{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,2]],"date-time":"2026-10-02T00:06:01Z","timestamp":1790899561215,"version":"4.1.0"},"reference-count":197,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2023,3,1]],"date-time":"2023-03-01T00:00:00Z","timestamp":1677628800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2023,3,1]],"date-time":"2023-03-01T00:00:00Z","timestamp":1677628800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2024,3,1]],"date-time":"2024-03-01T00:00:00Z","timestamp":1709251200000},"content-version":"vor","delay-in-days":366,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100001279","name":"Great Ormond Street Hospital Charity","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100001279","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\/100014653","name":"Manchester Biomedical Research Centre","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100014653","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"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2023,3]]},"DOI":"10.1016\/j.ymthe.2022.11.015","type":"journal-article","created":{"date-parts":[[2022,11,30]],"date-time":"2022-11-30T11:22:00Z","timestamp":1669807320000},"page":"657-675","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":25,"title":["Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier"],"prefix":"10.1016","volume":"31","author":[{"given":"Bethan J.","family":"Critchley","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"H. Bobby","family":"Gaspar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8024-566X","authenticated-orcid":false,"given":"Sara","family":"Benedetti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2022.11.015_bib1","doi-asserted-by":"crossref","first-page":"27","DOI":"10.1038\/s41572-018-0025-4","article-title":"Lysosomal storage diseases","volume":"4","author":"Platt","year":"2018","journal-title":"Nat. Rev. Dis. Primers"},{"key":"10.1016\/j.ymthe.2022.11.015_bib2","doi-asserted-by":"crossref","first-page":"387","DOI":"10.1007\/s10545-010-9093-7","article-title":"The birth prevalence of lysosomal storage disorders in the Czech Republic: comparison with data in different populations","volume":"33","author":"Poupetov\u00e1","year":"2010","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.ymthe.2022.11.015_bib3","doi-asserted-by":"crossref","first-page":"189","DOI":"10.1002\/wnan.73","article-title":"New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders","volume":"2","author":"Muro","year":"2010","journal-title":"Wiley Interdiscip. Rev. Nanomed Nanobiotechnol"},{"key":"10.1016\/j.ymthe.2022.11.015_bib4","doi-asserted-by":"crossref","first-page":"1153","DOI":"10.1517\/14712598.2011.582036","article-title":"Gene therapy for lysosomal storage disorders","volume":"11","author":"Gritti","year":"2011","journal-title":"Expert Opin. Biol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib5","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1016\/j.addr.2017.05.004","article-title":"Lysosomal enzyme replacement therapies: historical development, clinical outcomes, and future perspectives","volume":"118","author":"Solomon","year":"2017","journal-title":"Adv. Drug Deliv. Rev."},{"key":"10.1016\/j.ymthe.2022.11.015_bib6","doi-asserted-by":"crossref","first-page":"1155","DOI":"10.1016\/j.ymthe.2017.03.025","article-title":"Hematopoietic stem cell gene therapy for storage disease: current and new indications","volume":"25","author":"Biffi","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib7","doi-asserted-by":"crossref","first-page":"433","DOI":"10.3389\/fped.2019.00433","article-title":"Hematopoietic stem cell transplantation in inborn errors of metabolism","volume":"7","author":"Tan","year":"2019","journal-title":"Front. Pediatr."},{"key":"10.1016\/j.ymthe.2022.11.015_bib8","doi-asserted-by":"crossref","first-page":"1065","DOI":"10.3390\/ijms17071065","article-title":"Less is more: substrate reduction therapy for lysosomal storage disorders","volume":"17","author":"Coutinho","year":"2016","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.ymthe.2022.11.015_bib9","doi-asserted-by":"crossref","first-page":"655","DOI":"10.1080\/14712598.2019.1607837","article-title":"An update on gene therapy for lysosomal storage disorders","volume":"19","author":"Nagree","year":"2019","journal-title":"Expert Opin. Biol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib10","doi-asserted-by":"crossref","first-page":"1138","DOI":"10.1038\/mt.2015.62","article-title":"Pharmacological chaperone therapy: preclinical development, clinical translation, and prospects for the treatment of lysosomal storage disorders","volume":"23","author":"Parenti","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib11","series-title":"Emery and Rimoin's Principles and Practice of Medical Genetics and Genomics","first-page":"205","article-title":"7 - Therapies for Lysosomal Storage Diseases","author":"Desnick","year":"2019"},{"key":"10.1016\/j.ymthe.2022.11.015_bib12","doi-asserted-by":"crossref","first-page":"709","DOI":"10.1016\/j.ymthe.2020.01.001","article-title":"Immune responses to viral gene therapy vectors","volume":"28","author":"Shirley","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib13","doi-asserted-by":"crossref","first-page":"929","DOI":"10.2174\/0929867323666160210130608","article-title":"Role of nanotechnology for enzyme replacement therapy in lysosomal diseases. A focus on gaucher's disease","volume":"23","author":"Mart\u00edn-Banderas","year":"2016","journal-title":"Curr. Med. Chem."},{"key":"10.1016\/j.ymthe.2022.11.015_bib14","doi-asserted-by":"crossref","first-page":"83","DOI":"10.1016\/j.ymgme.2018.11.011","article-title":"Enzyme enhancement therapeutics for lysosomal storage diseases: current status and perspective","volume":"126","author":"Thomas","year":"2019","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib15","doi-asserted-by":"crossref","first-page":"405","DOI":"10.3389\/fncel.2019.00405","article-title":"The evolving concept of the blood brain barrier (BBB): from a single static barrier to a heterogeneous and dynamic relay center","volume":"13","author":"Villabona-Rueda","year":"2019","journal-title":"Front. Cell. Neurosci."