{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,5]],"date-time":"2026-11-05T18:50:44Z","timestamp":1793904644133,"version":"4.8.0"},"reference-count":77,"publisher":"Elsevier BV","issue":"8","license":[{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2025,9,1]],"date-time":"2025-09-01T00:00:00Z","timestamp":1756684800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2025,7,2]],"date-time":"2025-07-02T00:00:00Z","timestamp":1751414400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["The Journal of Prevention of Alzheimer's Disease"],"published-print":{"date-parts":[[2025,9]]},"DOI":"10.1016\/j.tjpad.2025.100269","type":"journal-article","created":{"date-parts":[[2025,7,15]],"date-time":"2025-07-15T21:04:00Z","timestamp":1752613440000},"page":"100269","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":7,"title":["CTAD taskforce: genetic therapies in Alzheimer\u2019s disease"],"prefix":"10.1016","volume":"12","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-7670-0175","authenticated-orcid":false,"given":"D.","family":"Jakabek","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"A.M.","family":"Isaacs","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"B.","family":"De Strooper","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M.","family":"Tuszynski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R.","family":"Lane","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"O.","family":"Uspenskaya","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"E.","family":"McDade","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"M.S.","family":"Rafii","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"C.J.","family":"Mummery","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"issue":"6","key":"10.1016\/j.tjpad.2025.100269_bib0001","doi-asserted-by":"crossref","first-page":"512","DOI":"10.1001\/jama.2023.13239","article-title":"Donanemab in early symptomatic Alzheimer disease: the TRAILBLAZER-ALZ 2 randomized clinical trial","volume":"330","author":"Sims","year":"2023","journal-title":"JAMA"},{"issue":"6","key":"10.1016\/j.tjpad.2025.100269_bib0002","doi-asserted-by":"crossref","first-page":"1437","DOI":"10.1038\/s41591-023-02326-3","article-title":"Tau-targeting antisense oligonucleotide MAPT(Rx) in mild Alzheimer's disease: a phase 1b, randomized, placebo-controlled trial","volume":"29","author":"Mummery","year":"2023","journal-title":"Nat Med"},{"issue":"S24","key":"10.1016\/j.tjpad.2025.100269_bib0003","doi-asserted-by":"crossref","DOI":"10.1002\/alz.082650","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":"Alzheimer Dement"},{"issue":"8","key":"10.1016\/j.tjpad.2025.100269_bib0004","doi-asserted-by":"crossref","first-page":"553","DOI":"10.1038\/s41583-024-00829-7","article-title":"Gene therapy for CNS disorders: modalities, delivery and translational challenges","volume":"25","author":"Gao","year":"2024","journal-title":"Nat Rev Neurosci"},{"key":"10.1016\/j.tjpad.2025.100269_bib0005","unstructured":"Qalsody Prescribing Information. Food and Drug Administration; 2023."},{"issue":"7","key":"10.1016\/j.tjpad.2025.100269_bib0006","doi-asserted-by":"crossref","first-page":"834","DOI":"10.1001\/jamaneurol.2018.0233","article-title":"Adeno-associated viral vector (Serotype 2)-nerve growth factor for patients with Alzheimer disease: a randomized clinical trial","volume":"75","author":"Rafii","year":"2018","journal-title":"JAMA Neurol"},{"issue":"5","key":"10.1016\/j.tjpad.2025.100269_bib0007","doi-asserted-by":"crossref","first-page":"551","DOI":"10.1038\/nm1239","article-title":"A phase 1 clinical trial of nerve growth factor gene therapy for Alzheimer disease","volume":"11","author":"Tuszynski","year":"2005","journal-title":"Nat Med"},{"issue":"1","key":"10.1016\/j.tjpad.2025.100269_bib0008","doi-asserted-by":"crossref","first-page":"1112","DOI":"10.1007\/s12035-024-04285-3","article-title":"From