asserted-by":"publisher","award":["MR\/N026101\/1"],"award-info":[{"award-number":["MR\/N026101\/1"]}],"id":[{"id":"10.13039\/501100000265","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100032470","name":"Jameel Education Foundation","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100032470","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100019256","name":"NIHR Great Ormond Street Hospital Biomedical Research Centre","doi-asserted-by":"publisher","award":["562868"],"award-info":[{"award-number":["562868"]}],"id":[{"id":"10.13039\/501100019256","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100014013","name":"UK Research and Innovation","doi-asserted-by":"publisher","award":["EP\/Z000165\/1"],"award-info":[{"award-number":["EP\/Z000165\/1"]}],"id":[{"id":"10.13039\/100014013","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000833","name":"Rosetrees","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000833","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010269","name":"Wellcome Trust","doi-asserted-by":"publisher","award":["204841\/Z\/16\/Z"],"award-info":[{"award-number":["204841\/Z\/16\/Z"]}],"id":[{"id":"10.13039\/100010269","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100010269","name":"Wellcome Trust","doi-asserted-by":"publisher","award":["223082\/Z\/21\/Z"],"award-info":[{"award-number":["223082\/Z\/21\/Z"]}],"id":[{"id":"10.13039\/100010269","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000325","name":"Wellbeing of Women","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000325","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100011417","name":"United Kingdom Clinical Research Collaboration","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100011417","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100006500","name":"Cure Huntington&apos;s Disease Initiative","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006500","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000272","name":"National Institute for Health and Care Research","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000272","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100017510","name":"UK Dementia Research Institute","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100017510","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000282","name":"Sir Jules Thorn Charitable Trust","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000282","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000861","name":"Sparks","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100000861","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100000781","name":"European Research Council","doi-asserted-by":"publisher","award":["EP\/Z000165\/1"],"award-info":[{"award-number":["EP\/Z000165\/1"]}],"id":[{"id":"10.13039\/501100000781","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100012357","name":"LifeArc","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100012357","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001279","name":"Great Ormond Street Hospital Children&apos;s Charity","doi-asserted-by":"publisher","award":["V2403"],"award-info":[{"award-number":["V2403"]}],"id":[{"id":"10.13039\/501100001279","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001279","name":"Great Ormond Street Hospital Children&apos;s Charity","doi-asserted-by":"publisher","award":["V4422"],"award-info":[{"award-number":["V4422"]}],"id":[{"id":"10.13039\/501100001279","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100003784","name":"Great Ormond Street Hospital for Children","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100003784","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100005725","name":"CHDI Foundation Los Angeles","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100005725","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["pharmrev.aspetjournals.org","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Pharmacological Reviews"],"published-print":{"date-parts":[[2026,1]]},"DOI":"10.1016\/j.pharmr.2025.100093","type":"journal-article","created":{"date-parts":[[2025,10,3]],"date-time":"2025-10-03T15:12:22Z","timestamp":1759504342000},"page":"100093","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":4,"title":["Genetic therapies for neurological diseases"],"prefix":"10.1016","volume":"78","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0044-0949","authenticated-orcid":false,"given":"Ahad A.","family":"Rahim","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Manju A.","family":"Kurian","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-8440-3074","authenticated-orcid":false,"given":"Haiyan","family":"Zhou","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0000-9588-2656","authenticated-orcid":false,"given":"Ross","family":"Ferguson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sarah J.","family":"Tabrizi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3963-9296","authenticated-orcid":false,"given":"Gabriele","family":"Lignani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7015-5196","authenticated-orcid":false,"given":"Kristian","family":"Aquilina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Simon N.","family":"Waddington","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.pharmr.2025.100093_bib1","doi-asserted-by":"crossref","first-page":"23","DOI":"10.1016\/j.coviro.2017.06.006","article-title":"Origin of the retroviruses: when, where, and how?","volume":"25","author":"Hayward","year":"2017","journal-title":"Curr Opin Virol"},{"issue":"3685","key":"10.1016\/j.pharmr.2025.100093_bib2","doi-asserted-by":"crossref","first-page":"754","DOI":"10.1126\/science.149.3685.754","article-title":"Adenovirus-associated defective virus particles","volume":"149","author":"Atchison","year":"1965","journal-title":"Science"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib3","doi-asserted-by":"crossref","first-page":"120","DOI":"10.1002\/jgm.324","article-title":"Analysis of site-specific transgene integration following cotransduction with recombinant adeno-associated virus and a rep encoding plasmid","volume":"5","author":"Huttner","year":"2003","journal-title":"J Gene Med"},{"issue":"15","key":"10.1016\/j.pharmr.2025.100093_bib4","doi-asserted-by":"crossref","first-page":"7874","DOI":"10.1128\/JVI.78.15.7874-7882.2004","article-title":"Adeno-associated virus site-specific integration and AAVS1 disruption","volume":"78","author":"Hamilton","year":"2004","journal-title":"J Virol"},{"issue":"11","key":"10.1016\/j.pharmr.2025.100093_bib5","doi-asserted-by":"crossref","first-page":"1950","DOI":"10.1038\/mt.2011.107","article-title":"Stable human FIX expression after 0.9G intrauterine gene transfer of self-complementary adeno-associated viral vector 5 and 8 in macaques","volume":"19","author":"Mattar","year":"2011","journal-title":"Mol Ther"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib6","doi-asserted-by":"crossref","first-page":"320","DOI":"10.1111\/hae.14936","article-title":"Long-term safety and efficacy outcomes of valoctocogene roxaparvovec gene transfer up to 6 years post-treatment","volume":"30","author":"Symington","year":"2024","journal-title":"Haemophilia"},{"issue":"10","key":"10.1016\/j.pharmr.2025.100093_bib7","doi-asserted-by":"crossref","first-page":"789","DOI":"10.1038\/s41573-023-00766-7","article-title":"AAV-based in vivo gene therapy for neurological disorders","volume":"22","author":"Ling","year":"2023","journal-title":"Nat Rev Drug Discov"},{"issue":"594","key":"10.1016\/j.pharmr.2025.100093_bib8","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aaw1564","article-title":"Gene therapy restores dopamine transporter expression and ameliorates pathology in iPSC and mouse models of infantile parkinsonism","volume":"13","author":"Ng","year":"2021","journal-title":"Sci Transl Med"},{"issue":"13","key":"10.1016\/j.pharmr.2025.100093_bib9","first-page":"1427","article-title":"Eladocagene exuparvovec: first approval","volume":"82","author":"Keam","year":"2022","journal-title":"Drugs"},{"issue":"7","key":"10.1016\/j.pharmr.2025.100093_bib10","doi-asserted-by":"crossref","first-page":"1964","DOI":"10.1093\/brain\/awaa189","article-title":"Gene therapy for global brain diseases: one small step for mice, one giant leap for humans","volume":"143","author":"Rahim","year":"2020","journal-title":"Brain"},{"issue":"11","key":"10.1016\/j.pharmr.2025.100093_bib11","doi-asserted-by":"crossref","first-page":"2882","DOI":"10.1038\/s44321-024-00148-5","article-title":"Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47","volume":"16","author":"Wiseman","year":"2024","journal-title":"EMBO Mol Med"},{"issue":"10","key":"10.1016\/j.pharmr.2025.100093_bib12","doi-asserted-by":"crossref","DOI":"10.1172\/JCI164575","article-title":"Intrathecal AAV9\/AP4M1 gene therapy for hereditary spastic paraplegia 50 shows safety and efficacy in preclinical studies","volume":"133","author":"Chen","year":"2023","journal-title":"J Clin Invest"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib13","doi-asserted-by":"crossref","first-page":"438","DOI":"10.1016\/j.neurol.2025.02.008","article-title":"Recent progress and challenges in gene therapy for pharmacoresistant focal epilepsy","volume":"181","author":"Kullmann","year":"2025","journal-title":"Rev Neurol"},{"issue":"792","key":"10.1016\/j.pharmr.2025.100093_bib14","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.adq3614","article-title":"Self-regulating gene therapy ameliorates phenotypes and overcomes gene dosage sensitivity in a mouse model of Rett syndrome","volume":"17","author":"Ross","year":"2025","journal-title":"Sci Transl Med"},{"key":"10.1016\/j.pharmr.2025.100093_bib15","doi-asserted-by":"crossref","first-page":"196","DOI":"10.1016\/j.omtm.2021.06.013","article-title":"Gene therapy using an ortholog of human fragile X mental retardation protein partially rescues behavioral abnormalities and EEG activity","volume":"22","author":"Hooper","year":"2021","journal-title":"Mol Ther Methods Clin Dev"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib16","doi-asserted-by":"crossref","first-page":"205","DOI":"10.1016\/S1474-4422(23)00419-2","article-title":"Challenges and opportunities in spinal muscular atrophy therapeutics","volume":"23","author":"Yeo","year":"2024","journal-title":"Lancet Neurol"},{"issue":"9","key":"10.1016\/j.pharmr.2025.100093_bib17","doi-asserted-by":"crossref","first-page":"641","DOI":"10.1038\/nrd.2018.110","article-title":"Gene therapy for neurological disorders: progress and prospects","volume":"17","author":"Deverman","year":"2018","journal-title":"Nat Rev Drug Discov"},{"issue":"7","key":"10.1016\/j.pharmr.2025.100093_bib18","doi-asserted-by":"crossref","first-page":"2014","DOI":"10.1093\/brain\/awy126","article-title":"A novel adeno-associated virus capsid with enhanced neurotropism corrects a lysosomal transmembrane enzyme deficiency","volume":"141","author":"Tordo","year":"2018","journal-title":"Brain"},{"key":"10.1016\/j.pharmr.2025.100093_bib19","doi-asserted-by":"crossref","first-page":"3075","DOI":"10.1038\/ncomms4075","article-title":"Drawing a high-resolution functional map of adeno-associated virus capsid by massively parallel sequencing","volume":"5","author":"Adachi","year":"2014","journal-title":"Nat Commun"},{"issue":"11","key":"10.1016\/j.pharmr.2025.100093_bib20","doi-asserted-by":"crossref","first-page":"807","DOI":"10.1038\/gt.2016.62","article-title":"Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism","volume":"23","author":"Powell","year":"2016","journal-title":"Gene Ther"},{"issue":"8","key":"10.1016\/j.pharmr.2025.100093_bib21","doi-asserted-by":"crossref","first-page":"1172","DOI":"10.1038\/nn.4593","article-title":"Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems","volume":"20","author":"Chan","year":"2017","journal-title":"Nat Neurosci"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib22","doi-asserted-by":"crossref","first-page":"204","DOI":"10.1038\/nbt.3440","article-title":"Cre-dependent selection yields AAV variants for widespread gene transfer to the adult brain","volume":"34","author":"Deverman","year":"2016","journal-title":"Nat Biotechnol"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib23","doi-asserted-by":"crossref","first-page":"106","DOI":"10.1038\/s41593-021-00969-4","article-title":"AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset","volume":"25","author":"Goertsen","year":"2022","journal-title":"Nat Neurosci"},{"issue":"1\u20132","key":"10.1016\/j.pharmr.2025.100093_bib24","doi-asserted-by":"crossref","first-page":"61","DOI":"10.1089\/hum.2021.069","article-title":"The AAV9 variant capsid AAV-F mediates widespread transgene expression in nonhuman primate spinal cord after intrathecal administration","volume":"33","author":"Beharry","year":"2022","journal-title":"Hum Gene Ther"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib25","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1089\/hum.2018.098","article-title":"Age-related seroprevalence of antibodies against AAV-LK03 in a UK population cohort","volume":"30","author":"Perocheau","year":"2019","journal-title":"Hum Gene Ther"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib26","doi-asserted-by":"crossref","first-page":"450","DOI":"10.1038\/gt.2012.101","article-title":"Global CNS gene delivery and evasion of anti-AAV-neutralizing antibodies by intrathecal AAV administration in non-human primates","volume":"20","author":"Gray","year":"2013","journal-title":"Gene Ther"},{"key":"10.1016\/j.pharmr.2025.100093_bib27","doi-asserted-by":"crossref","first-page":"771","DOI":"10.1016\/j.omtm.2020.04.001","article-title":"Intra-CSF AAV9 and AAVrh10 administration in nonhuman primates: promising routes and vectors for which neurological diseases?","volume":"17","author":"Bey","year":"2020","journal-title":"Mol Ther Methods Clin Dev"},{"issue":"21\u201322","key":"10.1016\/j.pharmr.2025.100093_bib28","doi-asserted-by":"crossref","first-page":"1095","DOI":"10.1089\/hum.2023.060","article-title":"Positron emission tomography quantitative assessment of off-target whole-body biodistribution of I-124-labeled adeno-associated virus capsids administered to cerebral spinal fluid","volume":"34","author":"Rosenberg","year":"2023","journal-title":"Hum Gene Ther"},{"issue":"9","key":"10.1016\/j.pharmr.2025.100093_bib29","doi-asserted-by":"crossref","first-page":"494","DOI":"10.1038\/s41434-021-00237-x","article-title":"Retroviral gene therapy in Germany with a view on previous experience and future perspectives","volume":"28","author":"Morgan","year":"2021","journal-title":"Gene Ther"},{"issue":"11","key":"10.1016\/j.pharmr.2025.100093_bib30","doi-asserted-by":"crossref","first-page":"1481","DOI":"10.1038\/sj.gt.3300768","article-title":"Gene transfer vectors derived from equine infectious anemia virus","volume":"5","author":"Olsen","year":"1998","journal-title":"Gene Ther"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib31","doi-asserted-by":"crossref","first-page":"308","DOI":"10.1002\/1521-2254(200009\/10)2:5<308::AID-JGM131>3.0.CO;2-3","article-title":"Lentiviral vectors: excellent tools for experimental gene transfer and promising candidates for gene therapy","volume":"2","author":"Vigna","year":"2000","journal-title":"J Gene Med"},{"issue":"21","key":"10.1016\/j.pharmr.2025.100093_bib32","doi-asserted-by":"crossref","first-page":"11382","DOI":"10.1073\/pnas.93.21.11382","article-title":"Efficient transfer, integration, and sustained long-term expression of the transgene in adult rat brains injected with a lentiviral