},{"key":"10.1016\/j.ymthe.2022.11.015_bib16","doi-asserted-by":"crossref","first-page":"1094","DOI":"10.1002\/glia.20990","article-title":"The perivascular astroglial sheath provides a complete covering of the brain microvessels: an electron microscopic 3D reconstruction","volume":"58","author":"Mathiisen","year":"2010","journal-title":"Glia"},{"key":"10.1016\/j.ymthe.2022.11.015_bib17","doi-asserted-by":"crossref","first-page":"152","DOI":"10.3109\/09687688.2014.937468","article-title":"The blood-brain barrier: structure, function and therapeutic approaches to cross it","volume":"31","author":"Tajes","year":"2014","journal-title":"Mol. Membr. Biol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib18","doi-asserted-by":"crossref","first-page":"3323","DOI":"10.1161\/STROKEAHA.110.608257","article-title":"Blood-brain barrier breakdown in acute and chronic cerebrovascular disease","volume":"42","author":"Yang","year":"2011","journal-title":"Stroke"},{"key":"10.1016\/j.ymthe.2022.11.015_bib19","doi-asserted-by":"crossref","first-page":"e23993","DOI":"10.4161\/tisb.23993","article-title":"Disruption of blood-brain barrier in Alzheimer disease pathogenesis","volume":"1","author":"Kook","year":"2013","journal-title":"Tissue Barriers"},{"key":"10.1016\/j.ymthe.2022.11.015_bib20","doi-asserted-by":"crossref","first-page":"272","DOI":"10.2741\/4206","article-title":"Disruption of the blood-brain barrier in Parkinson\u2019s disease: curse or route to a cure?","volume":"19","author":"Lee","year":"2014","journal-title":"Front. Biosci."},{"key":"10.1016\/j.ymthe.2022.11.015_bib21","first-page":"125","article-title":"Diabetes mellitus and blood-brain barrier dysfunction: an overview","volume":"2","author":"Prasad","year":"2014","journal-title":"J.\u00a0Pharmacovigil."},{"key":"10.1016\/j.ymthe.2022.11.015_bib22","doi-asserted-by":"crossref","DOI":"10.1128\/mBio.01675-19","article-title":"Gamma interferon alters junctional integrity via rho kinase, resulting in blood-brain barrier leakage in experimental viral encephalitis","volume":"10","author":"Bonney","year":"2019","journal-title":"mBio"},{"key":"10.1016\/j.ymthe.2022.11.015_bib23","doi-asserted-by":"crossref","first-page":"706","DOI":"10.1038\/s41467-019-08641-z","article-title":"Japanese encephalitis virus neuropenetrance is driven by mast cell chymase","volume":"10","author":"Hsieh","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2022.11.015_bib24","doi-asserted-by":"crossref","first-page":"1177","DOI":"10.2174\/1381612822666151221150733","article-title":"Evolving drug delivery strategies\u00a0to overcome the blood brain barrier","volume":"22","author":"Hersh","year":"2016","journal-title":"Curr. Pharm. Des."},{"key":"10.1016\/j.ymthe.2022.11.015_bib25","doi-asserted-by":"crossref","first-page":"749047","DOI":"10.3389\/fneur.2021.749047","article-title":"Focused ultrasound mediated opening of the blood-brain barrier for neurodegenerative diseases","volume":"12","author":"Fishman","year":"2021","journal-title":"Front. Neurol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib26","doi-asserted-by":"crossref","first-page":"e27877","DOI":"10.1371\/journal.pone.0027877","article-title":"Targeted delivery of neural stem cells to the brain using MRI-guided focused ultrasound to disrupt the blood-brain barrier","volume":"6","author":"Burgess","year":"2011","journal-title":"PLoS One"},{"key":"10.1016\/j.ymthe.2022.11.015_bib27","doi-asserted-by":"crossref","first-page":"109","DOI":"10.1186\/s13023-017-0649-6","article-title":"Transcranial pulsed ultrasound facilitates brain uptake of laronidase in enzyme replacement therapy for Mucopolysaccharidosis type I disease","volume":"12","author":"Hsu","year":"2017","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.ymthe.2022.11.015_bib28","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1023\/A:1007049806660","article-title":"Osmotic opening of the blood\u2013brain barrier: principles, mechanism, and therapeutic applications","volume":"20","author":"Rapoport","year":"2000","journal-title":"Cell. Mol. Neurobiol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib29","doi-asserted-by":"crossref","first-page":"1841","DOI":"10.1038\/sj.gt.3302081","article-title":"Widespread distribution of beta-hexosaminidase activity in the brain of a Sandhoff mouse model after coinjection of adenoviral vector and mannitol","volume":"10","author":"Bourgoin","year":"2003","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib30","doi-asserted-by":"crossref","first-page":"1065","DOI":"10.1038\/sj.gt.3302961","article-title":"Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice","volume":"14","author":"Fu","year":"2007","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib31","doi-asserted-by":"crossref","first-page":"1340","DOI":"10.1038\/gt.2009.85","article-title":"Mannitol-facilitated CNS entry of rAAV2 vector significantly delayed the neurological disease progression in MPS IIIB mice","volume":"16","author":"McCarty","year":"2009","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib32","doi-asserted-by":"crossref","first-page":"71","DOI":"10.1016\/j.jconrel.2014.09.018","article-title":"Intra-arterial delivery of AAV vectors to the mouse brain after mannitol mediated blood brain barrier disruption","volume":"196","author":"Foley","year":"2014","journal-title":"J.\u00a0Control Release"},{"key":"10.1016\/j.ymthe.2022.11.015_bib33","first-page":"82","article-title":"Mannitol: a review of its clinical uses","volume":"12","author":"Shawkat","year":"2012","journal-title":"Continuing Education Anaesth. Crit. Care Pain"},{"key":"10.1016\/j.ymthe.2022.11.015_bib34","doi-asserted-by":"crossref","first-page":"476","DOI":"10.1177\/0271678X15614589","article-title":"Pharmacologic manipulation of lysosomal enzyme transport across the blood-brain barrier","volume":"36","author":"Urayama","year":"2016","journal-title":"J.\u00a0Cereb. Blood Flow Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib35","doi-asserted-by":"crossref","first-page":"12873","DOI":"10.1073\/pnas.0705611104","article-title":"Epinephrine enhances lysosomal enzyme delivery across the blood\u2013brain barrier by up-regulation of the mannose 6-phosphate receptor","volume":"104","author":"Urayama","year":"2007","journal-title":"PNAS"},{"key":"10.1016\/j.ymthe.2022.11.015_bib36","doi-asserted-by":"crossref","first-page":"e0142347","DOI":"10.1371\/journal.pone.0142347","article-title":"Alpha adrenergic induction of transport of lysosomal enzyme across the blood-brain barrier","volume":"10","author":"Urayama","year":"2015","journal-title":"PLoS One"},{"key":"10.1016\/j.ymthe.2022.11.015_bib37","doi-asserted-by":"crossref","first-page":"178","DOI":"10.1016\/j.ymgme.2008.01.007","article-title":"Acidic amino acid tag enhances response to enzyme replacement in mucopolysaccharidosis type VII