lab bench to hope: emerging gene therapies in clinical trials for Alzheimer's Disease","volume":"62","author":"Ortega","year":"2025","journal-title":"Mol Neurobiol"},{"issue":"5","key":"10.1016\/j.tjpad.2025.100269_bib0009","doi-asserted-by":"crossref","first-page":"1406","DOI":"10.1038\/s41591-024-02973-0","article-title":"Progranulin AAV gene therapy for frontotemporal dementia: translational studies and phase 1\/2 trial interim results","volume":"30","author":"Sevigny","year":"2024","journal-title":"Nat Med"},{"key":"10.1016\/j.tjpad.2025.100269_bib0010","unstructured":"Spinraza Prescribing Information. Food and Drug Administration; 2016."},{"issue":"1","key":"10.1016\/j.tjpad.2025.100269_bib0011","doi-asserted-by":"crossref","first-page":"64","DOI":"10.1186\/s12943-021-01487-4","article-title":"Strategies to overcome the main challenges of the use of CRISPR\/Cas9 as a replacement for cancer therapy","volume":"21","author":"Rasul","year":"2022","journal-title":"Mol Cancer"},{"issue":"6","key":"10.1016\/j.tjpad.2025.100269_bib0012","doi-asserted-by":"crossref","first-page":"887","DOI":"10.1038\/s44318-024-00057-w","article-title":"New precision medicine avenues to the prevention of Alzheimer's disease from insights into the structure and function of gamma-secretases","volume":"43","author":"De Strooper","year":"2024","journal-title":"EMBO J"},{"issue":"6","key":"10.1016\/j.tjpad.2025.100269_bib0013","doi-asserted-by":"crossref","first-page":"2821","DOI":"10.1038\/s41380-022-01518-6","article-title":"Abeta profiles generated by Alzheimer's disease causing PSEN1 variants determine the pathogenicity of the mutation and predict age at disease onset","volume":"27","author":"Petit","year":"2022","journal-title":"Mol Psychiatry"},{"issue":"3","key":"10.1016\/j.tjpad.2025.100269_bib0014","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1016\/j.cell.2017.07.004","article-title":"Alzheimer's-causing mutations shift abeta length by destabilizing gamma-secretase-abetan interactions","volume":"170","author":"Szaruga","year":"2017","journal-title":"Cell"},{"issue":"42","key":"10.1016\/j.tjpad.2025.100269_bib0015","doi-asserted-by":"crossref","DOI":"10.1073\/pnas.2306714120","article-title":"Human Presenilin-1 delivered by AAV9 rescues impaired gamma-secretase activity, memory deficits, and neurodegeneration in Psen mutant mice","volume":"120","author":"Montenegro","year":"2023","journal-title":"Proc Natl Acad Sci U S A."},{"issue":"4","key":"10.1016\/j.tjpad.2025.100269_bib0016","doi-asserted-by":"crossref","DOI":"10.1016\/j.dhjo.2020.100947","article-title":"Cause of death in adults with Down syndrome in the United States","volume":"13","author":"Landes","year":"2020","journal-title":"Disabil Health J"},{"issue":"2","key":"10.1016\/j.tjpad.2025.100269_bib0017","doi-asserted-by":"crossref","first-page":"152","DOI":"10.1001\/jamaneurol.2018.3616","article-title":"Association of dementia with mortality among adults with Down syndrome older than 35 years","volume":"76","author":"Hithersay","year":"2019","journal-title":"JAMA Neurol"},{"issue":"11","key":"10.1016\/j.tjpad.2025.100269_bib0018","doi-asserted-by":"crossref","first-page":"930","DOI":"10.1016\/S1474-4422(21)00245-3","article-title":"Alzheimer's disease associated with Down syndrome: a genetic form of dementia","volume":"20","author":"Fortea","year":"2021","journal-title":"Lancet Neurol"},{"issue":"7","key":"10.1016\/j.tjpad.2025.100269_bib0019","doi-asserted-by":"crossref","first-page":"716","DOI":"10.1002\/dneu.22709","article-title":"Down syndrome, Alzheimer disease, and cerebral amyloid angiopathy: the complex triangle of brain amyloidosis","volume":"79","author":"Carmona-Iragui","year":"2019","journal-title":"Dev