vector","volume":"93","author":"Naldini","year":"1996","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"5259","key":"10.1016\/j.pharmr.2025.100093_bib33","doi-asserted-by":"crossref","first-page":"263","DOI":"10.1126\/science.272.5259.263","article-title":"In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector","volume":"272","author":"Naldini","year":"1996","journal-title":"Science"},{"issue":"6","key":"10.1016\/j.pharmr.2025.100093_bib34","doi-asserted-by":"crossref","first-page":"687","DOI":"10.1038\/nbt1216","article-title":"Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration","volume":"24","author":"Montini","year":"2006","journal-title":"Nat Biotechnol"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib35","doi-asserted-by":"crossref","first-page":"1726","DOI":"10.1172\/JCI22922","article-title":"Lentivector-mediated SMN replacement in a mouse model of spinal muscular atrophy","volume":"114","author":"Azzouz","year":"2004","journal-title":"J Clin Invest"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib36","doi-asserted-by":"crossref","first-page":"208","DOI":"10.1016\/j.jneumeth.2006.04.011","article-title":"Transduction of motor neurons and muscle fibers by intramuscular injection of HIV-1-based vectors pseudotyped with select rabies virus glycoproteins","volume":"157","author":"Mentis","year":"2006","journal-title":"J Neurosci Methods"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib37","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1056\/NEJM200301163480314","article-title":"A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency","volume":"348","author":"Hacein-Bey-Abina","year":"2003","journal-title":"N Engl J Med"},{"issue":"9","key":"10.1016\/j.pharmr.2025.100093_bib38","doi-asserted-by":"crossref","first-page":"3143","DOI":"10.1172\/JCI35798","article-title":"Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients","volume":"118","author":"Howe","year":"2008","journal-title":"J Clin Invest"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib39","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1038\/nm1365","article-title":"Effective gene therapy with nonintegrating lentiviral vectors","volume":"12","author":"Y\u00e1\u00f1ez-Mu\u00f1oz","year":"2006","journal-title":"Nat Med"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib40","doi-asserted-by":"crossref","first-page":"509","DOI":"10.1038\/gt.2008.186","article-title":"Efficient gene delivery to the adult and fetal CNS using pseudotyped non-integrating lentiviral vectors","volume":"16","author":"Rahim","year":"2009","journal-title":"Gene Ther"},{"issue":"17","key":"10.1016\/j.pharmr.2025.100093_bib41","doi-asserted-by":"crossref","first-page":"1630","DOI":"10.1056\/NEJMoa1700554","article-title":"Hematopoietic stem-cell gene therapy for cerebral adrenoleukodystrophy","volume":"377","author":"Eichler","year":"2017","journal-title":"N Engl J Med"},{"issue":"10322","key":"10.1016\/j.pharmr.2025.100093_bib42","doi-asserted-by":"crossref","first-page":"372","DOI":"10.1016\/S0140-6736(21)02017-1","article-title":"Lentiviral haematopoietic stem-cell gene therapy for early-onset metachromatic leukodystrophy: long-term results from a non-randomised, open-label, phase 1\/2 trial and expanded access","volume":"399","author":"Fumagalli","year":"2022","journal-title":"Lancet"},{"key":"10.1016\/j.pharmr.2025.100093_bib43","doi-asserted-by":"crossref","first-page":"399","DOI":"10.1016\/j.omtm.2019.04.001","article-title":"Pre-clinical safety and efficacy of lentiviral vector-mediated ex vivo stem cell gene therapy for the treatment of mucopolysaccharidosis IIIA","volume":"13","author":"Ellison","year":"2019","journal-title":"Mol Ther Methods Clin Dev"},{"issue":"7","key":"10.1016\/j.pharmr.2025.100093_bib44","doi-asserted-by":"crossref","DOI":"10.15252\/emmm.201708730","article-title":"Brain-targeted stem cell gene therapy corrects mucopolysaccharidosis type II via multiple mechanisms","volume":"10","author":"Gleitz","year":"2018","journal-title":"EMBO Mol Med"},{"issue":"24","key":"10.1016\/j.pharmr.2025.100093_bib45","doi-asserted-by":"crossref","first-page":"3660","DOI":"10.1002\/1873-3468.13687","article-title":"Adenoviruses across the animal kingdom: a walk in the zoo","volume":"593","author":"Harrach","year":"2019","journal-title":"FEBS Lett"},{"key":"10.1016\/j.pharmr.2025.100093_bib46","doi-asserted-by":"crossref","first-page":"183","DOI":"10.1186\/1749-8090-8-183","article-title":"Assessment of a novel, capsid-modified adenovirus with an improved vascular gene transfer profile","volume":"8","author":"White","year":"2013","journal-title":"J Cardiothorac Surg"},{"issue":"21\u201322","key":"10.1016\/j.pharmr.2025.100093_bib47","doi-asserted-by":"crossref","first-page":"1109","DOI":"10.1089\/hum.2022.178","article-title":"Engineering cancer selective virotherapies: are the pieces of the puzzle falling into place?","volume":"33","author":"Swift","year":"2022","journal-title":"Hum Gene Ther"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib48","doi-asserted-by":"crossref","first-page":"219","DOI":"10.1038\/ng0393-219","article-title":"A model system for in vivo gene transfer into the central nervous system using an adenoviral vector","volume":"3","author":"Davidson","year":"1993","journal-title":"Nat Genet"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib49","doi-asserted-by":"crossref","first-page":"224","DOI":"10.1038\/ng0393-224","article-title":"Transfer of a foreign gene into the brain using adenovirus vectors","volume":"3","author":"Akli","year":"1993","journal-title":"Nat Genet"},{"issue":"5097","key":"10.1016\/j.pharmr.2025.100093_bib50","doi-asserted-by":"crossref","first-page":"988","DOI":"10.1126\/science.8382374","article-title":"An adenovirus vector for gene transfer into neurons and glia in the brain","volume":"259","author":"Le Gal La Salle","year":"1993","journal-title":"Science"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib51","doi-asserted-by":"crossref","first-page":"37","DOI":"10.1089\/hum.1997.8.1-37","article-title":"Innate immune mechanisms dominate elimination of adenoviral vectors following in vivo administration","volume":"8","author":"Worgall","year":"1997","journal-title":"Hum Gene Ther"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib52","doi-asserted-by":"crossref","first-page":"473","DOI":"10.1038\/sj.gt.3300412","article-title":"Adenovirus-mediated gene transfer: influence of transgene, mouse strain and type of immune response on persistence of transgene expression","volume":"4","author":"Michou","year":"1997","journal-title":"Gene Ther"},{"issue":"17","key":"10.1016\/j.pharmr.2025.100093_bib53","doi-asserted-by":"crossref","first-page":"2103","DOI":"10.1089\/hum.1997.8.17-2103","article-title":"A recombinant E1-deleted canine adenoviral vector capable of transduction and expression of a transgene in human-derived cells and in vivo","volume":"8","author":"Klonjkowski","year":"1997","journal-title":"Hum Gene Ther"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib54","doi-asserted-by":"crossref","first-page":"2283","DOI":"10.1096\/fj.01-0321fje","article-title":"Preferential transduction of neurons by canine adenovirus vectors and their efficient retrograde transport in vivo","volume":"15","author":"Soudais","year":"2001","journal-title":"FASEB J"},{"issue":"37","key":"10.1016\/j.pharmr.2025.100093_bib55","doi-asserted-by":"crossref","first-page":"9558","DOI":"10.1523\/JNEUROSCI.0132-16.2016","article-title":"Coxsackievirus adenovirus receptor loss impairs adult neurogenesis, synapse content, and hippocampus plasticity","volume":"36","author":"Zussy","year":"2016","journal-title":"J Neurosci"},{"key":"10.1016\/j.pharmr.2025.100093_bib56","doi-asserted-by":"crossref","first-page":"28","DOI":"10.3389\/fnana.2020.00028","article-title":"Location of the cell adhesion molecule \u201ccoxsackievirus and adenovirus receptor\u201d in the adult mouse brain","volume":"14","author":"Wehbi","year":"2020","journal-title":"Front Neuroanat"},{"issue":"14","key":"10.1016\/j.pharmr.2025.100093_bib57","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.98202","article-title":"Exogenous LRRK2G2019S induces parkinsonian-like pathology in a nonhuman primate","volume":"3","author":"Mestre-Franc\u00e9s","year":"2018","journal-title":"JCI Insight"},{"key":"10.1016\/j.pharmr.2025.100093_bib58","doi-asserted-by":"crossref","first-page":"76","DOI":"10.3389\/fnmol.2020.00076","article-title":"Targeted transgene expression in cholinergic interneurons in the monkey striatum using canine adenovirus serotype 2 vectors","volume":"13","author":"Martel","year":"2020","journal-title":"Front Mol Neurosci"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib59","doi-asserted-by":"crossref","first-page":"391","DOI":"10.1096\/fj.03-0438fje","article-title":"Long-term in vivo transduction of neurons throughout the rat CNS using novel helper-dependent CAV-2 vectors","volume":"18","author":"Soudais","year":"2004","journal-title":"FASEB J"},{"key":"10.1016\/j.pharmr.2025.100093_bib60","doi-asserted-by":"crossref","first-page":"71","DOI":"10.3389\/fnmol.2019.00071","article-title":"CAV-2 vector development and gene transfer in the central and peripheral nervous systems","volume":"12","author":"Del Rio","year":"2019","journal-title":"Front Mol Neurosci"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib61","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1016\/j.cell.2015.07.014","article-title":"Intact-brain analyses reveal distinct information carried by SNc dopamine subcircuits","volume":"162","author":"Lerner","year":"2015","journal-title":"Cell"},{"issue":"7704","key":"10.1016\/j.pharmr.2025.100093_bib62","doi-asserted-by":"crossref","first-page":"183","DOI":"10.1038\/s41586-018-0078-2","article-title":"A midline thalamic circuit determines reactions to visual threat","volume":"557","author":"Salay","year":"2018","journal-title":"Nature"},{"issue":"1\u20132","key":"10.1016\/j.pharmr.2025.100093_bib63","doi-asserted-by":"crossref","first-page":"311","DOI":"10.1016\/j.cell.2016.12.027","article-title":"Integrated control of predatory hunting by the central nucleus of the amygdala","volume":"168","author":"Han","year":"2017","journal-title":"Cell"},{"issue":"7643","key":"10.1016\/j.pharmr.2025.100093_bib64","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1038\/nature21376","article-title":"Prefrontal cortex output circuits guide reward seeking through divergent cue encoding","volume":"543","author":"Otis","year":"2017","journal-title":"Nature"},{"issue":"7731","key":"10.1016\/j.pharmr.2025.100093_bib65","doi-asserted-by":"crossref","first-page":"397","DOI":"10.1038\/s41586-018-0682-1","article-title":"Dopamine enhances signal-to-noise ratio in cortical-brainstem encoding of aversive stimuli","volume":"563","author":"Vander Weele","year":"2018","journal-title":"Nature"},{"issue":"7624","key":"10.1016\/j.pharmr.2025.100093_bib66","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1038\/nature19789","article-title":"A cholinergic basal forebrain feeding circuit modulates appetite suppression","volume":"538","author":"Herman","year":"2016","journal-title":"Nature"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib67","doi-asserted-by":"crossref","first-page":"698","DOI":"10.3390\/v15030698","article-title":"Viral vectors in gene therapy: where do we stand in 2023?","volume":"15","author":"Lundstrom","year":"2023","journal-title":"Viruses"},{"issue":"suppl 1","key":"10.1016\/j.pharmr.2025.100093_bib68","doi-asserted-by":"crossref","first-page":"S139","DOI":"10.1002\/jgm.497","article-title":"CAR chasing: canine adenovirus vectors-all bite and no bark?","volume":"6","author":"Kremer","year":"2004","journal-title":"J Gene Med"},{"issue":"9","key":"10.1016\/j.pharmr.2025.100093_bib69","doi-asserted-by":"crossref","first-page":"936","DOI":"10.1038\/gt.2011.157","article-title":"In utero administration of Ad5 and AAV pseudotypes to the fetal brain leads to efficient, widespread and long-term gene expression","volume":"19","author":"Rahim","year":"2012","journal-title":"Gene Ther"},{"key":"10.1016\/j.pharmr.2025.100093_bib70","article-title":"Oncolytic adenovirus: prospects for cancer immunotherapy","volume":"12","author":"Zhao","year":"2021","journal-title":"Front Microbiol"},{"issue":"22","key":"10.1016\/j.pharmr.2025.100093_bib71","doi-asserted-by":"crossref","first-page":"12394","DOI":"10.1128\/JVI.01068-07","article-title":"Relaxed repression of herpes simplex virus type 1 genomes in Murine trigeminal neurons","volume":"81","author":"Terry-Allison","year":"2007","journal-title":"J Virol"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib72","doi-asserted-by":"crossref","first-page":"1507","DOI":"10.1038\/s41598-017-01635-1","article-title":"Engineered HSV vector achieves safe long-term transgene expression in the central nervous system","volume":"7","author":"Verlengia","year":"2017","journal-title":"Sci Rep"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib73","doi-asserted-by":"crossref","first-page":"176","DOI":"10.1002\/jimd.12697","article-title":"Gene therapy for neurotransmitter-related disorders","volume":"47","author":"Chu","year":"2024","journal-title":"J Inherit Metab Dis"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib74","doi-asserted-by":"crossref","first-page":"S56","DOI":"10.1016\/j.ymgme.2020.12.126","article-title":"Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: update on phase I\/II clinical trial","volume":"132","author":"Kinsella","year":"2021","journal-title":"Mol Genet Metab"},{"issue":"21","key":"10.1016\/j.pharmr.2025.100093_bib75","doi-asserted-by":"crossref","first-page":"1929","DOI":"10.1056\/NEJMoa2106596","article-title":"Hematopoietic stem- and progenitor-cell gene therapy for Hurler syndrome","volume":"385","author":"Gentner","year":"2021","journal-title":"N Engl J Med"},{"key":"10.1016\/j.pharmr.2025.100093_bib76","doi-asserted-by":"crossref","DOI":"10.1038\/mtm.2016.36","article-title":"Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery","volume":"3","author":"Fu","year":"2016","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.pharmr.2025.100093_bib77","doi-asserted-by":"crossref","first-page":"160","DOI":"10.1016\/j.omtm.2018.02.005","article-title":"Development of intrathecal AAV9 gene therapy for giant axonal neuropathy","volume":"9","author":"Bailey","year":"2018","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.pharmr.2025.100093_bib78","doi-asserted-by":"crossref","first-page":"303","DOI":"10.1016\/j.omtm.2023.06.001","article-title":"Adeno-associated virus-mediated gene therapy in a patient with Canavan disease using dual routes of administration and immune modulation","volume":"30","author":"Corti","year":"2023","journal-title":"Mol Ther Methods Clin Dev"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib79","doi-asserted-by":"crossref","first-page":"251","DOI":"10.1038\/s41591-021-01664-4","article-title":"AAV gene therapy for Tay-Sachs disease","volume":"28","author":"Flotte","year":"2022","journal-title":"Nat Med"},{"key":"10.1016\/j.pharmr.2025.100093_bib375","author":"uniQure"},{"key":"10.1016\/j.pharmr.2025.100093_bib374","doi-asserted-by":"crossref","first-page":"4043","DOI":"10.1093\/brain\/awae266","article-title":"Exon 1-targeting miRNA reduces the pathogenic exon 1 HTT protein in Huntington\u2019s disease models","volume":"147","author":"Sogorb-Gonzalez","year":"2024","journal-title":"Brain J. Neurol."