mice","volume":"94","author":"Monta\u00f1o","year":"2008","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib38","doi-asserted-by":"crossref","first-page":"2999","DOI":"10.1073\/pnas.1222742110","article-title":"Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrier","volume":"110","author":"Wang","year":"2013","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2022.11.015_bib39","doi-asserted-by":"crossref","first-page":"547","DOI":"10.1038\/mt.2013.267","article-title":"Effective intravenous therapy for neurodegenerative disease with a therapeutic enzyme and a peptide that mediates delivery to the brain","volume":"22","author":"Meng","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib40","doi-asserted-by":"crossref","first-page":"3122","DOI":"10.1523\/JNEUROSCI.4785-13.2014","article-title":"Comparison of five peptide vectors for improved brain delivery of the lysosomal enzyme arylsulfatase A","volume":"34","author":"B\u00f6ckenhoff","year":"2014","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.ymthe.2022.11.015_bib41","doi-asserted-by":"crossref","first-page":"2021","DOI":"10.1124\/dmd.112.046375","article-title":"Glycemic control and chronic dosing of rhesus monkeys with a fusion protein of iduronidase and a monoclonal antibody against the human insulin receptor","volume":"40","author":"Boado","year":"2012","journal-title":"Drug Metab. Dispos."},{"key":"10.1016\/j.ymthe.2022.11.015_bib42","doi-asserted-by":"crossref","first-page":"2317","DOI":"10.1002\/bit.25289","article-title":"Insulin receptor antibody-iduronate 2-sulfatase fusion protein: pharmacokinetics, anti-drug antibody, and safety pharmacology in Rhesus monkeys","volume":"111","author":"Boado","year":"2014","journal-title":"Biotechnol. Bioeng."},{"key":"10.1016\/j.ymthe.2022.11.015_bib43","doi-asserted-by":"crossref","first-page":"eaay1163","DOI":"10.1126\/scitranslmed.aay1163","article-title":"Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice","volume":"12","author":"Ullman","year":"2020","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2022.11.015_bib44","doi-asserted-by":"crossref","first-page":"1366","DOI":"10.1016\/j.ymthe.2018.02.032","article-title":"A blood-brain-barrier-penetrating anti-human transferrin receptor antibody fusion protein for neuronopathic mucopolysaccharidosis II","volume":"26","author":"Sonoda","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib45","doi-asserted-by":"crossref","first-page":"456","DOI":"10.1016\/j.ymthe.2018.12.005","article-title":"Iduronate-2-Sulfatase with anti-human transferrin receptor antibody for neuropathic mucopolysaccharidosis II: a phase 1\/2 trial","volume":"27","author":"Okuyama","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib46","doi-asserted-by":"crossref","first-page":"671","DOI":"10.1016\/j.ymthe.2020.09.039","article-title":"A phase 2\/3 trial of pabinafusp alfa, IDS fused with anti-human transferrin receptor antibody, targeting neurodegeneration in MPS-II","volume":"29","author":"Okuyama","year":"2021","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib47","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1254\/fpj.21080","article-title":"Pharmacological property, mechanism of action and clinical study results of Pabinafusp Alfa (Genetical Recombination) (IZCARGO((R)) I.V. Infusion 10\u00a0mg) as the therapeutic for Mucopolysaccharidosis type-II (Hunter syndrome)","volume":"157","author":"Yamamoto","year":"2022","journal-title":"Nihon Yakurigaku Zasshi."},{"key":"10.1016\/j.ymthe.2022.11.015_bib48","doi-asserted-by":"crossref","first-page":"2616","DOI":"10.1073\/pnas.0712147105","article-title":"Chemically modified \u03b2-glucuronidase crosses blood\u2013brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII","volume":"105","author":"Grubb","year":"2008","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2022.11.015_bib49","doi-asserted-by":"crossref","first-page":"17022","DOI":"10.1073\/pnas.1214779109","article-title":"Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII","volume":"109","author":"Huynh","year":"2012","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2022.11.015_bib50","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1016\/j.expneurol.2011.04.004","article-title":"Impact of high-dose, chemically modified sulfamidase on pathology in a murine model of MPS IIIA","volume":"230","author":"Rozaklis","year":"2011","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib51","first-page":"100510","article-title":"Intravenous delivery of a chemically modified sulfamidase efficiently reduces heparan sulfate storage and brain pathology in mucopolysaccharidosis IIIA mice","volume":"21","author":"Gustavsson","year":"2019","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.ymthe.2022.11.015_bib52","first-page":"100554","article-title":"Impact of chemical modification of sulfamidase on distribution to brain interstitial fluid and to CSF after an intravenous administration in awake, freely-moving rats","volume":"22","author":"Janson","year":"2020","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.ymthe.2022.11.015_bib53","doi-asserted-by":"crossref","first-page":"33","DOI":"10.1016\/j.ymgme.2015.03.002","article-title":"Determination of the role of injection site on the efficacy of intra-CSF enzyme replacement therapy in MPS IIIA mice","volume":"115","author":"Beard","year":"2015","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib54","doi-asserted-by":"crossref","first-page":"425","DOI":"10.1007\/s13346-019-00683-6","article-title":"Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B","volume":"10","author":"Grover","year":"2020","journal-title":"Drug Deliv. Transl. Res."},{"key":"10.1016\/j.ymthe.2022.11.015_bib55","doi-asserted-by":"crossref","first-page":"79","DOI":"10.1016\/j.expneurol.2014.09.008","article-title":"Delivery of therapeutic protein for prevention of neurodegenerative changes: comparison of different CSF-delivery methods","volume":"263","author":"Marshall","year":"2015","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib56","doi-asserted-by":"crossref","first-page":"325","DOI":"10.1016\/j.ymgme.2011.06.018","article-title":"Intrathecal tripeptidyl-peptidase 1 reduces lysosomal storage in a canine model of late infantile neuronal ceroid lipofuscinosis","volume":"104","author":"Vuillemenot","year":"2011","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib57","doi-asserted-by":"crossref","first-page":"1591","DOI":"10.1002\/jnr.23423","article-title":"Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease)","volume":"92","author":"Katz","year":"2014","journal-title":"J.\u00a0Neurosci. Res."