Neurobiol"},{"issue":"1","key":"10.1016\/j.tjpad.2025.100269_bib0020","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1016\/S1474-4422(22)00408-2","article-title":"Comparison of amyloid burden in individuals with Down syndrome versus autosomal dominant Alzheimer's disease: a cross-sectional study","volume":"22","author":"Boerwinkle","year":"2023","journal-title":"Lancet Neurol"},{"issue":"5","key":"10.1016\/j.tjpad.2025.100269_bib0021","doi-asserted-by":"crossref","first-page":"500","DOI":"10.1016\/S1474-4422(24)00084-X","article-title":"Comparison of tau spread in people with Down syndrome versus autosomal-dominant Alzheimer's disease: a cross-sectional study","volume":"23","author":"Wisch","year":"2024","journal-title":"Lancet Neurol"},{"issue":"2","key":"10.1016\/j.tjpad.2025.100269_bib0022","doi-asserted-by":"crossref","first-page":"459","DOI":"10.3233\/JAD-160836","article-title":"Down syndrome, partial trisomy 21, and absence of Alzheimer's disease: the role of APP","volume":"56","author":"Doran","year":"2017","journal-title":"J Alzheimer Dis"},{"issue":"3","key":"10.1016\/j.tjpad.2025.100269_bib0023","doi-asserted-by":"crossref","first-page":"380","DOI":"10.1002\/ana.410430316","article-title":"Molecular mapping of Alzheimer-type dementia in Down's syndrome","volume":"43","author":"Prasher","year":"1998","journal-title":"Ann Neurol"},{"issue":"7","key":"10.1016\/j.tjpad.2025.100269_bib0024","doi-asserted-by":"crossref","first-page":"2093","DOI":"10.1038\/s41591-024-03127-y","article-title":"Publisher correction: APOE4 homozygosity represents a distinct genetic form of Alzheimer's disease","volume":"30","author":"Fortea","year":"2024","journal-title":"Nat Med"},{"issue":"2","key":"10.1016\/j.tjpad.2025.100269_bib0025","doi-asserted-by":"crossref","first-page":"441","DOI":"10.3233\/JAD-2010-1230","article-title":"A physiological role for amyloid-\u03b2 protein: enhancement of learning and memory","volume":"19","author":"Morley","year":"2010","journal-title":"J Alzheimer Dis"},{"issue":"1","key":"10.1016\/j.tjpad.2025.100269_bib0026","doi-asserted-by":"crossref","first-page":"126","DOI":"10.1016\/j.bbr.2007.08.003","article-title":"Amyloid precursor protein knockout mice show age-dependent deficits in passive avoidance learning","volume":"186","author":"Senechal","year":"2008","journal-title":"Behav Brain Res"},{"key":"10.1016\/j.tjpad.2025.100269_bib0027","article-title":"Lessons from tau-deficient mice","volume":"2012","author":"Ke","year":"2012","journal-title":"Int J Alzheimer Dis"},{"key":"10.1016\/j.tjpad.2025.100269_bib0028","unstructured":"Alzforum. Beyond antibodies: from CTAD, new attempts at outflanking Alzheimer\u2019s 2024 [Available from: https:\/\/www.alzforum.org\/news\/conference-coverage\/beyond-antibodies-ctad-new-attempts-outflanking-alzheimers."},{"issue":"7409","key":"10.1016\/j.tjpad.2025.100269_bib0029","doi-asserted-by":"crossref","first-page":"96","DOI":"10.1038\/nature11283","article-title":"A mutation in APP protects against Alzheimer's disease and age-related cognitive decline","volume":"488","author":"Jonsson","year":"2012","journal-title":"Nature"},{"key":"10.1016\/j.tjpad.2025.100269_bib0030","article-title":"Long term rescue of Alzheimer's deficits in vivo by one-time gene-editing of App C-terminus","author":"Aulston","year":"2024","journal-title":"bioRxiv."