},{"issue":"6","key":"10.1016\/j.pharmr.2025.100093_bib80","doi-asserted-by":"crossref","first-page":"506","DOI":"10.1089\/hum.2013.238","article-title":"Intracerebral administration of adeno-associated viral vector serotype rh.10 carrying human SGSH and SUMF1 cDNAs in children with mucopolysaccharidosis type IIIA disease: results of a phase I\/II trial","volume":"25","author":"Tardieu","year":"2014","journal-title":"Hum Gene Ther"},{"issue":"9","key":"10.1016\/j.pharmr.2025.100093_bib81","doi-asserted-by":"crossref","first-page":"712","DOI":"10.1016\/S1474-4422(17)30169-2","article-title":"Intracerebral gene therapy in children with mucopolysaccharidosis type IIIB syndrome: an uncontrolled phase 1\/2 clinical trial","volume":"16","author":"Tardieu","year":"2017","journal-title":"Lancet Neurol"},{"issue":"165","key":"10.1016\/j.pharmr.2025.100093_bib82","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.3003454","article-title":"Long-term follow-up after gene therapy for Canavan disease","volume":"4","author":"Leone","year":"2012","journal-title":"Sci Transl Med"},{"issue":"572","key":"10.1016\/j.pharmr.2025.100093_bib83","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.abb5413","article-title":"Slowing late infantile Batten disease by direct brain parenchymal administration of a rh.10 adeno-associated virus expressing CLN2","volume":"12","author":"Sondhi","year":"2020","journal-title":"Sci Transl Med"},{"issue":"7\u20138","key":"10.1016\/j.pharmr.2025.100093_bib84","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1089\/hum.2019.367","article-title":"Postmortem analysis in a clinical trial of AAV2-NGF gene therapy for Alzheimer\u2019s disease identifies a need for improved vector delivery","volume":"31","author":"Castle","year":"2020","journal-title":"Hum Gene Ther"},{"issue":"20","key":"10.1016\/j.pharmr.2025.100093_bib85","doi-asserted-by":"crossref","first-page":"1662","DOI":"10.1212\/WNL.0b013e3181c29356","article-title":"Safety and tolerability of putaminal AADC gene therapy for Parkinson disease","volume":"73","author":"Christine","year":"2009","journal-title":"Neurology"},{"issue":"9","key":"10.1016\/j.pharmr.2025.100093_bib86","doi-asserted-by":"crossref","first-page":"1731","DOI":"10.1038\/mt.2010.135","article-title":"A phase I study of aromatic L-amino acid decarboxylase gene therapy for Parkinson's disease","volume":"18","author":"Muramatsu","year":"2010","journal-title":"Mol Ther"},{"issue":"9923","key":"10.1016\/j.pharmr.2025.100093_bib87","doi-asserted-by":"crossref","first-page":"1138","DOI":"10.1016\/S0140-6736(13)61939-X","article-title":"Long-term safety and tolerability of ProSavin, a lentiviral vector-based gene therapy for Parkinson\u2019s disease: a dose escalation, open-label, phase 1\/2 trial","volume":"383","author":"Palfi","year":"2014","journal-title":"Lancet"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib88","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1038\/sj.mt.6300010","article-title":"Striatal delivery of neurturin by CERE-120, an AAV2 vector for the treatment of dopaminergic neuron degeneration in Parkinson's disease","volume":"15","author":"Gasmi","year":"2007","journal-title":"Mol Ther"},{"issue":"7","key":"10.1016\/j.pharmr.2025.100093_bib89","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.90133","article-title":"Long-term follow-up of a randomized AAV2-GAD gene therapy trial for Parkinson's disease","volume":"2","author":"Niethammer","year":"2017","journal-title":"JCI Insight"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib90","doi-asserted-by":"crossref","first-page":"3632","DOI":"10.1016\/j.ymthe.2022.08.003","article-title":"Long-term safety of MRI-guided administration of AAV2-GDNF and gadoteridol in the putamen of individuals with Parkinson's disease","volume":"30","author":"Rocco","year":"2022","journal-title":"Mol Ther"},{"issue":"18","key":"10.1016\/j.pharmr.2025.100093_bib91","doi-asserted-by":"crossref","first-page":"1698","DOI":"10.1212\/WNL.0b013e3182904faa","article-title":"Safety\/feasibility of targeting the substantia nigra with AAV2-neurturin in Parkinson patients","volume":"80","author":"Bartus","year":"2013","journal-title":"Neurology"},{"issue":"134","key":"10.1016\/j.pharmr.2025.100093_bib92","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.3003640","article-title":"Gene therapy for aromatic L-amino acid decarboxylase deficiency","volume":"4","author":"Hwu","year":"2012","journal-title":"Sci Transl Med"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib93","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1016\/S2352-4642(17)30125-6","article-title":"Efficacy and safety of AAV2 gene therapy in children with aromatic L-amino acid decarboxylase deficiency: an open-label, phase 1\/2 trial","volume":"1","author":"Chien","year":"2017","journal-title":"Lancet Child Adolesc Health"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib94","doi-asserted-by":"crossref","first-page":"509","DOI":"10.1016\/j.ymthe.2021.11.005","article-title":"Long-term efficacy and safety of eladocagene exuparvovec in patients with AADC deficiency","volume":"30","author":"Tai","year":"2022","journal-title":"Mol Ther"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib95","doi-asserted-by":"crossref","first-page":"644","DOI":"10.1002\/ana.25467","article-title":"Gene therapy improves brain white matter in aromatic L-amino acid decarboxylase deficiency","volume":"85","author":"Tseng","year":"2019","journal-title":"Ann Neurol"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib96","doi-asserted-by":"crossref","first-page":"322","DOI":"10.1093\/brain\/awy331","article-title":"Gene therapy improves motor and mental function of aromatic L-amino acid decarboxylase deficiency","volume":"142","author":"Kojima","year":"2019","journal-title":"Brain"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib97","doi-asserted-by":"crossref","first-page":"4251","DOI":"10.1038\/s41467-021-24524-8","article-title":"Gene therapy for aromatic L-amino acid decarboxylase deficiency by MR-guided direct delivery of AAV2-AADC to midbrain dopaminergic neurons","volume":"12","author":"Pearson","year":"2021","journal-title":"Nat Commun"},{"issue":"6","key":"10.1016\/j.pharmr.2025.100093_bib98","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1097\/CCO.0000000000000995","article-title":"Promises of oncolytic viral therapy for adult and children with brain glioma","volume":"35","author":"Pati\u00f1o-Garc\u00eda","year":"2023","journal-title":"Curr Opin Oncol"},{"key":"10.1016\/j.pharmr.2025.100093_bib99","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1016\/bs.ircmb.2023.04.001","article-title":"Oncolytic viruses as treatment for adult and pediatric high-grade gliomas: on the way to clinical success","volume":"379","author":"Herv\u00e1s-Corpi\u00f3n","year":"2023","journal-title":"Int Rev Cell Mol Biol"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib100","doi-asserted-by":"crossref","first-page":"280","DOI":"10.1073\/pnas.75.1.280","article-title":"Inhibition of rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide","volume":"75","author":"Zamecnik","year":"1978","journal-title":"Proc Natl Acad Sci U S A"},{"key":"10.1016\/j.pharmr.2025.100093_bib101","doi-asserted-by":"crossref","first-page":"307","DOI":"10.1146\/annurev-med-041217-010829","article-title":"Therapeutic antisense oligonucleotides are coming of age","volume":"70","author":"Bennett","year":"2019","journal-title":"Annu Rev Med"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib102","doi-asserted-by":"crossref","first-page":"455","DOI":"10.1038\/s12276-022-00757-5","article-title":"RNA therapy: rich history, various applications and unlimited future prospects","volume":"54","author":"Kim","year":"2022","journal-title":"Exp Mol Med"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib103","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1038\/s41573-024-00912-9","article-title":"RNAi-based drug design: considerations and future directions","volume":"23","author":"Tang","year":"2024","journal-title":"Nat Rev Drug Discov"},{"issue":"8","key":"10.1016\/j.pharmr.2025.100093_bib104","doi-asserted-by":"crossref","first-page":"1759","DOI":"10.1016\/j.ymthe.2020.06.015","article-title":"Delivery of oligonucleotides to the liver with GalNAc: from research to registered therapeutic drug","volume":"28","author":"Debacker","year":"2020","journal-title":"Mol Ther"},{"key":"10.1016\/j.pharmr.2025.100093_bib105","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1186\/s40035-017-0101-9","article-title":"RNAi mechanisms in Huntington's disease therapy: siRNA versus shRNA","volume":"6","author":"Aguiar","year":"2017","journal-title":"Transl Neurodegener"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib106","doi-asserted-by":"crossref","first-page":"5802","DOI":"10.1038\/s41467-022-33061-x","article-title":"Chemical engineering of therapeutic siRNAs for allele-specific gene silencing in Huntington\u2019s disease models","volume":"13","author":"Conroy","year":"2022","journal-title":"Nat Commun"},{"issue":"6","key":"10.1016\/j.pharmr.2025.100093_bib107","doi-asserted-by":"crossref","first-page":"1661","DOI":"10.1016\/j.ymthe.2023.05.006","article-title":"Di-valent siRNA-mediated silencing of MSH3 blocks somatic repeat expansion in mouse models of Huntington\u2019s disease","volume":"31","author":"O'Reilly","year":"2023","journal-title":"Mol Ther"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib108","doi-asserted-by":"crossref","first-page":"347","DOI":"10.1038\/nchembio.1790","article-title":"siRNA screen identifies QPCT as a druggable target for Huntington\u2019s disease","volume":"11","author":"Jimenez-Sanchez","year":"2015","journal-title":"Nat Chem Biol"},{"key":"10.1016\/j.pharmr.2025.100093_bib109","doi-asserted-by":"crossref","first-page":"32","DOI":"10.1016\/j.neulet.2016.07.059","article-title":"Silencing strategies for therapy of SOD1-mediated ALS","volume":"636","author":"van Zundert","year":"2017","journal-title":"Neurosci Lett"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib110","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtn.2024.102291","article-title":"Identification of selective and non-selective C9ORF72 targeting in vivo active siRNAs","volume":"35","author":"Gilbert","year":"2024","journal-title":"Mol Ther Nucleic Acids"},{"issue":"10","key":"10.1016\/j.pharmr.2025.100093_bib111","doi-asserted-by":"crossref","first-page":"1343","DOI":"10.1038\/nn1531","article-title":"Targeting BACE1 with siRNAs ameliorates Alzheimer disease neuropathology in a transgenic model","volume":"8","author":"Singer","year":"2005","journal-title":"Nat Neurosci"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib112","doi-asserted-by":"crossref","first-page":"661","DOI":"10.1093\/nar\/gkh208","article-title":"Targeting Alzheimer's disease genes with RNA interference: an efficient strategy for silencing mutant alleles","volume":"32","author":"Miller","year":"2004","journal-title":"Nucleic Acids Res"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib113","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1016\/j.cell.2016.12.044","article-title":"ApoE2, ApoE3, and ApoE4 differentially stimulate APP transcription and A\u03b2 secretion","volume":"168","author":"Huang","year":"2017","journal-title":"Cell"},{"key":"10.1016\/j.pharmr.2025.100093_bib114","doi-asserted-by":"crossref","DOI":"10.1016\/j.addr.2023.114968","article-title":"siRNA drug delivery across the blood-brain barrier in Alzheimer's disease","volume":"199","author":"Imran Sajid","year":"2023","journal-title":"Adv Drug Deliv Rev"},{"issue":"S24","key":"10.1016\/j.pharmr.2025.100093_bib115","article-title":"Interim phase 1 part A results for ALN-APP, the first investigational RNAi therapeutic in development for Alzheimer\u2019s disease","volume":"19","author":"Cohen","year":"2023","journal-title":"Alzheimers Dem"},{"issue":"18","key":"10.1016\/j.pharmr.2025.100093_bib116","doi-asserted-by":"crossref","first-page":"3446","DOI":"10.1038\/s41388-018-0665-6","article-title":"Mechanisms involved in the activation of C\/EBP\u03b1 by small activating RNA in hepatocellular carcinoma","volume":"38","author":"Zhao","year":"2019","journal-title":"Oncogene"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib117","doi-asserted-by":"crossref","first-page":"151","DOI":"10.4155\/tde-2018-0061","article-title":"Developing small activating RNA as a therapeutic: current challenges and promises","volume":"10","author":"Kwok","year":"2019","journal-title":"Ther Deliv"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib118","doi-asserted-by":"crossref","first-page":"2705","DOI":"10.1016\/j.ymthe.2017.07.018","article-title":"Development and mechanism of small activating RNA targeting CEBPA, a novel therapeutic in clinical trials for liver cancer","volume":"25","author":"Voutila","year":"2017","journal-title":"Mol Ther"},{"key":"10.1016\/j.pharmr.2025.100093_bib119","doi-asserted-by":"crossref","DOI":"10.1038\/srep39311","article-title":"RNA activation of haploinsufficient Foxg1 gene in murine neocortex","volume":"6","author":"Fimiani","year":"2016","journal-title":"Sci Rep"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib120","doi-asserted-by":"crossref","first-page":"591","DOI":"10.3390\/cells10030591","article-title":"Small activating RNAs: towards the development of new therapeutic agents and clinical treatments","volume":"10","author":"Ghanbarian","year":"2021","journal-title":"Cells"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib121","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1093\/nar\/gkab1256","article-title":"Reexamining assumptions about miRNA-guided gene silencing","volume":"50","author":"Kilikevicius","year":"2022","journal-title":"Nucleic Acids Res"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib122","doi-asserted-by":"crossref","first-page":"1469","DOI":"10.3390\/ijms25031469","article-title":"Trials and tribulations of microRNA therapeutics","volume":"25","author":"Seyhan","year":"2024","journal-title":"Int J Mol Sci"},{"key":"10.1016\/j.pharmr.2025.100093_bib123","doi-asserted-by":"crossref","first-page":"127","DOI":"10.1016\/j.jare.2020.08.012","article-title":"Therapeutic advances of miRNAs: a preclinical and clinical update","volume":"28","author":"Chakraborty","year":"2021","journal-title":"J Adv Res"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib124","doi-asserted-by":"crossref","first-page":"314","DOI":"10.3390\/genes14020314","article-title":"miRNAs: the road from bench to bedside","volume":"14","author":"Iacomino","year":"2023","journal-title":"Genes"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib125","article-title":"Design, characterization, and lead selection of therapeutic miRNAs targeting huntingtin for development of gene therapy for Huntington's disease","volume":"5","author":"Miniarikova","year":"2016","journal-title":"Mol Ther Nucleic Acids"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib126","doi-asserted-by":"crossref","first-page":"1","DOI":"10.3233\/JHD-240017","article-title":"Huntington\u2019s disease clinical trials corner: March 2024","volume":"13","author":"Estevez-Fraga","year":"2024","journal-title":"J Huntingtons Dis"},{"key":"10.1016\/j.pharmr.2025.100093_bib127","doi-asserted-by":"crossref","DOI":"10.1016\/j.addr.2021.114002","article-title":"mRNA - A game changer in regenerative medicine, cell-based therapy and reprogramming strategies","volume":"179","author":"Chabanovska","year":"2021","journal-title":"Adv Drug Deliv Rev"},{"issue":"11","key":"10.1016\/j.pharmr.2025.100093_bib128","doi-asserted-by":"crossref","first-page":"1586","DOI":"10.1038\/s41587-022-01491-z","article-title":"Unlocking the promise of mRNA therapeutics","volume":"40","author":"Rohner","year":"2022","journal-title":"Nat Biotechnol"},{"issue":"729","key":"10.1016\/j.pharmr.2025.100093_bib129","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.adh1334","article-title":"mRNA therapy corrects defective glutathione metabolism and restores ureagenesis in preclinical argininosuccinic aciduria","volume":"16","author":"Gurung","year":"2024","journal-title":"Sci Transl Med"},{"issue":"8009","key":"10.1016\/j.pharmr.2025.100093_bib130","doi-asserted-by":"crossref","first-page":"872","DOI":"10.1038\/s41586-024-07266-7","article-title":"Interim analyses of a first-in-human phase 1\/2 mRNA trial for propionic acidaemia","volume":"628","author":"Koeberl","year":"2024","journal-title":"Nature"},{"issue":"9","key":"10.1016\/j.pharmr.2025.100093_bib131","doi-asserted-by":"crossref","first-page":"2934","DOI":"10.1093\/brain\/awae135","article-title":"Unleashing