},{"key":"10.1016\/j.ymthe.2022.11.015_bib58","doi-asserted-by":"crossref","first-page":"122","DOI":"10.1016\/j.ymgme.2012.05.005","article-title":"Enzyme replacement therapy (ERT) procedure for mucopolysaccharidosis type II (MPS II) by intraventricular administration (IVA) in murine MPS II","volume":"107","author":"Higuchi","year":"2012","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib59","doi-asserted-by":"crossref","first-page":"e16313","DOI":"10.1371\/journal.pone.0016313","article-title":"Comparative analysis of acid sphingomyelinase distribution in the CNS of rats and mice following intracerebroventricular delivery","volume":"6","author":"Treleaven","year":"2011","journal-title":"PLoS One"},{"key":"10.1016\/j.ymthe.2022.11.015_bib60","doi-asserted-by":"crossref","first-page":"261","DOI":"10.1016\/j.expneurol.2011.06.019","article-title":"Distribution of acid sphingomyelinase in rodent and non-human primate brain after intracerebroventricular infusion","volume":"231","author":"Ziegler","year":"2011","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib61","doi-asserted-by":"crossref","first-page":"349","DOI":"10.1016\/j.expneurol.2008.10.021","article-title":"Intracerebroventricular infusion of acid sphingomyelinase corrects CNS manifestations in a mouse model of Niemann-Pick A disease","volume":"215","author":"Dodge","year":"2009","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib62","doi-asserted-by":"crossref","first-page":"2520","DOI":"10.1096\/fj.06-6169com","article-title":"Single-dose intracerebroventricular administration of galactocerebrosidase improves survival in a mouse model of globoid cell leukodystrophy","volume":"21","author":"Lee","year":"2007","journal-title":"FASEB J."},{"key":"10.1016\/j.ymthe.2022.11.015_bib63","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1016\/j.ymgme.2005.04.013","article-title":"Penetration, diffusion, and uptake of recombinant human alpha-L-iduronidase after intraventricular injection into the rat brain","volume":"86","author":"Belichenko","year":"2005","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib64","doi-asserted-by":"crossref","first-page":"116","DOI":"10.1016\/j.ymgme.2013.09.008","article-title":"High-dose enzyme replacement therapy in murine Hurler syndrome","volume":"111","author":"Ou","year":"2014","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib65","doi-asserted-by":"crossref","first-page":"780","DOI":"10.1002\/ajmg.a.38073","article-title":"Intrathecal enzyme replacement therapy reverses cognitive decline in mucopolysaccharidosis type I","volume":"173","author":"Nestrasil","year":"2017","journal-title":"Am. J. Med. Genet. A."},{"key":"10.1016\/j.ymthe.2022.11.015_bib66","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1016\/j.ymgme.2015.05.005","article-title":"Intrathecal enzyme replacement therapy improves motor function and survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis","volume":"116","author":"Lu","year":"2015","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib67","doi-asserted-by":"crossref","first-page":"e30341","DOI":"10.1371\/journal.pone.0030341","article-title":"CNS penetration of intrathecal-lumbar idursulfase in the monkey, dog and mouse: implications for neurological outcomes of lysosomal storage disorder","volume":"7","author":"Calias","year":"2012","journal-title":"PLoS One"},{"key":"10.1016\/j.ymthe.2022.11.015_bib68","doi-asserted-by":"crossref","first-page":"513","DOI":"10.1111\/j.1600-0463.2011.02760.x","article-title":"Glycosaminoglycan storage in neuroanatomical regions of mucopolysaccharidosis I dogs following intrathecal recombinant human iduronidase","volume":"119","author":"Chen","year":"2011","journal-title":"APMIS"},{"key":"10.1016\/j.ymthe.2022.11.015_bib69","doi-asserted-by":"crossref","first-page":"422","DOI":"10.1016\/j.nbd.2011.04.014","article-title":"Enzyme replacement reduces neuropathology in MPS IIIA dogs","volume":"43","author":"Crawley","year":"2011","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.ymthe.2022.11.015_bib70","doi-asserted-by":"crossref","first-page":"218","DOI":"10.1016\/j.expneurol.2011.04.019","article-title":"Treatment of canine fucosidosis by intracisternal enzyme infusion","volume":"230","author":"Kondagari","year":"2011","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib71","doi-asserted-by":"crossref","first-page":"268","DOI":"10.1016\/j.ymgme.2011.03.011","article-title":"Biodistribution and pharmacodynamics of recombinant human alpha-L-iduronidase (rhIDU) in mucopolysaccharidosis type I-affected cats following multiple intrathecal administrations","volume":"103","author":"Vite","year":"2011","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib72","doi-asserted-by":"crossref","first-page":"1197","DOI":"10.1111\/j.1460-9568.2009.06666.x","article-title":"Examination of intravenous and intra-CSF protein delivery for treatment of neurological disease","volume":"29","author":"Hemsley","year":"2009","journal-title":"Eur. J. Neurosci."},{"key":"10.1016\/j.ymthe.2022.11.015_bib73","doi-asserted-by":"crossref","first-page":"383","DOI":"10.1016\/j.ymgme.2009.07.013","article-title":"Effect of cisternal sulfamidase delivery in MPS IIIA Huntaway dogs--a proof of principle study","volume":"98","author":"Hemsley","year":"2009","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib74","doi-asserted-by":"crossref","first-page":"61","DOI":"10.1016\/j.ymgme.2006.12.012","article-title":"Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid","volume":"91","author":"Dickson","year":"2007","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib75","doi-asserted-by":"crossref","first-page":"313","DOI":"10.1016\/j.ymgme.2006.10.005","article-title":"Injection of recombinant human sulfamidase into the CSF via the cerebellomedullary cistern in MPS IIIA mice","volume":"90","author":"Hemsley","year":"2007","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib76","doi-asserted-by":"crossref","first-page":"163","DOI":"10.1016\/j.ymgme.2004.07.003","article-title":"Intrathecal enzyme replacement therapy reduces lysosomal storage in the brain and meninges of the canine model of MPS I","volume":"83","author":"Kakkis","year":"2004","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib77","doi-asserted-by":"crossref","first-page":"2743","DOI":"10.1016\/j.ymthe.2017.08.007","article-title":"Guanidinylated neomycin conjugation enhances intranasal enzyme replacement in the brain","volume":"25","author":"Tong","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib78","doi-asserted-by":"crossref","first-page":"131","DOI":"10.1016\/j.ymgme.2012.02.006","article-title":"Lysosomal enzyme can bypass the blood-brain barrier and reach the CNS following intranasal administration","volume":"106","author":"Wolf","year":"2012","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib79","doi-asserted-by":"crossref","first-page":"e1801271","DOI":"10.1002\/adhm.201801271","article-title":"TPP1 delivery to lysosomes with extracellular vesicles and their enhanced brain distribution in the animal model of batten disease","volume":"8","author":"Haney","year":"2019","journal-title":"Adv. Healthc. Mater."