},{"issue":"11","key":"10.1016\/j.tjpad.2025.100269_bib0031","doi-asserted-by":"crossref","first-page":"1680","DOI":"10.1038\/s41591-019-0611-3","article-title":"Resistance to autosomal dominant Alzheimer's disease in an APOE3 Christchurch homozygote: a case report","volume":"25","author":"Arboleda-Velasquez","year":"2019","journal-title":"Nat Med"},{"issue":"5","key":"10.1016\/j.tjpad.2025.100269_bib0032","first-page":"415","article-title":"Anti-tau antibody semorinemab fails to slow Alzheimer disease","volume":"328","author":"Slomski","year":"2022","journal-title":"JAMA"},{"issue":"12","key":"10.1016\/j.tjpad.2025.100269_bib0033","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":"39","key":"10.1016\/j.tjpad.2025.100269_bib0034","doi-asserted-by":"crossref","first-page":"15596","DOI":"10.1523\/JNEUROSCI.5195-12.2013","article-title":"Early BDNF treatment ameliorates cell loss in the entorhinal cortex of APP transgenic mice","volume":"33","author":"Nagahara","year":"2013","journal-title":"J Neurosci"},{"key":"10.1016\/j.tjpad.2025.100269_bib0035","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1016\/j.conb.2021.10.001","article-title":"New insights and therapeutic opportunities for progranulin-deficient frontotemporal dementia","volume":"72","author":"Amin","year":"2022","journal-title":"Curr Opin Neurobiol"},{"issue":"10 Pt B","key":"10.1016\/j.tjpad.2025.100269_bib0036","doi-asserted-by":"crossref","first-page":"2237","DOI":"10.1016\/j.bbadis.2015.05.011","article-title":"Genetics of the neuronal ceroid lipofuscinoses (Batten disease)","volume":"1852","author":"Mole","year":"2015","journal-title":"Biochim Biophys Acta"},{"issue":"8","key":"10.1016\/j.tjpad.2025.100269_bib0037","doi-asserted-by":"crossref","first-page":"2607","DOI":"10.1093\/brain\/awae168","article-title":"Failure of C9orf72 sense repeat-targeting antisense oligonucleotides: lessons learned and the path forward","volume":"147","author":"Cammack","year":"2024","journal-title":"Brain"},{"issue":"7","key":"10.1016\/j.tjpad.2025.100269_bib0038","doi-asserted-by":"crossref","first-page":"761","DOI":"10.1136\/jnnp-2021-328710","article-title":"Development of a sensitive trial-ready poly(GP) CSF biomarker assay for C9orf72-associated frontotemporal dementia and amyotrophic lateral sclerosis","volume":"93","author":"Wilson","year":"2022","journal-title":"J Neurol Neurosurg Psychiatry"},{"key":"10.1016\/j.tjpad.2025.100269_bib0039","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 Nucl Acid"},{"issue":"9","key":"10.1016\/j.tjpad.2025.100269_bib0040","doi-asserted-by":"crossref","first-page":"901","DOI":"10.1016\/S1474-4422(24)00216-3","article-title":"Safety, tolerability, and pharmacokinetics of antisense oligonucleotide BIIB078 in adults with C9orf72-associated amyotrophic lateral sclerosis: a phase 1, randomised, double blinded, placebo-controlled, multiple ascending dose study","volume":"23","author":"van den Berg","year":"2024","journal-title":"Lancet Neurol"},{"issue":"6","key":"10.1016\/j.tjpad.2025.100269_bib0041","doi-asserted-by":"crossref","first-page":"845","DOI":"10.1007\/s00401-013-1200-z","article-title":"C9orf72 frontotemporal lobar degeneration is characterised by frequent neuronal sense and antisense RNA foci","volume":"126","author":"Mizielinska","year":"2013","journal-title":"Acta Neuropathol"},{"issue":"6201","key":"10.1016\/j.tjpad.2025.100269_bib0042","doi-asserted-by":"crossref","first-page":"1192","DOI":"10.1126\/science.1256800","article-title":"C9orf72 repeat expansions cause neurodegeneration in Drosophila through arginine-rich proteins","volume":"345","author":"Mizielinska","year":"2014","journal-title":"Science"},{"issue":"1","key":"10.1016\/j.tjpad.2025.100269_bib0043","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1007\/s00401-023-02652-3","article-title":"G(2)C(4) targeting antisense oligonucleotides potently mitigate TDP-43 dysfunction in human C9orf72 ALS\/FTD induced pluripotent stem cell derived neurons","volume":"147","author":"Rothstein","year":"2023","journal-title":"Acta Neuropathol"},{"issue":"1","key":"10.1016\/j.tjpad.2025.100269_bib0044","doi-asserted-by":"crossref","first-page":"459","DOI":"10.1038\/s41467-024-55550-x","article-title":"Dual-targeting CRISPR-CasRx reduces C9orf72 ALS\/FTD sense and antisense repeat RNAs in vitro and in vivo","volume":"16","author":"Kempthorne","year":"2025","journal-title":"Nat