the potential of mRNA therapeutics for inherited neurological diseases","volume":"147","author":"Monfrini","year":"2024","journal-title":"Brain"},{"key":"10.1016\/j.pharmr.2025.100093_bib132","doi-asserted-by":"crossref","DOI":"10.1038\/srep20019","article-title":"Intrathecal delivery of frataxin mRNA encapsulated in lipid nanoparticles to dorsal root ganglia as a potential therapeutic for Friedreich's ataxia","volume":"6","author":"Nabhan","year":"2016","journal-title":"Sci Rep"},{"issue":"13","key":"10.1016\/j.pharmr.2025.100093_bib133","doi-asserted-by":"crossref","first-page":"2189","DOI":"10.2174\/0929867325666181008142831","article-title":"Aptamer-based targeted drug delivery systems: current potential and challenges","volume":"27","author":"He","year":"2020","journal-title":"Curr Med Chem"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib134","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1038\/nrd1955","article-title":"Pegaptanib, a targeted anti-VEGF aptamer for ocular vascular disease","volume":"5","author":"Ng","year":"2006","journal-title":"Nat Rev Drug Discov"},{"issue":"14","key":"10.1016\/j.pharmr.2025.100093_bib135","doi-asserted-by":"crossref","DOI":"10.3390\/ijms241411780","article-title":"Exploring the potential of aptamers in targeting neuroinflammation and neurodegenerative disorders: opportunities and challenges","volume":"24","author":"Kong","year":"2023","journal-title":"Int J Mol Sci"},{"issue":"8","key":"10.1016\/j.pharmr.2025.100093_bib136","doi-asserted-by":"crossref","first-page":"3862","DOI":"10.1021\/jacs.9b11490","article-title":"Enhanced in vivo blood-brain barrier penetration by circular tau-transferrin receptor bifunctional aptamer for tauopathy therapy","volume":"142","author":"Li","year":"2020","journal-title":"J Am Chem Soc"},{"issue":"20","key":"10.1016\/j.pharmr.2025.100093_bib137","doi-asserted-by":"crossref","first-page":"1868","DOI":"10.1056\/NEJMoa2035790","article-title":"100,000 genomes pilot on rare-disease diagnosis in health care\u2014preliminary report","volume":"385","author":"Smedley","year":"2021","journal-title":"N Engl J Med"},{"issue":"7971","key":"10.1016\/j.pharmr.2025.100093_bib138","doi-asserted-by":"crossref","first-page":"828","DOI":"10.1038\/s41586-023-06277-0","article-title":"A framework for individualized splice-switching oligonucleotide therapy","volume":"619","author":"Kim","year":"2023","journal-title":"Nature"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib139","doi-asserted-by":"crossref","DOI":"10.15252\/emmm.202013243","article-title":"Delivery of oligonucleotide-based therapeutics: challenges and opportunities","volume":"13","author":"Hammond","year":"2021","journal-title":"EMBO Mol Med"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib140","doi-asserted-by":"crossref","first-page":"594","DOI":"10.1080\/15476286.2022.2066334","article-title":"Clinical advances of RNA therapeutics for treatment of neurological and neuromuscular diseases","volume":"19","author":"Holm","year":"2022","journal-title":"RNA Biol"},{"issue":"7","key":"10.1016\/j.pharmr.2025.100093_bib141","doi-asserted-by":"crossref","first-page":"1710","DOI":"10.1016\/j.ymthe.2017.05.009","article-title":"Fatty acid-modified gapmer antisense oligonucleotide and serum albumin constructs for pharmacokinetic modulation","volume":"25","author":"Hvam","year":"2017","journal-title":"Mol Ther"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib142","doi-asserted-by":"crossref","first-page":"1130","DOI":"10.3390\/pharmaceutics15041130","article-title":"Enhancing the effectiveness of oligonucleotide therapeutics using cell-penetrating peptide conjugation, chemical modification, and carrier-based delivery strategies","volume":"15","author":"Anwar","year":"2023","journal-title":"Pharmaceutics"},{"issue":"10","key":"10.1016\/j.pharmr.2025.100093_bib143","doi-asserted-by":"crossref","first-page":"673","DOI":"10.1038\/s41573-020-0075-7","article-title":"Advances in oligonucleotide drug delivery","volume":"19","author":"Roberts","year":"2020","journal-title":"Nat Rev Drug Discov"},{"issue":"39","key":"10.1016\/j.pharmr.2025.100093_bib144","doi-asserted-by":"crossref","first-page":"10962","DOI":"10.1073\/pnas.1605731113","article-title":"Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy","volume":"113","author":"Hammond","year":"2016","journal-title":"Proc Natl Acad Sci U S A"},{"key":"10.1016\/j.pharmr.2025.100093_bib145","doi-asserted-by":"crossref","DOI":"10.1016\/j.pneurobio.2019.101665","article-title":"Targeting the transferrin receptor for brain drug delivery","volume":"181","author":"Johnsen","year":"2019","journal-title":"Prog Neurobiol"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib146","doi-asserted-by":"crossref","first-page":"341","DOI":"10.1038\/nbt.1807","article-title":"Delivery of siRNA to the mouse brain by systemic injection of targeted exosomes","volume":"29","author":"Alvarez-Erviti","year":"2011","journal-title":"Nat Biotechnol"},{"issue":"17","key":"10.1016\/j.pharmr.2025.100093_bib147","doi-asserted-by":"crossref","first-page":"1644","DOI":"10.1056\/NEJMoa1813279","article-title":"Patient-customized oligonucleotide therapy for a rare genetic disease","volume":"381","author":"Kim","year":"2019","journal-title":"N Engl J Med"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib148","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1073\/pnas.75.1.285","article-title":"Inhibition of Rous sarcoma viral RNA translation by a specific oligodeoxyribonucleotide","volume":"75","author":"Stephenson","year":"1978","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"6669","key":"10.1016\/j.pharmr.2025.100093_bib149","doi-asserted-by":"crossref","first-page":"806","DOI":"10.1038\/35888","article-title":"Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans","volume":"391","author":"Fire","year":"1998","journal-title":"Nature"},{"issue":"5441","key":"10.1016\/j.pharmr.2025.100093_bib150","doi-asserted-by":"crossref","first-page":"950","DOI":"10.1126\/science.286.5441.950","article-title":"A species of small antisense RNA in posttranscriptional gene silencing in plants","volume":"286","author":"Hamilton","year":"1999","journal-title":"Science"},{"issue":"17","key":"10.1016\/j.pharmr.2025.100093_bib151","doi-asserted-by":"crossref","first-page":"17181","DOI":"10.1074\/jbc.M311683200","article-title":"Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs","volume":"279","author":"Wu","year":"2004","journal-title":"J Biol Chem"},{"issue":"19","key":"10.1016\/j.pharmr.2025.100093_bib152","doi-asserted-by":"crossref","first-page":"9350","DOI":"10.1093\/nar\/gkv964","article-title":"A cytoplasmic pathway for gapmer antisense oligonucleotide-mediated gene silencing in mammalian cells","volume":"43","author":"Castanotto","year":"2015","journal-title":"Nucleic Acids Res"},{"issue":"18","key":"10.1016\/j.pharmr.2025.100093_bib153","doi-asserted-by":"crossref","first-page":"8955","DOI":"10.1093\/nar\/gkv920","article-title":"The rates of the major steps in the molecular mechanism of RNase H1-dependent antisense oligonucleotide induced degradation of RNA","volume":"43","author":"Vickers","year":"2015","journal-title":"Nucleic Acids Res"},{"issue":"9","key":"10.1016\/j.pharmr.2025.100093_bib154","doi-asserted-by":"crossref","first-page":"2075","DOI":"10.1016\/j.ymthe.2017.06.002","article-title":"RNase H1-dependent antisense oligonucleotides are robustly active in directing RNA cleavage in both the cytoplasm and the nucleus","volume":"25","author":"Liang","year":"2017","journal-title":"Mol Ther"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib155","doi-asserted-by":"crossref","first-page":"506","DOI":"10.1016\/j.bbrc.2015.06.171","article-title":"XRN2 is required for the degradation of target RNAs by RNase H1-dependent antisense oligonucleotides","volume":"464","author":"Hori","year":"2015","journal-title":"Biochem Biophys Res Commun"},{"issue":"7","key":"10.1016\/j.pharmr.2025.100093_bib156","doi-asserted-by":"crossref","first-page":"3351","DOI":"10.1093\/nar\/gkw065","article-title":"RNA cleavage products generated by antisense oligonucleotides and siRNAs are processed by the RNA surveillance machinery","volume":"44","author":"Lima","year":"2016","journal-title":"Nucleic Acids Res"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib157","doi-asserted-by":"crossref","first-page":"1113","DOI":"10.1093\/nar\/19.5.1113","article-title":"Inhibition of translation initiation by antisense oligonucleotides via an RNase-H independent mechanism","volume":"19","author":"Boiziau","year":"1991","journal-title":"Nucleic Acids Res"},{"issue":"8","key":"10.1016\/j.pharmr.2025.100093_bib158","doi-asserted-by":"crossref","first-page":"875","DOI":"10.1038\/nbt.3589","article-title":"Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames","volume":"34","author":"Liang","year":"2016","journal-title":"Nat Biotechnol"},{"issue":"16","key":"10.1016\/j.pharmr.2025.100093_bib159","doi-asserted-by":"crossref","first-page":"9528","DOI":"10.1093\/nar\/gkx632","article-title":"Antisense oligonucleotides targeting translation inhibitory elements in 5\u2032 UTRs can selectively increase protein levels","volume":"45","author":"Liang","year":"2017","journal-title":"Nucleic Acids Res"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib160","doi-asserted-by":"crossref","first-page":"6166","DOI":"10.1038\/s41467-023-41799-1","article-title":"Secondary structures that regulate mRNA translation provide insights for ASO-mediated modulation of cardiac hypertrophy","volume":"14","author":"Hedaya","year":"2023","journal-title":"Nat Commun"},{"issue":"13","key":"10.1016\/j.pharmr.2025.100093_bib376","doi-asserted-by":"crossref","first-page":"6900","DOI":"10.1093\/nar\/gkz500","article-title":"mRNA levels can be reduced by antisense oligonucleotides via no-go decay pathway","volume":"47","author":"Liang","year":"2019","journal-title":"Nucleic Acids Res"},{"issue":"7294","key":"10.1016\/j.pharmr.2025.100093_bib161","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1038\/nature09000","article-title":"Deciphering the splicing code","volume":"465","author":"Barash","year":"2010","journal-title":"Nature"},{"issue":"11","key":"10.1016\/j.pharmr.2025.100093_bib162","doi-asserted-by":"crossref","first-page":"917","DOI":"10.1038\/s41573-023-00775-6","article-title":"Therapeutic approaches for Duchenne muscular dystrophy","volume":"22","author":"Roberts","year":"2023","journal-title":"Nat Rev Drug Discov"},{"issue":"10213","key":"10.1016\/j.pharmr.2025.100093_bib163","doi-asserted-by":"crossref","first-page":"2025","DOI":"10.1016\/S0140-6736(19)32910-1","article-title":"Muscular dystrophies","volume":"394","author":"Mercuri","year":"2019","journal-title":"Lancet"},{"issue":"9","key":"10.1016\/j.pharmr.2025.100093_bib164","doi-asserted-by":"crossref","first-page":"5871","DOI":"10.1093\/nar\/gku184","article-title":"Nonsense-mediated decay as a terminating mechanism for antisense oligonucleotides","volume":"42","author":"Ward","year":"2014","journal-title":"Nucleic Acids Res"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib165","doi-asserted-by":"crossref","first-page":"834","DOI":"10.1016\/j.ajhg.2008.01.014","article-title":"Antisense masking of an hnRNP A1\/A2 intronic splicing silencer corrects SMN2 splicing in transgenic mice","volume":"82","author":"Hua","year":"2008","journal-title":"Am J Hum Genet"},{"issue":"72","key":"10.1016\/j.pharmr.2025.100093_bib166","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.3001777","article-title":"Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy","volume":"3","author":"Passini","year":"2011","journal-title":"Sci Transl Med"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib168","doi-asserted-by":"crossref","first-page":"1099","DOI":"10.1056\/NEJMoa2204705","article-title":"Trial of antisense oligonucleotide tofersen for SOD1 ALS","volume":"387","author":"Miller","year":"2022","journal-title":"N Engl J Med"},{"issue":"8","key":"10.1016\/j.pharmr.2025.100093_bib169","doi-asserted-by":"crossref","first-page":"3558","DOI":"10.1172\/JCI99081","article-title":"Antisense oligonucleotides extend survival and reverse decrement in muscle response in ALS models","volume":"128","author":"McCampbell","year":"2018","journal-title":"J Clin Invest"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib172","doi-asserted-by":"crossref","first-page":"117","DOI":"10.1038\/s41591-021-01557-6","article-title":"Suppression of mutant C9orf72 expression by a potent mixed backbone antisense oligonucleotide","volume":"28","author":"Tran","year":"2022","journal-title":"Nat Med"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib173","doi-asserted-by":"crossref","first-page":"104","DOI":"10.1038\/s41591-021-01615-z","article-title":"Antisense oligonucleotide silencing of FUS expression as a therapeutic approach in amyotrophic lateral sclerosis","volume":"28","author":"Korobeynikov","year":"2022","journal-title":"Nat Med"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib174","doi-asserted-by":"crossref","first-page":"1159","DOI":"10.1007\/s13311-022-01285-w","article-title":"Approaches to gene modulation therapy for ALS","volume":"19","author":"Meijboom","year":"2022","journal-title":"Neurotherapeutics"},{"issue":"23","key":"10.1016\/j.pharmr.2025.100093_bib175","doi-asserted-by":"crossref","first-page":"2203","DOI":"10.1056\/NEJMc2300400","article-title":"Tominersen in adults with manifest Huntington's disease","volume":"389","author":"McColgan","year":"2023","journal-title":"N Engl J Med"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib176","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1007\/s40259-022-00519-9","article-title":"Antisense oligonucleotide therapy: from design to the Huntington disease clinic","volume":"36","author":"Rook","year":"2022","journal-title":"BioDrugs"},{"key":"10.1016\/j.pharmr.2025.100093_bib179","doi-asserted-by":"crossref","first-page":"623","DOI":"10.1016\/j.omtn.2020.06.027","article-title":"Splice-switching antisense oligonucleotides reduce LRRK2 kinase activity in human LRRK2 transgenic mice","volume":"21","author":"Korecka","year":"2020","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.pharmr.2025.100093_bib180","author":"Meglio"},{"key":"10.1016\/j.pharmr.2025.100093_bib181","author":"Novartis"},{"issue":"6","key":"10.1016\/j.pharmr.2025.100093_bib182","doi-asserted-by":"crossref","first-page":"1437","DOI":"10.1038\/s41591-023-02326-3","article-title":"Tau-targeting antisense oligonucleotide MAPTRx in mild Alzheimer's disease: a phase 1b, randomized, placebo-controlled trial","volume":"29","author":"Mummery","year":"2023","journal-title":"Nat Med"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib183","doi-asserted-by":"crossref","first-page":"1344","DOI":"10.1001\/jamaneurol.2023.3861","article-title":"Exploratory tau biomarker results from a multiple ascending-dose study of BIIB080 in Alzheimer disease: a randomized clinical trial","volume":"80","author":"Edwards","year":"2023","journal-title":"JAMA Neurol"},{"issue":"558","key":"10.1016\/j.pharmr.2025.100093_bib184","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.aaz6100","article-title":"Antisense oligonucleotides increase Scn1a expression and reduce seizures and SUDEP incidence in a mouse model of Dravet syndrome","volume":"12","author":"Han","year":"2020","journal-title":"Sci Transl Med"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib185","doi-asserted-by":"crossref","first-page":"3501","DOI":"10.1038\/s41467-020-17093-9","article-title":"Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression","volume":"11","author":"Lim","year":"2020","journal-title":"Nat Commun"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib186","doi-asserted-by":"crossref","first-page":"1231","DOI":"10.1093\/brain\/awad349","article-title":"Antisense