},{"key":"10.1016\/j.ymthe.2022.11.015_bib80","doi-asserted-by":"crossref","first-page":"2591","DOI":"10.2217\/nnm-2017-0221","article-title":"Polyethylene glycol-b-poly(lactic acid) polymersomes as vehicles for enzyme replacement therapy","volume":"12","author":"Kelly","year":"2017","journal-title":"Nanomedicine"},{"key":"10.1016\/j.ymthe.2022.11.015_bib81","doi-asserted-by":"crossref","first-page":"467","DOI":"10.1007\/s10545-012-9534-6","article-title":"Comparative binding, endocytosis, and biodistribution of antibodies and antibody-coated carriers for targeted delivery of lysosomal enzymes to ICAM-1 versus transferrin receptor","volume":"36","author":"Papademetriou","year":"2013","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.ymthe.2022.11.015_bib82","doi-asserted-by":"crossref","first-page":"1293","DOI":"10.1002\/jnr.23805","article-title":"Quantum dots and potential therapy for Krabbe's disease","volume":"94","author":"Dawson","year":"2016","journal-title":"J.\u00a0Neurosci. Res."},{"key":"10.1016\/j.ymthe.2022.11.015_bib83","doi-asserted-by":"crossref","first-page":"045003","DOI":"10.1088\/2399-1984\/aadd34","article-title":"Quantitative analysis of recombinant glucocerebrosidase brain delivery via lipid nanoparticles","volume":"2","author":"Goldsmith","year":"2018","journal-title":"Nano Futures"},{"key":"10.1016\/j.ymthe.2022.11.015_bib84","doi-asserted-by":"crossref","first-page":"eaax7462","DOI":"10.1126\/sciadv.aax7462","article-title":"Brain-targeted enzyme-loaded nanoparticles: a breach through the blood-brain barrier for enzyme replacement therapy in Krabbe disease","volume":"5","author":"Del Grosso","year":"2019","journal-title":"Sci. Adv."},{"key":"10.1016\/j.ymthe.2022.11.015_bib85","doi-asserted-by":"crossref","first-page":"2014","DOI":"10.3390\/ijms20082014","article-title":"Targeting brain disease in MPSII: preclinical evaluation of IDS-loaded PLGA nanoparticles","volume":"20","author":"Rigon","year":"2019","journal-title":"Int. J. Mol. Sci."},{"key":"10.1016\/j.ymthe.2022.11.015_bib86","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.jconrel.2017.02.016","article-title":"Potential of surfactant-coated nanoparticles to improve brain delivery of arylsulfatase A","volume":"253","author":"Schuster","year":"2017","journal-title":"J.\u00a0Control Release"},{"key":"10.1016\/j.ymthe.2022.11.015_bib87","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1007\/s10545-016-9994-1","article-title":"Slow, continuous enzyme replacement via spinal CSF in dogs with the paediatric-onset neurodegenerative disease, MPS IIIA","volume":"40","author":"King","year":"2017","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.ymthe.2022.11.015_bib88","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1016\/j.expneurol.2015.11.013","article-title":"Low-dose, continual enzyme delivery ameliorates some aspects of established brain disease in a mouse model of a childhood-onset neurodegenerative disorder","volume":"278","author":"King","year":"2016","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib89","first-page":"59","article-title":"Continual low-dose infusion of sulfamidase is superior to intermittent high-dose delivery in ameliorating neuropathology in the MPS IIIA mouse brain","volume":"29","author":"Beard","year":"2016","journal-title":"JIMD Rep."},{"key":"10.1016\/j.ymthe.2022.11.015_bib90","doi-asserted-by":"crossref","first-page":"2760","DOI":"10.1093\/hmg\/ddr175","article-title":"Intracerebroventricular enzyme infusion corrects central nervous system pathology and dysfunction in a mouse model of metachromatic leukodystrophy","volume":"20","author":"Stroobants","year":"2011","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2022.11.015_bib91","doi-asserted-by":"crossref","first-page":"649","DOI":"10.1038\/mt.2008.9","article-title":"Intraventricular enzyme replacement improves disease phenotypes in a mouse model of late infantile neuronal ceroid lipofuscinosis","volume":"16","author":"Chang","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib92","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1038\/gim.2015.36","article-title":"A phase I\/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II","volume":"18","author":"Muenzer","year":"2016","journal-title":"Genet. Med."},{"key":"10.1016\/j.ymthe.2022.11.015_bib93","doi-asserted-by":"crossref","first-page":"1036","DOI":"10.1002\/ajmg.a.35869","article-title":"Improvement of CNS defects via continuous intrathecal enzyme replacement by\u00a0osmotic\u00a0pump in mucopolysaccharidosis type II mice","volume":"161A","author":"Sohn","year":"2013","journal-title":"Am. J. Med. Genet. A."},{"key":"10.1016\/j.ymthe.2022.11.015_bib94","unstructured":"https:\/\/www.clinigengroup.com\/news\/news-container\/2021\/clinigen-receives-marketing-approval-for-hunterase-idursulfase-beta-icv-in-japan\/. (Clinigen Group)."},{"key":"10.1016\/j.ymthe.2022.11.015_bib95","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"},{"key":"10.1016\/j.ymthe.2022.11.015_bib96","doi-asserted-by":"crossref","first-page":"254","DOI":"10.1212\/01.wnl.0000247744.10990.e6","article-title":"Image-guided, direct convective delivery of glucocerebrosidase for neuronopathic Gaucher disease","volume":"68","author":"Lonser","year":"2007","journal-title":"Neurology"},{"key":"10.1016\/j.ymthe.2022.11.015_bib97","doi-asserted-by":"crossref","first-page":"115","DOI":"10.3171\/2010.4.PEDS09507","article-title":"Gene therapy for late infantile neuronal ceroid lipofuscinosis: neurosurgical considerations","volume":"6","author":"Souweidane","year":"2010","journal-title":"J.\u00a0Neurosurg. Pediatr."},{"key":"10.1016\/j.ymthe.2022.11.015_bib98","doi-asserted-by":"crossref","first-page":"659","DOI":"10.1007\/s13311-011-0067-8","article-title":"Neural stem cell transplantation as a therapeutic approach for treating lysosomal storage diseases","volume":"8","author":"Shihabuddin","year":"2011","journal-title":"Neurotherapeutics"},{"key":"10.1016\/j.ymthe.2022.11.015_bib99","doi-asserted-by":"crossref","first-page":"749","DOI":"10.1089\/hum.2016.088","article-title":"Stem cell therapy for the central nervous system in lysosomal storage diseases","volume":"27","author":"Siddiqi","year":"2016","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib100","doi-asserted-by":"crossref","first-page":"e13707","DOI":"10.1111\/eci.13707","article-title":"Mesenchymal stem cells for lysosomal storage and polyglutamine disorders: possible shared mechanisms","volume":"52","author":"Oliveira Miranda","year":"2022","journal-title":"Eur. J. Clin. Invest."