Commun"},{"key":"10.1016\/j.tjpad.2025.100269_bib0045","doi-asserted-by":"crossref","DOI":"10.1016\/S1474-4422(24)00476-9","article-title":"Crossing the blood-brain barrier: emerging therapeutic strategies for neurological disease","author":"Pedder","year":"2025","journal-title":"Lancet Neurol"},{"issue":"3","key":"10.1016\/j.tjpad.2025.100269_bib0046","doi-asserted-by":"crossref","first-page":"427","DOI":"10.1007\/s11095-022-03185-2","article-title":"Focused ultrasound-mediated drug delivery in humans - a path towards translation in neurodegenerative diseases","volume":"39","author":"Wasielewska","year":"2022","journal-title":"Pharm Res"},{"issue":"1","key":"10.1016\/j.tjpad.2025.100269_bib0047","doi-asserted-by":"crossref","DOI":"10.1080\/19420862.2023.2261509","article-title":"Delivery of the Brainshuttle amyloid-beta antibody fusion trontinemab to non-human primate brain and projected efficacious dose regimens in humans","volume":"15","author":"Grimm","year":"2023","journal-title":"MAbs"},{"issue":"9","key":"10.1016\/j.tjpad.2025.100269_bib0048","doi-asserted-by":"crossref","first-page":"e2","DOI":"10.1136\/jnnp-2022-abn2.97","article-title":"053\u2005Brainshuttle AD: investigating safety, tolerability, and PK\/PD of RG6102 in prodromal\/mild-to-moderate AD","volume":"93","author":"Kulic","year":"2022","journal-title":"J Neurol Neurosurg Psychiatry"},{"key":"10.1016\/j.tjpad.2025.100269_bib0049","unstructured":"BioArctic. BrainTransporter\u2122 dramatically improves antibody delivery to the brain 2024 [Available from: https:\/\/www.bioarctic.com\/en\/braintransporter-dramatically-improves-antibody-delivery-to-the-brain\/."},{"issue":"6701","key":"10.1016\/j.tjpad.2025.100269_bib0050","doi-asserted-by":"crossref","first-page":"1220","DOI":"10.1126\/science.adm8386","article-title":"An AAV capsid reprogrammed to bind human transferrin receptor mediates brain-wide gene delivery","volume":"384","author":"Huang","year":"2024","journal-title":"Science"},{"issue":"760","key":"10.1016\/j.tjpad.2025.100269_bib0051","doi-asserted-by":"crossref","first-page":"eadi2245","DOI":"10.1126\/scitranslmed.adi2245","article-title":"Targeting the transferrin receptor to transport antisense oligonucleotides across the mammalian blood-brain barrier","volume":"16","author":"Barker","year":"2024","journal-title":"Sci Transl Med"},{"issue":"6","key":"10.1016\/j.tjpad.2025.100269_bib0052","doi-asserted-by":"crossref","DOI":"10.3390\/pharmaceutics15061748","article-title":"Novel Human\/non-Human primate cross-reactive anti-transferrin receptor nanobodies for brain delivery of biologics","volume":"15","author":"Rue","year":"2023","journal-title":"Pharmaceutics"},{"key":"10.1016\/j.tjpad.2025.100269_bib0053","article-title":"Immunogenicity and toxicity of AAV gene therapy","volume":"13","year":"2022","journal-title":"Front Immunol"},{"issue":"15\u201316","key":"10.1016\/j.tjpad.2025.100269_bib0054","doi-asserted-by":"crossref","first-page":"808","DOI":"10.1089\/hum.2020.167","article-title":"Adeno-associated virus-induced dorsal root ganglion pathology","volume":"31","author":"Hordeaux","year":"2020","journal-title":"Hum Gene Ther"},{"issue":"3","key":"10.1016\/j.tjpad.2025.100269_bib0055","doi-asserted-by":"crossref","DOI":"10.1016\/j.omtm.2024.101273","article-title":"Global seroprevalence of neutralizing antibodies against adeno-associated virus serotypes used for human gene therapies","volume":"32","author":"Chhabra","year":"2024","journal-title":"Mol Ther Method Clin Dev"},{"issue":"1","key":"10.1016\/j.tjpad.2025.100269_bib0056","doi-asserted-by":"crossref","first-page":"78","DOI":"10.1038\/s41392-024-01780-w","article-title":"Adeno-associated virus as a delivery vector for gene therapy of human diseases","volume":"9","author":"Wang","year":"2024","journal-title":"Signal Transduct Target