oligonucleotides restore excitability, GABA signalling and sodium current density in a Dravet syndrome model","volume":"147","author":"Yuan","year":"2024","journal-title":"Brain"},{"issue":"23","key":"10.1016\/j.pharmr.2025.100093_bib189","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.146090","article-title":"Antisense oligonucleotide therapy for KCNT1 encephalopathy","volume":"7","author":"Burbano","year":"2022","journal-title":"JCI Insight"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib190","doi-asserted-by":"crossref","first-page":"754","DOI":"10.1002\/ana.26861","article-title":"Long-term downregulation of the sodium channel gene Scn8a is therapeutic in mouse models of SCN8A epilepsy","volume":"95","author":"Hill","year":"2024","journal-title":"Ann Neurol"},{"issue":"23","key":"10.1016\/j.pharmr.2025.100093_bib191","doi-asserted-by":"crossref","DOI":"10.1172\/JCI152079","article-title":"Antisense oligonucleotide therapy reduces seizures and extends life span in an SCN2A gain-of-function epilepsy model","volume":"131","author":"Li","year":"2021","journal-title":"J Clin Invest"},{"issue":"10","key":"10.1016\/j.pharmr.2025.100093_bib192","doi-asserted-by":"crossref","first-page":"e125","DOI":"10.1111\/epi.17374","article-title":"Genetic interaction between Scn8a and potassium channel genes Kcna1 and Kcnq2","volume":"63","author":"Hill","year":"2022","journal-title":"Epilepsia"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib193","doi-asserted-by":"crossref","first-page":"339","DOI":"10.1002\/ana.25676","article-title":"Scn8a antisense oligonucleotide is protective in mouse models of SCN8A encephalopathy and Dravet syndrome","volume":"87","author":"Lenk","year":"2020","journal-title":"Ann Neurol"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib194","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1016\/j.cell.2004.09.011","article-title":"Ca(V)1.2 calcium channel dysfunction causes a multisystem disorder including arrhythmia and autism","volume":"119","author":"Splawski","year":"2004","journal-title":"Cell"},{"issue":"8009","key":"10.1016\/j.pharmr.2025.100093_bib195","doi-asserted-by":"crossref","first-page":"818","DOI":"10.1038\/s41586-024-07310-6","article-title":"Antisense oligonucleotide therapeutic approach for Timothy syndrome","volume":"628","author":"Chen","year":"2024","journal-title":"Nature"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib196","doi-asserted-by":"crossref","first-page":"834","DOI":"10.1136\/jmg.38.12.834","article-title":"Distinct phenotypes distinguish the molecular classes of Angelman syndrome","volume":"38","author":"Lossie","year":"2001","journal-title":"J Med Genet"},{"issue":"23","key":"10.1016\/j.pharmr.2025.100093_bib197","doi-asserted-by":"crossref","first-page":"2687","DOI":"10.1093\/hmg\/10.23.2687","article-title":"The IC-SNURF-SNRPN transcript serves as a host for multiple small nucleolar RNA species and as an antisense RNA for UBE3A","volume":"10","author":"Runte","year":"2001","journal-title":"Hum Mol Genet"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib198","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pgen.1004039","article-title":"Truncation of Ube3a-ATS unsilences paternal Ube3a and ameliorates behavioral defects in the Angelman syndrome mouse model","volume":"9","author":"Meng","year":"2013","journal-title":"PLoS Genet"},{"issue":"688","key":"10.1016\/j.pharmr.2025.100093_bib199","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.abf4077","article-title":"An ASO therapy for Angelman syndrome that targets an evolutionarily conserved region at the start of the UBE3A-AS transcript","volume":"15","author":"Dindot","year":"2023","journal-title":"Sci Transl Med"},{"issue":"15","key":"10.1016\/j.pharmr.2025.100093_bib200","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.145991","article-title":"Antisense oligonucleotide treatment rescues UBE3A expression and multiple phenotypes of an Angelman syndrome mouse model","volume":"6","author":"Milazzo","year":"2021","journal-title":"JCI Insight"},{"key":"10.1016\/j.pharmr.2025.100093_bib202","unstructured":"Roche\u2019s rugonersen trials: Angelman syndrome update. FAST; 2024."},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib203","doi-asserted-by":"crossref","first-page":"83","DOI":"10.1089\/nat.2021.0039","article-title":"Preparing n-of-1 antisense oligonucleotide treatments for rare neurological diseases in Europe: genetic, regulatory, and ethical perspectives","volume":"32","author":"Synofzik","year":"2022","journal-title":"Nucleic Acid Ther"},{"issue":"6","key":"10.1016\/j.pharmr.2025.100093_bib204","doi-asserted-by":"crossref","first-page":"811","DOI":"10.1007\/s00401-014-1269-z","article-title":"TDP-43 is a key player in the clinical features associated with Alzheimer's disease","volume":"127","author":"Josephs","year":"2014","journal-title":"Acta Neuropathol"},{"key":"10.1016\/j.pharmr.2025.100093_bib205","doi-asserted-by":"crossref","first-page":"25","DOI":"10.3389\/fnmol.2019.00025","article-title":"Molecular mechanisms of TDP-43 misfolding and pathology in amyotrophic lateral sclerosis","volume":"12","author":"Prasad","year":"2019","journal-title":"Front Mol Neurosci"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib206","doi-asserted-by":"crossref","DOI":"10.1172\/JCI140723","article-title":"Huntington's disease mice and human brain tissue exhibit increased G3BP1 granules and TDP43 mislocalization","volume":"131","author":"Sanchez","year":"2021","journal-title":"J Clin Invest"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib207","doi-asserted-by":"crossref","first-page":"813","DOI":"10.1007\/s00401-019-02042-8","article-title":"Splicing repression is a major function of TDP-43 in motor neurons","volume":"138","author":"Donde","year":"2019","journal-title":"Acta Neuropathol"},{"issue":"6637","key":"10.1016\/j.pharmr.2025.100093_bib377","doi-asserted-by":"crossref","first-page":"1140","DOI":"10.1126\/science.abq5622","article-title":"Mechanism of STMN2 cryptic splice-polyadenylation and its correction for TDP-43 proteinopathies","volume":"379","author":"Baughn","year":"2023","journal-title":"Science"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib208","doi-asserted-by":"crossref","first-page":"266","DOI":"10.1038\/s41392-023-01533-1","article-title":"Restoring expression of Stathmin-2: a novel strategy to treat TDP-43 proteinopathies","volume":"8","author":"Menge","year":"2023","journal-title":"Signal Transduct Target Ther"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib378","doi-asserted-by":"crossref","DOI":"10.15252\/emmm.202217157","article-title":"ASO targeting RBM3 temperature-controlled poison exon splicing prevents neurodegeneration in vivo","volume":"15","author":"Preu\u00dfner","year":"2023","journal-title":"EMBO Mol Med"},{"key":"10.1016\/j.pharmr.2025.100093_bib379","doi-asserted-by":"crossref","first-page":"731","DOI":"10.1016\/j.omtn.2020.12.027","article-title":"Targeting the 5\u2019 untranslated region of SMN2 as a therapeutic strategy for spinal muscular atrophy","volume":"23","author":"Winkelsas","year":"2021","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.pharmr.2025.100093_bib210","doi-asserted-by":"crossref","DOI":"10.1016\/j.nbd.2022.105795","article-title":"Combinatorial ASO-mediated therapy with low dose SMN and the protective modifier Chp1 is not sufficient to ameliorate SMA pathology hallmarks","volume":"171","author":"Muinos-B\u00fchl","year":"2022","journal-title":"Neurobiol Dis"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib211","doi-asserted-by":"crossref","first-page":"4198","DOI":"10.3390\/ijms24044198","article-title":"Long-term SMN- and Ncald-ASO combinatorial therapy in SMA mice and NCALD-ASO treatment in hiPSC-derived motor neurons show protective effects","volume":"24","author":"Mui\u00f1os-B\u00fchl","year":"2023","journal-title":"Int J Mol Sci"},{"issue":"10","key":"10.1016\/j.pharmr.2025.100093_bib212","doi-asserted-by":"crossref","first-page":"5235","DOI":"10.1093\/nar\/gkaa299","article-title":"Phosphorothioate modified oligonucleotide-protein interactions","volume":"48","author":"Crooke","year":"2020","journal-title":"Nucleic Acids Res"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib213","doi-asserted-by":"crossref","first-page":"1229","DOI":"10.1073\/pnas.1813376116","article-title":"Mesyl phosphoramidate antisense oligonucleotides as an alternative to phosphorothioates with improved biochemical and biological properties","volume":"116","author":"Miroshnichenko","year":"2019","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib214","doi-asserted-by":"crossref","first-page":"190","DOI":"10.1089\/nat.2020.0860","article-title":"Mesyl phosphoramidate oligonucleotides as potential splice-switching agents: impact of backbone structure on activity and intracellular localization","volume":"31","author":"Hammond","year":"2021","journal-title":"Nucleic Acid Ther"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib215","doi-asserted-by":"crossref","first-page":"1691","DOI":"10.1093\/nar\/gkaa031","article-title":"Understanding the effect of controlling phosphorothioate chirality in the DNA gap on the potency and safety of gapmer antisense oligonucleotides","volume":"48","author":"\u00d8stergaard","year":"2020","journal-title":"Nucleic Acids Res"},{"issue":"10","key":"10.1016\/j.pharmr.2025.100093_bib216","doi-asserted-by":"crossref","first-page":"5401","DOI":"10.1093\/nar\/gkac037","article-title":"Impact of guanidine-containing backbone linkages on stereopure antisense oligonucleotides in the CNS","volume":"50","author":"Kandasamy","year":"2022","journal-title":"Nucleic Acids Res"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib217","doi-asserted-by":"crossref","first-page":"401","DOI":"10.1089\/nat.2022.0005","article-title":"The combination of Mesyl-Phosphoramidate inter-nucleotide linkages and 2\u2032-O-methyl in selected positions in the antisense oligonucleotide enhances the performance of RNaseH1 active PS-ASOs","volume":"32","author":"Zhang","year":"2022","journal-title":"Nucleic Acid Ther"},{"key":"10.1016\/j.pharmr.2025.100093_bib218","doi-asserted-by":"crossref","first-page":"558","DOI":"10.1016\/j.omtn.2022.04.007","article-title":"Preclinical evaluation of WVE-004, aninvestigational stereopure oligonucleotide forthe treatment of C9orf72-associated ALS or FTD","volume":"28","author":"Liu","year":"2022","journal-title":"Mol Ther Nucleic Acids"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib219","doi-asserted-by":"crossref","first-page":"380","DOI":"10.1134\/S1068162021020291","article-title":"Synthesis of novel representatives of phosphoryl guanidine oligonucleotides","volume":"47","author":"Zhukov","year":"2021","journal-title":"Russ J Bioorg Chem"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib220","doi-asserted-by":"crossref","first-page":"1828","DOI":"10.1093\/nar\/gkab047","article-title":"Site-specific incorporation of 5\u2032-methyl DNA enhances the therapeutic profile of gapmer ASOs","volume":"49","author":"Vasquez","year":"2021","journal-title":"Nucleic Acids Res"},{"issue":"7","key":"10.1016\/j.pharmr.2025.100093_bib221","doi-asserted-by":"crossref","first-page":"3513","DOI":"10.1073\/pnas.96.7.3513","article-title":"A cytosine analog that confers enhanced potency to antisense oligonucleotides","volume":"96","author":"Flanagan","year":"1999","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"13","key":"10.1016\/j.pharmr.2025.100093_bib222","doi-asserted-by":"crossref","first-page":"7224","DOI":"10.1093\/nar\/gkac562","article-title":"Identification of nucleobase chemical modifications that reduce the hepatotoxicity of gapmer antisense oligonucleotides","volume":"50","author":"Yoshida","year":"2022","journal-title":"Nucleic Acids Res"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib223","article-title":"Favorable efficacy and reduced acute neurotoxicity by antisense oligonucleotides with 2\u2032,4\u2032-BNA\/LNA with 9-(aminoethoxy)phenoxazine","volume":"35","author":"Matsubayashi","year":"2024","journal-title":"Mol Ther Nucleic Acids"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib224","doi-asserted-by":"crossref","DOI":"10.1002\/hlca.202200169","article-title":"The MOE modification of RNA: origins and widescale impact on the oligonucleotide therapeutics field","volume":"106","author":"Hill","year":"2023","journal-title":"Helv Chim Acta"},{"issue":"19","key":"10.1016\/j.pharmr.2025.100093_bib225","doi-asserted-by":"crossref","first-page":"14514","DOI":"10.1016\/S0021-9258(19)85268-7","article-title":"Evaluation of 2\u2032-modified oligonucleotides containing 2\u2032-deoxy gaps as antisense inhibitors of gene-expression","volume":"268","author":"Monia","year":"1993","journal-title":"J Biol Chem"},{"key":"10.1016\/j.pharmr.2025.100093_bib226","series-title":"Antisense Drug Technology: Principles, Strategies, and Applications","author":"Lima","year":"2007"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib227","doi-asserted-by":"crossref","first-page":"910","DOI":"10.1016\/j.neuron.2016.02.003","article-title":"The biology of huntingtin","volume":"89","author":"Saudou","year":"2016","journal-title":"Neuron"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib228","doi-asserted-by":"crossref","first-page":"313","DOI":"10.1038\/nm.4490","article-title":"Haploinsufficiency leads to neurodegeneration in C9ORF72 ALS\/FTD human induced motor neurons","volume":"24","author":"Shi","year":"2018","journal-title":"Nat Med"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib229","first-page":"235","article-title":"Phosphorodiamidate morpholino oligomers: favorable properties for sequence-specific gene inactivation","volume":"3","author":"Iversen","year":"2001","journal-title":"Curr Opin Mol Ther"},{"issue":"6","key":"10.1016\/j.pharmr.2025.100093_bib230","doi-asserted-by":"crossref","first-page":"2853","DOI":"10.1093\/nar\/gkaa126","article-title":"Comparison of the efficacy of MOE and PMO modifications of systemic antisense oligonucleotides in a severe SMA mouse model","volume":"48","author":"Sheng","year":"2020","journal-title":"Nucleic Acids Res"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib231","doi-asserted-by":"crossref","DOI":"10.1038\/s41598-023-38698-2","article-title":"Convergent synthesis of phosphorodiamidate morpholino oligonucleotides (PMOs) by the H-phosphonate approach","volume":"13","author":"Tsurusaki","year":"2023","journal-title":"Sci Rep"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib380","doi-asserted-by":"crossref","first-page":"4036","DOI":"10.1038\/s41467-022-31636-2","article-title":"An LNA-amide modification that enhances the cell uptake and activity of phosphorothioate exon-skipping oligonucleotides","volume":"13","author":"Baker","year":"2022","journal-title":"Nat Commun"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib232","doi-asserted-by":"crossref","first-page":"245","DOI":"10.1089\/nat.2019.0794","article-title":"Lipid conjugates enhance endosomal release of antisense oligonucleotides into cells","volume":"29","author":"Wang","year":"2019","journal-title":"Nucleic Acid Ther"},{"issue":"9","key":"10.1016\/j.pharmr.2025.100093_bib233","doi-asserted-by":"crossref","first-page":"4658","DOI":"10.1093\/nar\/gkaa229","article-title":"Delivery of oligonucleotides to bone marrow to modulate ferrochelatase splicing in a mouse model of erythropoietic protoporphyria","volume":"48","author":"Halloy","year":"2020","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.pharmr.2025.100093_bib234","doi-asserted-by":"crossref","first-page":"1357","DOI":"10.1016\/j.omtn.2020.01.018","article-title":"Efficient delivery of antisense oligonucleotides using bioreducible lipid nanoparticles in vitro and in vivo","volume":"19","author":"Yang","year":"2020","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.pharmr.2025.100093_bib235","doi-asserted-by":"crossref","first-page":"773","DOI":"10.1016\/j.omtn.2023.05.005","article-title":"Lipid