},{"key":"10.1016\/j.ymthe.2022.11.015_bib101","doi-asserted-by":"crossref","first-page":"e1701211","DOI":"10.1126\/sciadv.1701211","article-title":"Intracerebroventricular delivery\u00a0of hematopoietic progenitors results in rapid and robust engraftment of microglia-like cells","volume":"3","author":"Capotondo","year":"2017","journal-title":"Sci. Adv."},{"key":"10.1016\/j.ymthe.2022.11.015_bib102","doi-asserted-by":"crossref","first-page":"15018","DOI":"10.1073\/pnas.1205858109","article-title":"Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation","volume":"109","author":"Capotondo","year":"2012","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2022.11.015_bib103","doi-asserted-by":"crossref","first-page":"439","DOI":"10.1038\/nm1548","article-title":"Stem cells act through multiple mechanisms to benefit mice with neurodegenerative metabolic disease","volume":"13","author":"Lee","year":"2007","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2022.11.015_bib104","doi-asserted-by":"crossref","first-page":"868","DOI":"10.1038\/mt.2013.29","article-title":"Busulfan conditioning enhances engraftment of hematopoietic donor-derived cells in the brain compared with irradiation","volume":"21","author":"Wilkinson","year":"2013","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib105","doi-asserted-by":"crossref","first-page":"517","DOI":"10.1038\/s41591-022-01691-9","article-title":"Hematopoietic stem cell transplantation chemotherapy causes microglia senescence and peripheral macrophage engraftment in the brain","volume":"28","author":"Sailor","year":"2022","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2022.11.015_bib106","doi-asserted-by":"crossref","first-page":"2720","DOI":"10.1016\/j.transproceed.2010.04.024","article-title":"Vascular endothelium changes after conditioning in hematopoietic stem cell transplantation: role of cyclophosphamide and busulfan","volume":"42","author":"Zeng","year":"2010","journal-title":"Transpl. Proc."},{"key":"10.1016\/j.ymthe.2022.11.015_bib107","doi-asserted-by":"crossref","first-page":"299","DOI":"10.1111\/j.1600-0609.2008.01031.x","article-title":"Endothelial cells do not express GSTA1: potential relevance to busulfan-mediated endothelial damage during hematopoetic stem cell transplantation","volume":"80","author":"Vassord","year":"2008","journal-title":"Eur. J. Haematol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib108","doi-asserted-by":"crossref","first-page":"523","DOI":"10.1016\/j.bbmt.2008.12.489","article-title":"Busulfan in hematopoietic stem cell transplantation","volume":"15","author":"Ciurea","year":"2009","journal-title":"Biol. Blood Marrow Transpl."},{"key":"10.1016\/j.ymthe.2022.11.015_bib109","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1038\/s41467-018-08201-x","article-title":"Selective hematopoietic stem cell ablation using CD117-antibody-drug-conjugates enables safe and effective transplantation with immunity preservation","volume":"10","author":"Czechowicz","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2022.11.015_bib110","doi-asserted-by":"crossref","first-page":"232","DOI":"10.1016\/j.ymgme.2016.08.003","article-title":"Non-myeloablative preconditioning with ACK2 (anti-c-kit antibody) is efficient in bone marrow transplantation for murine models of mucopolysaccharidosis type II","volume":"119","author":"Yokoi","year":"2016","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib111","doi-asserted-by":"crossref","first-page":"351ra105","DOI":"10.1126\/scitranslmed.aae0501","article-title":"Hematopoietic stem cell transplantation in immunocompetent hosts without radiation or chemotherapy","volume":"8","author":"Chhabra","year":"2016","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2022.11.015_bib112","doi-asserted-by":"crossref","first-page":"S92","DOI":"10.1016\/j.bbmt.2018.12.172","article-title":"Toxicity-free hematopoietic stem cell engraftment achieved with anti-CD117 monoclonal antibody conditioning","volume":"25","author":"Agarwal","year":"2019","journal-title":"Biol. Blood Marrow Transplant."},{"key":"10.1016\/j.ymthe.2022.11.015_bib113","doi-asserted-by":"crossref","first-page":"1130","DOI":"10.1182\/blood-2011-09-380436","article-title":"Durable donor engraftment after radioimmunotherapy using alpha-emitter astatine-211-labeled anti-CD45 antibody for conditioning in allogeneic hematopoietic cell transplantation","volume":"119","author":"Chen","year":"2012","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2022.11.015_bib114","doi-asserted-by":"crossref","first-page":"738","DOI":"10.1038\/nbt.3584","article-title":"Non-genotoxic conditioning for hematopoietic stem cell transplantation using a hematopoietic-cell-specific internalizing immunotoxin","volume":"34","author":"Palchaudhuri","year":"2016","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib115","doi-asserted-by":"crossref","first-page":"185","DOI":"10.1016\/j.stem.2019.05.018","article-title":"Antibody conditioning enables MHC-mismatched hematopoietic stem cell transplants and organ graft tolerance","volume":"25","author":"George","year":"2019","journal-title":"Cell Stem Cell"},{"key":"10.1016\/j.ymthe.2022.11.015_bib116","doi-asserted-by":"crossref","first-page":"3758","DOI":"10.1038\/s41467-019-11674-z","article-title":"Sustained microglial depletion with CSF1R inhibitor impairs parenchymal plaque development in an Alzheimer's disease model","volume":"10","author":"Spangenberg","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.ymthe.2022.11.015_bib117","doi-asserted-by":"crossref","first-page":"108443","DOI":"10.1016\/j.celrep.2020.108443","article-title":"Efficient strategies for microglia replacement in the central nervous system","volume":"33","author":"Xu","year":"2020","journal-title":"Cell Rep."},{"key":"10.1016\/j.ymthe.2022.11.015_bib118","doi-asserted-by":"crossref","first-page":"e8730","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."