Ther"},{"issue":"23","key":"10.1016\/j.tjpad.2025.100269_bib0057","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":"24","key":"10.1016\/j.tjpad.2025.100269_bib0058","doi-asserted-by":"crossref","first-page":"2307","DOI":"10.1056\/NEJMoa1900907","article-title":"Targeting huntingtin expression in patients with Huntington's disease","volume":"380","author":"Tabrizi","year":"2019","journal-title":"N Engl J Med"},{"issue":"12","key":"10.1016\/j.tjpad.2025.100269_bib0059","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"},{"key":"10.1016\/j.tjpad.2025.100269_bib0060","doi-asserted-by":"crossref","DOI":"10.1016\/j.eclinm.2024.102495","article-title":"Effects of tofersen treatment in patients with SOD1-ALS in a \"real-world\" setting - a 12-month multicenter cohort study from the German early access program","volume":"69","author":"Wiesenfarth","year":"2024","journal-title":"EClinicalMedicine"},{"key":"10.1016\/j.tjpad.2025.100269_bib0061","doi-asserted-by":"crossref","first-page":"1179","DOI":"10.3389\/fneur.2019.01179","article-title":"Routine cerebrospinal fluid (CSF) parameters in patients with spinal muscular atrophy (SMA) treated with Nusinersen","volume":"10","author":"Wurster","year":"2019","journal-title":"Front Neurol"},{"issue":"3","key":"10.1016\/j.tjpad.2025.100269_bib0062","doi-asserted-by":"crossref","DOI":"10.3390\/brainsci11030296","article-title":"Cerebrospinal fluid parameters in antisense oligonucleotide-treated adult 5q-spinal muscular atrophy patients","volume":"11","author":"Muschen","year":"2021","journal-title":"Brain Sci"},{"issue":"1","key":"10.1016\/j.tjpad.2025.100269_bib0063","doi-asserted-by":"crossref","DOI":"10.1016\/j.tjpad.2024.100001","article-title":"Key considerations for combination therapy in Alzheimer's clinical trials: perspectives from an expert advisory board convened by the Alzheimer's drug discovery foundation","volume":"12","author":"Cummings","year":"2025","journal-title":"J Prev Alzheimers Dis"},{"key":"10.1016\/j.tjpad.2025.100269_bib0064","article-title":"Amyloid reduction and dementia progression in dominantly inherited Alzheimer's Diseaseafter long-term gantenerumab treatment: results from the DIAN-TU trial","author":"Bateman","year":"2024","journal-title":"Preprints with The Lancet"},{"issue":"22","key":"10.1016\/j.tjpad.2025.100269_bib0065","doi-asserted-by":"crossref","first-page":"2302","DOI":"10.1056\/NEJMoa0806142","article-title":"Age, neuropathology, and dementia","volume":"360","author":"Savva","year":"2009","journal-title":"N Engl J Med"},{"issue":"4","key":"10.1016\/j.tjpad.2025.100269_bib0066","doi-asserted-by":"crossref","first-page":"1039","DOI":"10.3233\/JAD-142268","article-title":"Understanding the beneficial and detrimental effects of donepezil and rivastigmine to improve their therapeutic value","volume":"44","author":"Lane","year":"2015","journal-title":"J Alzheimer Dis"},{"issue":"3","key":"10.1016\/j.tjpad.2025.100269_bib0067","doi-asserted-by":"crossref","first-page":"323","DOI":"10.3390\/neuroglia5030022","article-title":"Functional glial activation mediates phenotypic effects of APOE\u025b4 and sex in Alzheimer\u2019s disease","volume":"5","author":"Lane","year":"2024","journal-title":"Neuroglia"},{"issue":"17","key":"10.1016\/j.tjpad.2025.100269_bib0068","first-page":"e2378","article-title":"Sex differences in CSF biomarkers vary by Alzheimer disease stage and APOE epsilon4 genotype","volume":"95","author":"Babapour","year":"2020","journal-title":"Neurology."