nanoparticle delivery limits antisense oligonucleotide activity and cellular distribution in the brain after intracerebroventricular injection","volume":"32","author":"Byrnes","year":"2023","journal-title":"Mol Ther Nucleic Acids"},{"key":"10.1016\/j.pharmr.2025.100093_bib236","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1016\/j.jconrel.2024.01.011","article-title":"Systemic delivery of mutant huntingtin lowering antisense oligonucleotides to the brain using apolipoprotein A-I nanodisks for Huntington disease","volume":"367","author":"Caron","year":"2024","journal-title":"J Control Release"},{"issue":"13","key":"10.1016\/j.pharmr.2025.100093_bib237","doi-asserted-by":"crossref","first-page":"8796","DOI":"10.1093\/nar\/gku531","article-title":"Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice","volume":"42","author":"Prakash","year":"2014","journal-title":"Nucleic Acids Res"},{"issue":"10","key":"10.1016\/j.pharmr.2025.100093_bib238","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.aat3386","article-title":"Targeted delivery of antisense oligonucleotides to pancreatic \u03b2-cells","volume":"4","author":"\u00c4mm\u00e4l\u00e4","year":"2018","journal-title":"Sci Adv"},{"issue":"15","key":"10.1016\/j.pharmr.2025.100093_bib239","doi-asserted-by":"crossref","first-page":"8471","DOI":"10.1021\/acs.jmedchem.0c00840","article-title":"Targeted delivery of antisense oligonucleotides using neurotensin peptides","volume":"63","author":"Nikan","year":"2020","journal-title":"J Med Chem"},{"key":"10.1016\/j.pharmr.2025.100093_bib257","doi-asserted-by":"crossref","DOI":"10.1016\/j.ebiom.2020.102944","article-title":"Anti-\u03b1-synuclein ASO delivered to monoamine neurons prevents \u03b1-synuclein accumulation in a Parkinson's disease-like mouse model and in monkeys","volume":"59","author":"Alarc\u00f3n-Ar\u00eds","year":"2020","journal-title":"EBiomedicine"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib240","doi-asserted-by":"crossref","first-page":"147","DOI":"10.1021\/bc200377d","article-title":"Cellular uptake and intracellular trafficking of antisense and siRNA oligonucleotides","volume":"23","author":"Juliano","year":"2012","journal-title":"Bioconjug Chem"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib241","doi-asserted-by":"crossref","first-page":"361","DOI":"10.1089\/nat.2022.0004","article-title":"Delivery of RNA therapeutics: the great endosomal escape","volume":"32","author":"Dowdy","year":"2022","journal-title":"Nucleic Acid Ther"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib242","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1093\/nar\/gkaa1112","article-title":"High-resolution visualization and quantification of nucleic acid-based therapeutics in cells and tissues using nanoscale secondary ion mass spectrometry (NanoSIMS)","volume":"49","author":"He","year":"2021","journal-title":"Nucleic Acids Res"},{"issue":"39","key":"10.1016\/j.pharmr.2025.100093_bib243","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.abo2954","article-title":"In vivo peptide-based delivery of a gene-modifying enzyme into cells of the central nervous system","volume":"8","author":"Allen","year":"2022","journal-title":"Sci Adv"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib244","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1038\/s42003-022-03132-2","article-title":"Novel endosomolytic compounds enable highly potent delivery of antisense oligonucleotides","volume":"5","author":"Bost","year":"2022","journal-title":"Commun Biol"},{"issue":"18","key":"10.1016\/j.pharmr.2025.100093_bib245","doi-asserted-by":"crossref","first-page":"2253","DOI":"10.3390\/cells12182253","article-title":"Therapeutic oligonucleotides: an outlook on chemical strategies to improve endosomal trafficking","volume":"12","author":"Mangla","year":"2023","journal-title":"Cells"},{"key":"10.1016\/j.pharmr.2025.100093_bib246","doi-asserted-by":"crossref","first-page":"17","DOI":"10.1016\/j.omtn.2022.08.019","article-title":"A cell-penetrating peptide enhances delivery and efficacy of phosphorodiamidate morpholino oligomers in mdx mice","volume":"30","author":"Gan","year":"2022","journal-title":"Mol Ther Nucleic Acids"},{"issue":"27","key":"10.1016\/j.pharmr.2025.100093_bib247","doi-asserted-by":"crossref","DOI":"10.1073\/pnas.2301067120","article-title":"Lipid nanoparticle topology regulates endosomal escape and delivery of RNA to the cytoplasm","volume":"120","author":"Zheng","year":"2023","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib248","doi-asserted-by":"crossref","first-page":"3889","DOI":"10.1016\/j.apsb.2021.03.042","article-title":"Antibody-drug conjugates: recent advances in linker chemistry","volume":"11","author":"Su","year":"2021","journal-title":"Acta Pharm Sin B"},{"issue":"20","key":"10.1016\/j.pharmr.2025.100093_bib249","doi-asserted-by":"crossref","first-page":"11401","DOI":"10.1093\/nar\/gkac641","article-title":"Enhanced exon skipping and prolonged dystrophin restoration achieved by TfR1-targeted delivery of antisense oligonucleotide using FORCE conjugation in mdx mice","volume":"50","author":"Desjardins","year":"2022","journal-title":"Nucleic Acids Res"},{"key":"10.1016\/j.pharmr.2025.100093_bib250","doi-asserted-by":"crossref","first-page":"46","DOI":"10.1016\/j.addr.2015.01.008","article-title":"Pharmacokinetics, biodistribution and cell uptake of antisense oligonucleotides","volume":"87","author":"Geary","year":"2015","journal-title":"Adv Drug Deliv Rev"},{"issue":"suppl 1","key":"10.1016\/j.pharmr.2025.100093_bib251","doi-asserted-by":"crossref","first-page":"S3","DOI":"10.1186\/1471-2377-9-S1-S3","article-title":"Characteristics of compounds that cross the blood-brain barrier","volume":"9","author":"Banks","year":"2009","journal-title":"BMC Neurol"},{"issue":"20","key":"10.1016\/j.pharmr.2025.100093_bib381","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.129240","article-title":"Brain pharmacology of intrathecal antisense oligonucleotides revealed through multimodal imaging","volume":"4","author":"Mazur","year":"2019","journal-title":"JCI Insight"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib382","doi-asserted-by":"crossref","first-page":"657","DOI":"10.1093\/nar\/gkaa1235","article-title":"The atlas of RNase H antisense oligonucleotide distribution and activity in the CNS of rodents and non-human primates following central administration","volume":"49","author":"Jafar-Nejad","year":"2021","journal-title":"Nucleic Acids Res"},{"issue":"14","key":"10.1016\/j.pharmr.2025.100093_bib383","doi-asserted-by":"crossref","first-page":"7109","DOI":"10.1093\/nar\/gkad371","article-title":"A single-cell map of antisense oligonucleotide activity in the brain","volume":"51","author":"Mortberg","year":"2023","journal-title":"Nucleic Acids Res"},{"issue":"24","key":"10.1016\/j.pharmr.2025.100093_bib252","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.152203","article-title":"Comparative route of administration studies using therapeutic siRNAs show widespread gene modulation in Dorset sheep","volume":"6","author":"Ferguson","year":"2021","journal-title":"JCI Insight"},{"key":"10.1016\/j.pharmr.2025.100093_bib253","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2023.101122","article-title":"Awake intracerebroventricular delivery and safety assessment of oligonucleotides in a large animal model","volume":"31","author":"Benatti","year":"2023","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.pharmr.2025.100093_bib254","author":"Veritas"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib255","doi-asserted-by":"crossref","first-page":"184","DOI":"10.1016\/j.ymgme.2018.05.003","article-title":"Best practices for the use of intracerebroventricular drug delivery devices","volume":"124","author":"Slavc","year":"2018","journal-title":"Mol Genet Metab"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib256","doi-asserted-by":"crossref","first-page":"51","DOI":"10.1016\/j.bjae.2019.11.002","article-title":"Complications associated with intrathecal drug delivery systems","volume":"20","author":"Delhaas","year":"2020","journal-title":"BJA Educ"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib258","doi-asserted-by":"crossref","first-page":"1500","DOI":"10.1097\/j.pain.0000000000002154","article-title":"Intranasal delivery of an antisense oligonucleotide to the RNA-binding protein HuR relieves nerve injury-induced neuropathic pain","volume":"162","author":"Borgonetti","year":"2021","journal-title":"Pain"},{"key":"10.1016\/j.pharmr.2025.100093_bib259","first-page":"39","article-title":"Structural and functional aspects of the blood-brain barrier","volume":"61","author":"Begley","year":"2003","journal-title":"Prog Drug Res"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib260","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1007\/s00418-020-01884-8","article-title":"Developmental patterns in human blood-brain barrier and blood-cerebrospinal fluid barrier ABC drug transporter expression","volume":"154","author":"Verscheijden","year":"2020","journal-title":"Histochem Cell Biol"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib261","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1111\/dmcn.14027","article-title":"Nusinersen treatment of spinal muscular atrophy: current knowledge and existing gaps","volume":"61","author":"Gidaro","year":"2019","journal-title":"Dev Med Child Neurol"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib262","doi-asserted-by":"crossref","first-page":"328","DOI":"10.1111\/nan.12408","article-title":"Age-associated changes in the blood-brain barrier: comparative studies in human and mouse","volume":"44","author":"Goodall","year":"2018","journal-title":"Neuropathol Appl Neurobiol"},{"key":"10.1016\/j.pharmr.2025.100093_bib263","doi-asserted-by":"crossref","first-page":"366","DOI":"10.1016\/j.omtm.2020.12.006","article-title":"Rapid evolution of blood-brain-barrier-penetrating AAV capsids by RNA-driven biopanning","volume":"20","author":"Nonnenmacher","year":"2021","journal-title":"Mol Ther Methods Clin Dev"},{"key":"10.1016\/j.pharmr.2025.100093_bib264","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1016\/j.omtm.2022.09.002","article-title":"Selection of rAAV vectors that cross the human blood-brain barrier and target the central nervous system using a transwell model","volume":"27","author":"Song","year":"2022","journal-title":"Mol Ther Methods Clin Dev"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib265","doi-asserted-by":"crossref","first-page":"46","DOI":"10.1186\/s12987-023-00447-y","article-title":"Ultrasound-mediated blood-brain barrier opening uncovers an intracerebral perivenous fluid network in persons with Alzheimer's disease","volume":"20","author":"Mehta","year":"2023","journal-title":"Fluids Barriers CNS"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib266","doi-asserted-by":"crossref","first-page":"137","DOI":"10.1186\/s13578-023-01079-3","article-title":"Cutting-edge advances in modeling the blood-brain barrier and tools for its reversible permeabilization for enhanced drug delivery into the brain","volume":"13","author":"Sharma","year":"2023","journal-title":"Cell Biosci"},{"issue":"19","key":"10.1016\/j.pharmr.2025.100093_bib267","doi-asserted-by":"crossref","first-page":"2395","DOI":"10.3390\/cells12192395","article-title":"Enhancing antisense oligonucleotide-based therapeutic delivery with DG9, a versatile cell-penetrating peptide","volume":"12","author":"Haque","year":"2023","journal-title":"Cells"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib268","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.160516","article-title":"DG9-conjugated morpholino rescues phenotype in SMA mice by reaching the CNS via a subcutaneous administration","volume":"8","author":"Aslesh","year":"2023","journal-title":"JCI Insight"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib269","doi-asserted-by":"crossref","first-page":"51","DOI":"10.3390\/biomedicines6020051","article-title":"Cell-penetrating peptides to enhance delivery of oligonucleotide-based therapeutics","volume":"6","author":"McClorey","year":"2018","journal-title":"Biomedicines"},{"issue":"24","key":"10.1016\/j.pharmr.2025.100093_bib270","doi-asserted-by":"crossref","DOI":"10.1172\/jci.insight.154142","article-title":"Antibody-oligonucleotide conjugate achieves CNS delivery in animal models for spinal muscular atrophy","volume":"7","author":"Hammond","year":"2022","journal-title":"JCI Insight"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib271","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1186\/s12987-023-00462-z","article-title":"Enhanced in vivo blood brain barrier transcytosis of macromolecular cargo using an engineered pH-sensitive mouse transferrin receptor binding nanobody","volume":"20","author":"Esparza","year":"2023","journal-title":"Fluids Barriers CNS"},{"key":"10.1016\/j.pharmr.2025.100093_bib272","doi-asserted-by":"crossref","unstructured":"Barker SJ, Thayer MB, Kim C, et al. Targeting transferrin receptor to transport antisense oligonucleotides across the blood-brain barrier. Preprint. Posted April 28, 2023. bioRxiv 2023.04.25.538145. https:\/\/doi.org\/10.1101\/2023.04.25.538145.","DOI":"10.1101\/2023.04.25.538145"},{"key":"10.1016\/j.pharmr.2025.100093_bib273","doi-asserted-by":"crossref","first-page":"913","DOI":"10.1016\/j.jconrel.2023.07.027","article-title":"Delivery of mutant huntingtin-lowering antisense oligonucleotides to the brain by intranasally administered apolipoprotein A-I nanodisks","volume":"360","author":"Aly","year":"2023","journal-title":"J Control Release"},{"issue":"21","key":"10.1016\/j.pharmr.2025.100093_bib384","first-page":"8173","article-title":"Systemic brain delivery of antisense oligonucleotides across the blood-brain barrier with a glucose-coated polymeric nanocarrier. Angew Chem Int","volume":"59","author":"Min","year":"2020","journal-title":"Engl"},{"issue":"30","key":"10.1016\/j.pharmr.2025.100093_bib274","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.abb4429","article-title":"Neurotransmitter-derived lipidoids (NT-lipidoids) for enhanced brain delivery through intravenous injection","volume":"6","author":"Ma","year":"2020","journal-title":"Sci Adv"},{"issue":"9","key":"10.1016\/j.pharmr.2025.100093_bib275","doi-asserted-by":"crossref","first-page":"366","DOI":"10.1021\/ar00057a002","article-title":"Antisense oligonucleotides","volume":"28","author":"De Mesmaeker","year":"2002","journal-title":"Acc Chem Res"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib276","doi-asserted-by":"crossref","first-page":"77","DOI":"10.1007\/s40291-020-00504-4","article-title":"Using RNA-seq to assess off-target effects of antisense oligonucleotides in human cell lines","volume":"25","author":"Michel","year":"2021","journal-title":"Mol Diagn Ther"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib277","doi-asserted-by":"crossref","first-page":"302","DOI":"10.1089\/nat.2013.0436","article-title":"Hepatotoxic potential of therapeutic oligonucleotides can be predicted from their sequence and modification pattern","volume":"23","author":"Hagedorn","year":"2013","journal-title":"Nucleic Acid Ther"},{"issue":"8","key":"10.1016\/j.pharmr.2025.100093_bib278","doi-asserted-by":"crossref","first-page":"4882","DOI":"10.1093\/nar\/gku142","article-title":"Sequence motifs associated with hepatotoxicity of locked nucleic acid--modified antisense oligonucleotides","volume":"42","author":"Burdick","year":"2014","journal-title":"Nucleic Acids Res"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib279","doi-asserted-by":"crossref","first-page":"2647","DOI":"10.3390\/pharmaceutics14122647","article-title":"Chemistry of therapeutic oligonucleotides that drives interactions with