},{"key":"10.1016\/j.ymthe.2022.11.015_bib119","doi-asserted-by":"crossref","first-page":"3070","DOI":"10.1172\/JCI28873","article-title":"Gene therapy of metachromatic leukodystrophy reverses neurological damage and deficits in mice","volume":"116","author":"Biffi","year":"2006","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.ymthe.2022.11.015_bib120","doi-asserted-by":"crossref","first-page":"1857","DOI":"10.1182\/blood-2009-12-256461","article-title":"The galactocerebrosidase enzyme contributes to the maintenance of a functional hematopoietic stem cell niche","volume":"116","author":"Visigalli","year":"2010","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2022.11.015_bib121","doi-asserted-by":"crossref","first-page":"1938","DOI":"10.1038\/mt.2013.141","article-title":"Myeloid\/Microglial driven autologous hematopoietic stem cell gene therapy corrects a neuronopathic lysosomal disease","volume":"21","author":"Sergijenko","year":"2013","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib122","doi-asserted-by":"crossref","first-page":"99","DOI":"10.1093\/brain\/awx311","article-title":"Macrophage enzyme and reduced inflammation drive brain correction of mucopolysaccharidosis IIIB by stem cell gene therapy","volume":"141","author":"Holley","year":"2018","journal-title":"Brain."},{"key":"10.1016\/j.ymthe.2022.11.015_bib123","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\u00a0vivo stem cell gene therapy for the treatment of mucopolysaccharidosis IIIA","volume":"13","author":"Ellison","year":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2022.11.015_bib124","doi-asserted-by":"crossref","first-page":"333","DOI":"10.1016\/j.omtm.2019.10.009","article-title":"Enhancing the therapeutic potential of sulfamidase for the treatment of mucopolysaccharidosis IIIA","volume":"15","author":"Sorrentino","year":"2019","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2022.11.015_bib125","doi-asserted-by":"crossref","first-page":"2693","DOI":"10.1093\/hmg\/ddm223","article-title":"Functional correction of CNS lesions in an MPS-IIIA mouse model by intracerebral AAV-mediated delivery of sulfamidase and SUMF1 genes","volume":"16","author":"Fraldi","year":"2007","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2022.11.015_bib126","doi-asserted-by":"crossref","first-page":"617","DOI":"10.1006\/mthe.2002.0594","article-title":"Enhanced secretion and uptake of beta-glucuronidase improves adeno-associated viral-mediated gene therapy of mucopolysaccharidosis type VII mice","volume":"5","author":"Elliger","year":"2002","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib127","doi-asserted-by":"crossref","first-page":"1233158","DOI":"10.1126\/science.1233158","article-title":"Lentiviral hematopoietic stem cell gene therapy benefits metachromatic leukodystrophy","volume":"341","author":"Biffi","year":"2013","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2022.11.015_bib128","doi-asserted-by":"crossref","first-page":"476","DOI":"10.1016\/S0140-6736(16)30374-9","article-title":"Lentiviral haemopoietic stem-cell gene therapy in early-onset metachromatic leukodystrophy: an ad-hoc analysis of a non-randomised, open-label, phase 1\/2 trial","volume":"388","author":"Sessa","year":"2016","journal-title":"Lancet"},{"key":"10.1016\/j.ymthe.2022.11.015_bib129","doi-asserted-by":"crossref","first-page":"372","DOI":"10.1016\/S0140-6736(21)02017-1","article-title":"Lentiviral hematopoetic 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.ymthe.2022.11.015_bib130","doi-asserted-by":"crossref","first-page":"187","DOI":"10.1016\/B978-0-12-386509-0.00010-7","article-title":"Lentiviral hematopoietic cell gene therapy for X-linked adrenoleukodystrophy","volume":"507","author":"Cartier","year":"2012","journal-title":"Methods Enzymol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib131","doi-asserted-by":"crossref","first-page":"818","DOI":"10.1126\/science.1171242","article-title":"Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy","volume":"326","author":"Cartier","year":"2009","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2022.11.015_bib132","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.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2022.11.015_bib133","unstructured":"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/libmeldy. (European Medicines Agency)."},{"key":"10.1016\/j.ymthe.2022.11.015_bib134","unstructured":"https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/skysona. (European Medicines Agency)."},{"key":"10.1016\/j.ymthe.2022.11.015_bib135","doi-asserted-by":"crossref","first-page":"1475","DOI":"10.1038\/sj.gt.3302338","article-title":"Brain transplantation of genetically modified bone marrow stromal cells corrects CNS pathology and cognitive function in MPS VII mice","volume":"11","author":"Sakurai","year":"2004","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib136","doi-asserted-by":"crossref","first-page":"1577","DOI":"10.3727\/096368912X636894","article-title":"Intracerebroventricular transplantation of human bone marrow-derived multipotent progenitor cells in an immunodeficient mouse model of mucopolysaccharidosis type I (MPS-I)","volume":"21","author":"Nan","year":"2012","journal-title":"Cell Transpl."},{"key":"10.1016\/j.ymthe.2022.11.015_bib137","doi-asserted-by":"crossref","first-page":"179","DOI":"10.1016\/j.nbd.2011.09.014","article-title":"Viral vectors for gene delivery to the central nervous system","volume":"48","author":"Lentz","year":"2012","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.ymthe.2022.11.015_bib138","doi-asserted-by":"crossref","first-page":"611","DOI":"10.3390\/biom11040611","article-title":"Gene therapy for lysosomal storage disorders: ongoing studies and clinical development","volume":"11","author":"Massaro","year":"2021","journal-title":"Biomolecules"},{"key":"10.1016\/j.ymthe.2022.11.015_bib139","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1038\/nbt.1515","article-title":"Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes","volume":"27","author":"Foust","year":"2009","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib140","doi-asserted-by":"crossref","first-page":"478","DOI":"10.1089\/hum.2016.087","article-title":"Adeno-associated virus-based gene therapy for CNS diseases","volume":"27","author":"Hocquemiller","year":"2016","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib141","doi-asserted-by":"crossref","first-page":"755","DOI":"10.1080\/14712598.2018.1492544","article-title":"Progress in gene and cell therapies for the neuronal ceroid lipofuscinoses","volume":"18","author":"Donsante","year":"2018","journal-title":"Expert Opin. Biol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib142","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1089\/hum.2018.016","article-title":"Systemic delivery of AAVB1-GAA clears glycogen and prolongs survival in a mouse model of pompe disease","volume":"30","author":"Keeler","year":"2019","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib143","doi-asserted-by":"crossref","first-page":"16036","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.ymthe.2022.11.015_bib144","doi-asserted-by":"crossref","first-page":"94","DOI":"10.1016\/j.jconrel.2016.09.011","article-title":"Gene therapy for the CNS using AAVs: the impact of systemic delivery by AAV9","volume":"241","author":"Saraiva","year":"2016","journal-title":"J.