},{"issue":"10\u201311","key":"10.1016\/j.tjpad.2025.100269_bib0069","doi-asserted-by":"crossref","first-page":"738","DOI":"10.1038\/s41434-023-00390-5","article-title":"Successes and challenges in clinical gene therapy","volume":"30","author":"Kohn","year":"2023","journal-title":"Gene Ther"},{"issue":"1","key":"10.1016\/j.tjpad.2025.100269_bib0070","doi-asserted-by":"crossref","DOI":"10.1002\/alz.14342","article-title":"The case for regulatory approval of amyloid-lowering immunotherapies in Alzheimer's disease based on clearcut biomarker evidence","volume":"21","author":"Aisen","year":"2025","journal-title":"Alzheimer Dement"},{"key":"10.1016\/j.tjpad.2025.100269_bib0071","unstructured":"Sciences W.L. Wave Life Sciences announces topline results from phase 1b\/2a FOCUS-C9 study of WVE-004 for C9orf72-associated amyotrophic Lateral sclerosis and frontotemporal dementia 2023 [Available from: https:\/\/ir.wavelifesciences.com\/news-releases\/news-release-details\/wave-life-sciences-announces-topline-results-phase-1b2a-focus-c9."},{"issue":"3","key":"10.1016\/j.tjpad.2025.100269_bib0072","doi-asserted-by":"crossref","first-page":"535","DOI":"10.1016\/j.cell.2016.03.014","article-title":"On the dependency of cellular protein levels on mRNA abundance","volume":"165","author":"Liu","year":"2016","journal-title":"Cell"},{"issue":"12","key":"10.1016\/j.tjpad.2025.100269_bib0073","doi-asserted-by":"crossref","first-page":"3580","DOI":"10.1111\/ene.15536","article-title":"APOE epsilon4 influences cognitive decline positively in APP and negatively in PSEN1 mutation carriers with autosomal-dominant Alzheimer's disease","volume":"29","author":"Almkvist","year":"2022","journal-title":"Eur J Neurol"},{"issue":"1","key":"10.1016\/j.tjpad.2025.100269_bib0074","doi-asserted-by":"crossref","first-page":"83","DOI":"10.1186\/s13073-022-01091-1","article-title":"Publisher correction: penetrance estimation of Alzheimer disease in SORL1 loss-of-function variant carriers using a family-based strategy and stratification by APOE genotypes","volume":"14","author":"Schramm","year":"2022","journal-title":"Genome Med"},{"key":"10.1016\/j.tjpad.2025.100269_bib0075","unstructured":"Long term follow-up after administration of Human gene therapy products. Food and Drug Administration; 2020."},{"issue":"7","key":"10.1016\/j.tjpad.2025.100269_bib0076","doi-asserted-by":"crossref","first-page":"645","DOI":"10.1016\/S1474-4422(22)00121-1","article-title":"Potential disease-modifying therapies for Huntington's disease: lessons learned and future opportunities","volume":"21","author":"Tabrizi","year":"2022","journal-title":"Lancet Neurol"},{"issue":"1","key":"10.1016\/j.tjpad.2025.100269_bib0077","doi-asserted-by":"crossref","first-page":"6265","DOI":"10.1038\/s41467-020-20096-1","article-title":"Patient and public perspectives on cell and gene therapies: a systematic review","volume":"11","author":"Aiyegbusi","year":"2020","journal-title":"Nat Commun"}],"container-title":["The Journal of Prevention of Alzheimer's Disease"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2274580725002122?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2274580725002122?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,9]],"date-time":"2025-11-09T07:39:11Z","timestamp":1762673951000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2274580725002122"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,9]]},"references-count":77,"journal-issue":{"issue":"8","published-print":{"date-parts":[[2025,9]]}},"alternative-id":["S2274580725002122"],"URL":"https:\/\/doi.org\/10.1016\/j.tjpad.2025.100269","relation":{},"ISSN":["2274-5807"],"issn-type":[{"value":"2274-5807","type":"print"}],"subject":[],"published":{"date-parts":[[2025,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"CTAD taskforce: genetic therapies in Alzheimer\u2019s disease","name":"articletitle","label":"Article Title"},{"value":"The Journal of Prevention of Alzheimer's Disease","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.tjpad.2025.100269","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2025 The Author(s). Published by Elsevier Masson SAS on behalf of SERDI Publisher.","name":"copyright","label":"Copyright"}],"article-number":"100269"}}