biomolecules","volume":"14","author":"Terada","year":"2022","journal-title":"Pharmaceutics"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib280","doi-asserted-by":"crossref","first-page":"7972","DOI":"10.1038\/s41467-023-43714-0","article-title":"Dynamic and static control of the off-target interactions of antisense oligonucleotides using toehold chemistry","volume":"14","author":"Terada","year":"2023","journal-title":"Nat Commun"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib281","doi-asserted-by":"crossref","first-page":"151","DOI":"10.1089\/nat.2021.0071","article-title":"Acute neurotoxicity of antisense oligonucleotides after intracerebroventricular injection into mouse brain can be predicted from sequence features","volume":"32","author":"Hagedorn","year":"2022","journal-title":"Nucleic Acid Ther"},{"issue":"7","key":"10.1016\/j.pharmr.2025.100093_bib282","doi-asserted-by":"crossref","first-page":"1808","DOI":"10.3390\/pharmaceutics15071808","article-title":"Ensemble-learning and feature selection techniques for enhanced antisense oligonucleotide efficacy prediction in exon skipping","volume":"15","author":"Zhu","year":"2023","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.pharmr.2025.100093_bib283","doi-asserted-by":"crossref","DOI":"10.3389\/fnins.2021.685645","article-title":"Central nervous system-endogenous TLR7 and TLR9 induce different immune responses and effects on experimental autoimmune encephalomyelitis","volume":"15","author":"Dieu","year":"2021","journal-title":"Front Neurosci"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib284","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1089\/nat.2022.0067","article-title":"Systematic analysis of chemical modifications of phosphorothioate antisense oligonucleotides that modulate their innate immune response","volume":"33","author":"Pollak","year":"2023","journal-title":"Nucleic Acid Ther"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib285","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1089\/nat.2022.0061","article-title":"Considerations in the preclinical assessment of the safety of antisense oligonucleotides","volume":"33","author":"Goyenvalle","year":"2023","journal-title":"Nucleic Acid Ther"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib286","doi-asserted-by":"crossref","first-page":"396","DOI":"10.1016\/j.tibtech.2022.12.022","article-title":"The genome editing revolution","volume":"41","author":"van der Oost","year":"2023","journal-title":"Trends Biotechnol"},{"key":"10.1016\/j.pharmr.2025.100093_bib287","doi-asserted-by":"crossref","DOI":"10.3389\/fnins.2021.632522","article-title":"In vivo genome editing therapeutic approaches for neurological disorders: where are we in the translational pipeline?","volume":"15","author":"Lubroth","year":"2021","journal-title":"Front Neurosci"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib288","doi-asserted-by":"crossref","first-page":"442","DOI":"10.1016\/j.cell.2013.06.044","article-title":"CRISPR-mediated modular RNA-guided regulation of transcription in eukaryotes","volume":"154","author":"Gilbert","year":"2013","journal-title":"Cell"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib289","doi-asserted-by":"crossref","first-page":"1097","DOI":"10.1038\/nbt.2756","article-title":"Epigenome editing","volume":"31","author":"Voigt","year":"2013","journal-title":"Nat Biotechnol"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib290","doi-asserted-by":"crossref","first-page":"510","DOI":"10.1038\/nbt.3199","article-title":"Epigenome editing by a CRISPR-Cas9-based acetyltransferase activates genes from promoters and enhancers","volume":"33","author":"Hilton","year":"2015","journal-title":"Nat Biotechnol"},{"issue":"7603","key":"10.1016\/j.pharmr.2025.100093_bib291","doi-asserted-by":"crossref","first-page":"420","DOI":"10.1038\/nature17946","article-title":"Programmable editing of a target base in genomic DNA without double-stranded DNA cleavage","volume":"533","author":"Komor","year":"2016","journal-title":"Nature"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib292","doi-asserted-by":"crossref","first-page":"500","DOI":"10.1038\/s41587-022-01527-4","article-title":"Drag-and-drop genome insertion of large sequences without double-strand DNA cleavage using CRISPR-directed integrases","volume":"41","author":"Yarnall","year":"2023","journal-title":"Nat Biotechnol"},{"issue":"18","key":"10.1016\/j.pharmr.2025.100093_bib293","doi-asserted-by":"crossref","first-page":"3983","DOI":"10.1016\/j.cell.2023.07.039","article-title":"Phage-assisted evolution and protein engineering yield compact, efficient prime editors","volume":"186","author":"Doman","year":"2023","journal-title":"Cell"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib294","doi-asserted-by":"crossref","first-page":"22","DOI":"10.1038\/s41551-024-01227-1","article-title":"Efficient site-specific integration of large genes in mammalian cells via continuously evolved recombinases and prime editing","volume":"9","author":"Pandey","year":"2025","journal-title":"Nat Biomed Eng"},{"issue":"7785","key":"10.1016\/j.pharmr.2025.100093_bib295","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1038\/s41586-019-1711-4","article-title":"Search-and-replace genome editing without double-strand breaks or donor DNA","volume":"576","author":"Anzalone","year":"2019","journal-title":"Nature"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib296","doi-asserted-by":"crossref","first-page":"253","DOI":"10.1038\/s41587-023-01758-z","article-title":"Efficient prime editing in mouse brain, liver and heart with dual AAVs","volume":"42","author":"\u2018Davis","year":"2024","journal-title":"Nat Biotechnol"},{"issue":"8","key":"10.1016\/j.pharmr.2025.100093_bib297","doi-asserted-by":"crossref","first-page":"1000","DOI":"10.1016\/j.tibtech.2023.03.004","article-title":"Prime editing: advances and therapeutic applications","volume":"41","author":"Zhao","year":"2023","journal-title":"Trends Biotechnol"},{"key":"10.1016\/j.pharmr.2025.100093_bib298","doi-asserted-by":"crossref","first-page":"429","DOI":"10.1016\/j.omtn.2018.03.007","article-title":"CRISPR\/Cas9 mediated disruption of the Swedish APP allele as a therapeutic approach for early-onset Alzheimer's disease","volume":"11","author":"Gy\u00f6rgy","year":"2018","journal-title":"Mol Ther Nucleic Acids"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib299","doi-asserted-by":"crossref","first-page":"378","DOI":"10.1007\/s12035-018-1068-y","article-title":"Targeted gene editing of glia maturation factor in microglia: a novel Alzheimer's disease therapeutic target","volume":"56","author":"Raikwar","year":"2019","journal-title":"Mol Neurobiol"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib300","doi-asserted-by":"crossref","first-page":"524","DOI":"10.1038\/s41593-019-0352-0","article-title":"In vivo neuronal gene editing via CRISPR-Cas9 amphiphilic nanocomplexes alleviates deficits in mouse models of Alzheimer's disease","volume":"22","author":"Park","year":"2019","journal-title":"Nat Neurosci"},{"issue":"3\u20134","key":"10.1016\/j.pharmr.2025.100093_bib301","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1038\/s41434-019-0116-1","article-title":"The deletion of mutant SOD1 via CRISPR\/Cas9\/sgRNA prolongs survival in an amyotrophic lateral sclerosis mouse model","volume":"27","author":"Duan","year":"2020","journal-title":"Gene Ther"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib302","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.aar3952","article-title":"In vivo genome editing improves motor function and extends survival in a mouse model of ALS","volume":"3","author":"Gaj","year":"2017","journal-title":"Sci Adv"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib303","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1007\/s00401-020-02154-6","article-title":"CRISPR deletion of the C9ORF72 promoter in ALS\/FTD patient motor neurons abolishes production of dipeptide repeat proteins and rescues neurodegeneration","volume":"140","author":"Krishnan","year":"2020","journal-title":"Acta Neuropathol"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib304","doi-asserted-by":"crossref","first-page":"6286","DOI":"10.1038\/s41467-022-33332-7","article-title":"CRISPR\/Cas9-mediated excision of ALS\/FTD-causing hexanucleotide repeat expansion in C9ORF72 rescues major disease mechanisms in vivo and in vitro","volume":"13","author":"Meijboom","year":"2022","journal-title":"Nat Commun"},{"issue":"7","key":"10.1016\/j.pharmr.2025.100093_bib305","doi-asserted-by":"crossref","first-page":"2719","DOI":"10.1172\/JCI92087","article-title":"CRISPR\/Cas9-mediated gene editing ameliorates neurotoxicity in mouse model of Huntington's disease","volume":"127","author":"Yang","year":"2017","journal-title":"J Clin Invest"},{"issue":"20","key":"10.1016\/j.pharmr.2025.100093_bib306","first-page":"4566","article-title":"Permanent inactivation of Huntington's disease mutation by personalized allele-specific CRISPR\/Cas9","volume":"25","author":"Shin","year":"2016","journal-title":"Hum Mol Genet"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib307","doi-asserted-by":"crossref","first-page":"12","DOI":"10.1016\/j.ymthe.2016.11.010","article-title":"CRISPR\/Cas9 editing of the mutant huntingtin allele in vitro and in vivo","volume":"25","author":"Monteys","year":"2017","journal-title":"Mol Ther"},{"key":"10.1016\/j.pharmr.2025.100093_bib308","doi-asserted-by":"crossref","first-page":"829","DOI":"10.1016\/j.omtn.2019.07.009","article-title":"CRISPR-Cas9-Mediated genome editing increases lifespan and improves motor deficits in a Huntington's disease mouse model","volume":"17","author":"Ekman","year":"2019","journal-title":"Mol Ther Nucleic Acids"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib309","doi-asserted-by":"crossref","first-page":"1486","DOI":"10.1007\/s12035-022-03150-5","article-title":"CRISPR\/Cas9 mediated therapeutic approach in Huntington's disease","volume":"60","author":"Alkanli","year":"2023","journal-title":"Mol Neurobiol"},{"issue":"7833","key":"10.1016\/j.pharmr.2025.100093_bib310","doi-asserted-by":"crossref","first-page":"281","DOI":"10.1038\/s41586-020-2835-2","article-title":"Cas9 gene therapy for Angelman syndrome traps Ube3a-ATS long non-coding RNA","volume":"587","author":"Wolter","year":"2020","journal-title":"Nature"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib311","doi-asserted-by":"crossref","DOI":"10.1126\/sciadv.abe1611","article-title":"Persistent repression of tau in the brain using engineered zinc finger protein transcription factors","volume":"7","author":"Wegmann","year":"2021","journal-title":"Sci Adv"},{"issue":"45","key":"10.1016\/j.pharmr.2025.100093_bib312","doi-asserted-by":"crossref","first-page":"E3136","DOI":"10.1073\/pnas.1206506109","article-title":"Synthetic zinc finger repressors reduce mutant huntingtin expression in the brain of R6\/2 mice","volume":"109","author":"Garriga-Canut","year":"2012","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib313","doi-asserted-by":"crossref","first-page":"1515","DOI":"10.1007\/s13311-021-01081-y","article-title":"Gene editing and modulation: the Holy Grail for the genetic epilepsies?","volume":"18","author":"Carpenter","year":"2021","journal-title":"Neurotherapeutics"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib314","doi-asserted-by":"crossref","first-page":"235","DOI":"10.1016\/j.ymthe.2019.08.018","article-title":"dCas9-based Scn1a gene activation restores inhibitory interneuron excitability and attenuates seizures in Dravet syndrome mice","volume":"28","author":"Colasante","year":"2020","journal-title":"Mol Ther"},{"key":"10.1016\/j.pharmr.2025.100093_bib315","doi-asserted-by":"crossref","DOI":"10.1016\/j.nbd.2020.104954","article-title":"CRISPR\/dCas9-based Scn1a gene activation in inhibitory neurons ameliorates epileptic and behavioral phenotypes of Dravet syndrome model mice","volume":"141","author":"Yamagata","year":"2020","journal-title":"Neurobiol Dis"},{"key":"10.1016\/j.pharmr.2025.100093_bib316","doi-asserted-by":"crossref","unstructured":"Tamura S, Nelson AD, Spratt PWE, et al. CRISPR activation rescues abnormalities in SCN2A haploinsufficiency-associated autism spectrum disorder. Preprint. Posted online April 01, 2022. bioRxiv. 2022.03.30.486483. https:\/\/doi.org\/10.1101\/2022.03.30.486483.","DOI":"10.1101\/2022.03.30.486483"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib317","doi-asserted-by":"crossref","first-page":"891","DOI":"10.1093\/brain\/awaa045","article-title":"In vivo CRISPRa decreases seizures and rescues cognitive deficits in a rodent model of epilepsy","volume":"143","author":"Colasante","year":"2020","journal-title":"Brain"},{"issue":"6424","key":"10.1016\/j.pharmr.2025.100093_bib318","doi-asserted-by":"crossref","DOI":"10.1126\/science.aau0629","article-title":"CRISPR-mediated activation of a promoter or enhancer rescues obesity caused by haploinsufficiency","volume":"363","author":"Matharu","year":"2019","journal-title":"Science"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib319","doi-asserted-by":"crossref","DOI":"10.15252\/emmm.202114797","article-title":"Parkinson's disease motor symptoms rescue by CRISPRa-reprogramming astrocytes into GABAergic neurons","volume":"14","author":"Giehrl-Schwab","year":"2022","journal-title":"EMBO Mol Med"},{"issue":"11\u201312","key":"10.1016\/j.pharmr.2025.100093_bib320","doi-asserted-by":"crossref","first-page":"579","DOI":"10.1089\/hum.2022.037","article-title":"Cell-selective adeno-associated virus-mediated SCN1A gene regulation therapy rescues mortality and seizure phenotypes in a Dravet syndrome mouse model and is well tolerated in nonhuman primates","volume":"33","author":"Tanenhaus","year":"2022","journal-title":"Hum Gene Ther"},{"issue":"11","key":"10.1016\/j.pharmr.2025.100093_bib321","doi-asserted-by":"crossref","first-page":"2638","DOI":"10.1016\/j.ymthe.2018.08.019","article-title":"Downregulation of SNCA expression by targeted editing of DNA methylation: a potential strategy for precision therapy in PD","volume":"26","author":"Kantor","year":"2018","journal-title":"Mol Ther"},{"issue":"679","key":"10.1016\/j.pharmr.2025.100093_bib322","doi-asserted-by":"crossref","DOI":"10.1126\/scitranslmed.add4666","article-title":"Multiplex epigenome editing of MECP2 to rescue Rett syndrome neurons","volume":"15","author":"Qian","year":"2023","journal-title":"Sci Transl Med"},{"issue":"6642","key":"10.1016\/j.pharmr.2025.100093_bib323","doi-asserted-by":"crossref","DOI":"10.1126\/science.adg6518","article-title":"Base editing rescue of spinal muscular atrophy in cells and in mice","volume":"380","author":"Arbab","year":"2023","journal-title":"Science"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib324","doi-asserted-by":"crossref","first-page":"1177","DOI":"10.1016\/j.ymthe.2020.01.005","article-title":"Treatment of a mouse model of ALS by in vivo base editing","volume":"28","author":"Lim","year":"2020","journal-title":"Mol Ther"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib325","doi-asserted-by":"crossref","first-page":"36","DOI":"10.1038\/s41392-023-01309-7","article-title":"CRISPR\/Cas9 therapeutics: progress and prospects","volume":"8","author":"Li","year":"2023","journal-title":"Signal Transduct Target Ther"},{"issue":"7582","key":"10.1016\/j.pharmr.2025.100093_bib326","doi-asserted-by":"crossref","first-page":"422","DOI":"10.1038\/nature16142","article-title":"A mechanism for the suppression of homologous recombination in G1 cells","volume":"528","author":"Orthwein","year":"2015","journal-title":"Nature"},{"issue":"7631","key":"10.1016\/j.pharmr.2025.100093_bib327","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1038\/nature20565","article-title":"In vivo genome editing via CRISPR\/Cas9 mediated homology-independent targeted integration","volume":"540","author":"Suzuki","year":"2016","journal-title":"Nature"},{"key":"10.1016\/j.pharmr.2025.100093_bib328","doi-asserted-by":"crossref","DOI":"10.7554\/eLife.65202","article-title":"An optimized CRISPR\/Cas9 approach for precise genome editing in neurons","volume":"10","author":"Fang","year":"2021","journal-title":"eLife"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib329","doi-asserted-by":"crossref","first-page":"4439","DOI":"10.1038\/s41467-019-12449-2","article-title":"High levels of AAV vector integration into CRISPR-induced DNA breaks","volume":"10","author":"Hanlon","year":"2019","journal-title":"Nat Commun"},{"key":"10.1016\/j.pharmr.2025.100093_bib330","doi-asserted-by":"crossref","DOI":"10.3389\/fbioe.2022.895713","article-title":"Viral vectors for the in vivo delivery of CRISPR components: advances and challenges","volume":"10","author":"Asmamaw Mengstie","year":"2022","journal-title":"Front Bioeng Biotechnol"},{"key":"10.1016\/j.pharmr.2025.100093_bib331","doi-asserted-by":"crossref","DOI":"10.3389\/fbioe.2023.1138596","article-title":"Immunogenicity of CRISPR therapeutics-Critical considerations for clinical translation","volume":"11","author":"Ewaisha","year":"2023","journal-title":"Front Bioeng Biotechnol"},{"key":"10.1016\/j.pharmr.2025.100093_bib332","doi-asserted-by":"crossref","DOI":"10.3389\/fbioe.2023.1143157","article-title":"Off-target effects in CRISPR\/Cas9 gene editing","volume":"11","author":"Guo","year":"2023","journal-title":"Front Bioeng Biotechnol"},{"key":"10.1016\/j.pharmr.2025.100093_bib333","first-page":"21","article-title":"Recent advancements in reducing the off-target effect of CRISPR-Cas9 genome editing","volume":"18","author":"Asmamaw Mengstie","year":"2024","journal-title":"Biologics"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib334","doi-asserted-by":"crossref","first-page":"97","DOI":"10.1038\/s41551-019-0501-5","article-title":"Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses","volume":"4","author":"Levy","year":"2020","journal-title":"Nat Biomed Eng"},{"issue":"15","key":"10.1016\/j.pharmr.2025.100093_bib335","doi-asserted-by":"crossref","first-page":"2806","DOI":"10.1016\/j.cell.2022.03.045","article-title":"Therapeutic in vivo delivery of gene editing agents","volume":"185","author":"Raguram","year":"2022","journal-title":"Cell"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib336","doi-asserted-by":"crossref","first-page":"250","DOI":"10.1016\/j.cell.2021.12.021","article-title":"Engineered virus-like particles for efficient in vivo delivery of therapeutic proteins","volume":"185","author":"Banskota","year":"2022","journal-title":"Cell"},{"key":"10.1016\/j.pharmr.2025.100093_bib337","doi-asserted-by":"crossref","DOI":"10.3389\/fgene.2021.673286","article-title":"Nanoparticle delivery of CRISPR\/Cas9 for genome editing","volume":"12","author":"Duan","year":"2021","journal-title":"Front Genet"},{"issue":"8","key":"10.1016\/j.pharmr.2025.100093_bib338","doi-asserted-by":"crossref","first-page":"2422","DOI":"10.1016\/j.ymthe.2023.06.019","article-title":"Genome editing in the mouse brain with minimally immunogenic Cas9 RNPs","volume":"31","author":"Stahl","year":"2023","journal-title":"Mol Ther"},{"key":"10.1016\/j.pharmr.2025.100093_bib339","doi-asserted-by":"crossref","first-page":"7","DOI":"10.3389\/fneng.2013.00007","article-title":"The blood-brain barrier: an engineering perspective","volume":"6","author":"Wong","year":"2013","journal-title":"Front Neuroeng"},{"issue":"6","key":"10.1016\/j.pharmr.2025.100093_bib340","doi-asserted-by":"crossref","first-page":"1058","DOI":"10.1038\/mt.2011.72","article-title":"Preclinical differences of intravascular AAV9 delivery to neurons and glia: a comparative study of adult mice and nonhuman primates","volume":"19","author":"Gray","year":"2011","journal-title":"Mol Ther"},{"issue":"9","key":"10.1016\/j.pharmr.2025.100093_bib341","doi-asserted-by":"crossref","first-page":"1574","DOI":"10.1038\/sj.gt.3300994","article-title":"Immune responses to adenovirus and adeno-associated virus in humans","volume":"6","author":"Chirmule","year":"1999","journal-title":"Gene Ther"},{"issue":"10","key":"10.1016\/j.pharmr.2025.100093_bib342","doi-asserted-by":"crossref","first-page":"1701","DOI":"10.1038\/s41591-021-01483-7","article-title":"Biodistribution of onasemnogene abeparvovec DNA, mRNA and SMN protein in human tissue","volume":"27","author":"Thomsen","year":"2021","journal-title":"Nat Med"},{"key":"10.1016\/j.pharmr.2025.100093_bib343","doi-asserted-by":"crossref","first-page":"20","DOI":"10.1016\/j.addr.2021.03.002","article-title":"Harnessing cerebrospinal fluid circulation for drug delivery to brain tissues","volume":"173","author":"Naseri Kouzehgarani","year":"2021","journal-title":"Adv Drug Deliv Rev"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib344","doi-asserted-by":"crossref","first-page":"1178","DOI":"10.1038\/gt.2013.48","article-title":"Adeno-associated virus type 6 is retrogradely transported in the non-human primate brain","volume":"20","author":"San Sebastian","year":"2013","journal-title":"Gene Ther"},{"issue":"7","key":"10.1016\/j.pharmr.2025.100093_bib345","doi-asserted-by":"crossref","first-page":"963","DOI":"10.1517\/17425247.2016.1171315","article-title":"CSF, blood-brain barrier, and brain drug delivery","volume":"13","author":"Pardridge","year":"2016","journal-title":"Expert Opin Drug Deliv"},{"issue":"10","key":"10.1016\/j.pharmr.2025.100093_bib346","doi-asserted-by":"crossref","first-page":"1763","DOI":"10.1212\/WNL.57.10.1763","article-title":"The cerebrospinal fluid production rate is reduced in dementia of the Alzheimer's type","volume":"57","author":"Silverberg","year":"2001","journal-title":"Neurology"},{"key":"10.1016\/j.pharmr.2025.100093_bib347","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1016\/j.omtm.2018.12.001","article-title":"Kinetics and MR-based monitoring of AAV9 vector delivery into cerebrospinal fluid of nonhuman primates","volume":"13","author":"Ohno","year":"2019","journal-title":"Mol Ther Methods Clin Dev"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib348","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1089\/hgtb.2015.129","article-title":"Cerebellomedullary cistern delivery for AAV-based gene therapy: a technical note for nonhuman primates","volume":"27","author":"Samaranch","year":"2016","journal-title":"Hum Gene Ther Methods"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib349","doi-asserted-by":"crossref","first-page":"411","DOI":"10.1016\/j.ymthe.2019.11.012","article-title":"A safe and reliable technique for CNS delivery of AAV vectors in the cisterna magna","volume":"28","author":"Taghian","year":"2020","journal-title":"Mol Ther"},{"key":"10.1016\/j.pharmr.2025.100093_bib350","doi-asserted-by":"crossref","DOI":"10.1038\/mtm.2014.51","article-title":"Widespread gene transfer in the central nervous system of cynomolgus macaques following delivery of AAV9 into the cisterna magna","volume":"1","author":"Hinderer","year":"2014","journal-title":"Mol Ther Methods Clin Dev"},{"issue":"6","key":"10.1016\/j.pharmr.2025.100093_bib351","doi-asserted-by":"crossref","first-page":"1751","DOI":"10.3171\/2020.4.JNS20701","article-title":"Direct convective delivery of adeno-associated virus gene therapy for treatment of neurological disorders","volume":"134","author":"Lonser","year":"2021","journal-title":"J Neurosurg"},{"issue":"7","key":"10.1016\/j.pharmr.2025.100093_bib352","doi-asserted-by":"crossref","first-page":"2407","DOI":"10.1073\/pnas.0810682106","article-title":"Efficient gene therapy-based method for the delivery of therapeutics to primate cortex","volume":"106","author":"Kells","year":"2009","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib353","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.jneumeth.2013.08.011","article-title":"Convection-enhanced delivery of AAV2 in white matter--a novel method for gene delivery to cerebral cortex","volume":"220","author":"Barua","year":"2013","journal-title":"J Neurosci Methods"},{"issue":"6","key":"10.1016\/j.pharmr.2025.100093_bib354","doi-asserted-by":"crossref","first-page":"2076","DOI":"10.1073\/pnas.91.6.2076","article-title":"Convection-enhanced delivery of macromolecules in the brain","volume":"91","author":"Bobo","year":"1994","journal-title":"Proc Natl Acad Sci U S A"},{"issue":"12","key":"10.1016\/j.pharmr.2025.100093_bib355","doi-asserted-by":"crossref","first-page":"1362","DOI":"10.1038\/nm1297-1362","article-title":"Tumor regression with regional distribution of the targeted toxin TF-CRM107 in patients with malignant brain tumors","volume":"3","author":"Laske","year":"1997","journal-title":"Nat Med"},{"key":"10.1016\/j.pharmr.2025.100093_bib356","doi-asserted-by":"crossref","first-page":"966","DOI":"10.3389\/fonc.2019.00966","article-title":"Convection-enhanced delivery: connection to and impact of interstitial fluid flow","volume":"9","author":"Stine","year":"2019","journal-title":"Front Oncol"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib357","doi-asserted-by":"crossref","first-page":"311","DOI":"10.3171\/jns.2005.103.2.0311","article-title":"Surface properties, more than size, limiting convective distribution of virus-sized particles and viruses in the central nervous system","volume":"103","author":"Chen","year":"2005","journal-title":"J Neurosurg"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib358","doi-asserted-by":"crossref","first-page":"530","DOI":"10.3171\/2019.4.JNS19826","article-title":"Infuse-as-you-go convective delivery to enhance coverage of elongated brain targets: technical note","volume":"133","author":"Sudhakar","year":"2020","journal-title":"J Neurosurg"},{"key":"10.1016\/j.pharmr.2025.100093_bib359","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1016\/j.jneumeth.2018.08.029","article-title":"Maximising coverage of brain structures using controlled reflux, convection-enhanced delivery and the recessed step catheter","volume":"308","author":"Lewis","year":"2018","journal-title":"J Neurosci Methods"},{"issue":"1","key":"10.1016\/j.pharmr.2025.100093_bib360","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1016\/j.jneumeth.2011.04.027","article-title":"An evaluation of the relationships between catheter design and tissue mechanics in achieving high-flow convection-enhanced delivery","volume":"199","author":"White","year":"2011","journal-title":"J Neurosci Methods"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib361","doi-asserted-by":"crossref","first-page":"315","DOI":"10.3171\/jns.1999.90.2.0315","article-title":"Variables affecting convection-enhanced delivery to the striatum: a systematic examination of rate of infusion, cannula size, infusate concentration, and tissue-cannula sealing time","volume":"90","author":"Chen","year":"1999","journal-title":"J Neurosurg"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib362","first-page":"ONS89","article-title":"Intracerebral infusate distribution by convection-enhanced delivery in humans with malignant gliomas: descriptive effects of target anatomy and catheter positioning","volume":"60","author":"Sampson","year":"2007","journal-title":"Neurosurgery"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib363","doi-asserted-by":"crossref","first-page":"358","DOI":"10.1007\/s13311-017-0520-4","article-title":"Convection-enhanced delivery","volume":"14","author":"Mehta","year":"2017","journal-title":"Neurotherapeutics"},{"issue":"suppl 2","key":"10.1016\/j.pharmr.2025.100093_bib364","doi-asserted-by":"crossref","first-page":"T27","DOI":"10.1016\/j.neuroimage.2008.11.012","article-title":"Real-time MR imaging of adeno-associated viral vector delivery to the primate brain","volume":"47","author":"Fiandaca","year":"2009","journal-title":"Neuroimage"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib365","doi-asserted-by":"crossref","first-page":"314","DOI":"10.3171\/jns.2004.101.2.0314","article-title":"A realistic brain tissue phantom for intraparenchymal infusion studies","volume":"101","author":"Chen","year":"2004","journal-title":"J Neurosurg"},{"key":"10.1016\/j.pharmr.2025.100093_bib366","doi-asserted-by":"crossref","first-page":"47","DOI":"10.1016\/j.jneumeth.2015.11.008","article-title":"Chronic, intermittent convection-enhanced delivery devices","volume":"259","author":"Lewis","year":"2016","journal-title":"J Neurosci Methods"},{"issue":"4","key":"10.1016\/j.pharmr.2025.100093_bib367","doi-asserted-by":"crossref","first-page":"575","DOI":"10.1007\/s11701-018-0899-x","article-title":"Robot-assisted stereotactic brainstem biopsy in children: prospective cohort study","volume":"13","author":"Dawes","year":"2019","journal-title":"J Robot Surg"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib368","doi-asserted-by":"crossref","first-page":"297","DOI":"10.3171\/2018.10.PEDS18227","article-title":"Accuracy of robot-assisted versus optical frameless navigated stereoelectroencephalography electrode placement in children","volume":"23","author":"Sharma","year":"2019","journal-title":"J Neurosurg Pediatr"},{"issue":"6","key":"10.1016\/j.pharmr.2025.100093_bib369","doi-asserted-by":"crossref","first-page":"635","DOI":"10.1080\/14737175.2016.1175939","article-title":"Interventional MRI-guided catheter placement and real time drug delivery to the central nervous system","volume":"16","author":"Han","year":"2016","journal-title":"Expert Rev Neurother"},{"issue":"5","key":"10.1016\/j.pharmr.2025.100093_bib370","doi-asserted-by":"crossref","first-page":"1173","DOI":"10.3171\/2014.11.JNS131888","article-title":"Accuracy of direct magnetic resonance imaging-guided placement of drug infusion cannulae","volume":"122","author":"Chittiboina","year":"2015","journal-title":"J Neurosurg"},{"issue":"3","key":"10.1016\/j.pharmr.2025.100093_bib371","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1159\/000323544","article-title":"Novel platform for MRI-guided convection-enhanced delivery of therapeutics: preclinical validation in nonhuman primate brain","volume":"89","author":"Richardson","year":"2011","journal-title":"Stereotact Funct Neurosurg"},{"issue":"2","key":"10.1016\/j.pharmr.2025.100093_bib372","doi-asserted-by":"crossref","first-page":"595","DOI":"10.3171\/2018.10.JNS182169","article-title":"Development of a novel frameless skull-mounted ball-joint guide array for use in image-guided neurosurgery","volume":"132","author":"Sudhakar","year":"2019","journal-title":"J Neurosurg"},{"key":"10.1016\/j.pharmr.2025.100093_bib373","doi-asserted-by":"crossref","DOI":"10.3389\/fnins.2020.580179","article-title":"Gene therapy for neurodegenerative diseases: slowing down the ticking clock","volume":"14","author":"Martier","year":"2020","journal-title":"Front Neurosci"}],"container-title":["Pharmacological Reviews"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0031699725075015?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S0031699725075015?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,3,19]],"date-time":"2026-03-19T11:12:22Z","timestamp":1773918742000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S0031699725075015"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,1]]},"references-count":375,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,1]]}},"alternative-id":["S0031699725075015"],"URL":"https:\/\/doi.org\/10.1016\/j.pharmr.2025.100093","relation":{},"ISSN":["0031-6997"],"issn-type":[{"value":"0031-6997","type":"print"}],"subject":[],"published":{"date-parts":[[2026,1]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Genetic therapies for neurological diseases","name":"articletitle","label":"Article Title"},{"value":"Pharmacological Reviews","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.pharmr.2025.100093","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Authors. Published by Elsevier Inc. on behalf of American Society for Pharmacology and Experimental Therapeutics.","name":"copyright","label":"Copyright"}],"article-number":"100093"}}