\u00a0Control Release"},{"key":"10.1016\/j.ymthe.2022.11.015_bib145","unstructured":"https:\/\/investors.abeonatherapeutics.com\/press-releases\/detail\/234\/ultragenyx-acquires-global-rights-to-aav-gene-therapy. (Abeona Therapeutics)."},{"key":"10.1016\/j.ymthe.2022.11.015_bib146","doi-asserted-by":"crossref","first-page":"S46","DOI":"10.1016\/j.ymgme.2017.12.103","article-title":"A phase 1\/2 clinical trial of systemic gene transfer of scAAV9.U1a.HSGSH for MPS IIIA: safety, tolerability, and preliminary evidence of biopotency","volume":"123","author":"Flanigan","year":"2018","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib147","unstructured":"https:\/\/clinicaltrials.gov\/ct2\/show\/NCT04088734. (ClinicalTrials.gov)."},{"key":"10.1016\/j.ymthe.2022.11.015_bib148","unstructured":"https:\/\/investors.abeonatherapeutics.com\/press-releases\/detail\/174\/abeona-therapeutics-announces-positive-interim-data-from. (Abeona Therapeutics)."},{"key":"10.1016\/j.ymthe.2022.11.015_bib149","doi-asserted-by":"crossref","first-page":"541","DOI":"10.1038\/s41592-020-0799-7","article-title":"Multiplexed Cre-dependent selection yields systemic AAVs for targeting distinct brain cell types","volume":"17","author":"Ravindra Kumar","year":"2020","journal-title":"Nat. Methods"},{"key":"10.1016\/j.ymthe.2022.11.015_bib150","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.ymthe.2022.11.015_bib151","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."},{"key":"10.1016\/j.ymthe.2022.11.015_bib152","doi-asserted-by":"crossref","first-page":"2242","DOI":"10.1016\/j.neuron.2022.05.003","article-title":"Engineered AAVs for non-invasive gene delivery to rodent and non-human primate nervous systems","volume":"110","author":"Chen","year":"2022","journal-title":"Neuron"},{"key":"10.1016\/j.ymthe.2022.11.015_bib153","doi-asserted-by":"crossref","first-page":"699","DOI":"10.1038\/mt.2011.287","article-title":"The AAV vector toolkit: poised at the clinical crossroads","volume":"20","author":"Asokan","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib154","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1089\/hum.2018.015","article-title":"Severe toxicity in nonhuman primates and piglets following high-dose intravenous administration of an adeno-associated virus vector expressing human SMN","volume":"29","author":"Hinderer","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib155","doi-asserted-by":"crossref","first-page":"695","DOI":"10.1089\/hum.2020.182","article-title":"Moving forward after two deaths in a gene therapy trial of myotubular myopathy","volume":"31","author":"Wilson","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib156","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1016\/j.omtm.2017.11.007","article-title":"Emerging issues in AAV-mediated in\u00a0vivo gene therapy","volume":"8","author":"Colella","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.ymthe.2022.11.015_bib157","doi-asserted-by":"crossref","first-page":"517","DOI":"10.4155\/tde.10.50","article-title":"Viral vectors and delivery strategies for CNS gene therapy","volume":"1","author":"Gray","year":"2010","journal-title":"Ther. Deliv."},{"key":"10.1016\/j.ymthe.2022.11.015_bib158","doi-asserted-by":"crossref","first-page":"463","DOI":"10.1089\/hum.2008.022","article-title":"Treatment of late infantile neuronal ceroid lipofuscinosis by CNS administration of a serotype 2 adeno-associated virus expressing CLN2 cDNA","volume":"19","author":"Worgall","year":"2008","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib159","doi-asserted-by":"crossref","first-page":"eabb5413","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."},{"key":"10.1016\/j.ymthe.2022.11.015_bib160","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."},{"key":"10.1016\/j.ymthe.2022.11.015_bib161","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."},{"key":"10.1016\/j.ymthe.2022.11.015_bib162","doi-asserted-by":"crossref","first-page":"1298","DOI":"10.1038\/mt.2015.99","article-title":"Neonatal systemic AAV induces tolerance to CNS gene therapy in MPS I dogs and nonhuman primates","volume":"23","author":"Hinderer","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib163","doi-asserted-by":"crossref","first-page":"1211","DOI":"10.1089\/hum.2019.217","article-title":"In\u00a0vivo gene therapy for mucopolysaccharidosis type III (sanfilippo syndrome): a new treatment horizon","volume":"30","author":"Marc\u00f3","year":"2019","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib164","doi-asserted-by":"crossref","first-page":"215","DOI":"10.1038\/gt.2017.4","article-title":"Extraneuronal pathology in a canine model of CLN2 neuronal ceroid lipofuscinosis after intracerebroventricular gene therapy that delays neurological disease progression","volume":"24","author":"Katz","year":"2017","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib165","doi-asserted-by":"crossref","first-page":"276","DOI":"10.1016\/j.exer.2016.03.023","article-title":"Intracerebroventricular gene therapy that delays neurological disease progression is associated with selective preservation of retinal ganglion cells in a canine model of CLN2 disease","volume":"146","author":"Whiting","year":"2016","journal-title":"Exp. Eye Res."},{"key":"10.1016\/j.ymthe.2022.11.015_bib166","doi-asserted-by":"crossref","first-page":"123","DOI":"10.1016\/j.nbd.2011.02.015","article-title":"Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I","volume":"43","author":"Wolf","year":"2011","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.ymthe.2022.11.015_bib167","doi-asserted-by":"crossref","first-page":"374","DOI":"10.1002\/jgm.2816","article-title":"Correction of murine mucopolysaccharidosis type IIIA central nervous system pathology by intracerebroventricular lentiviral-mediated gene delivery","volume":"16","author":"McIntyre","year":"2014","journal-title":"J.\u00a0Gene Med."},{"key":"10.1016\/j.ymthe.2022.11.015_bib168","doi-asserted-by":"crossref","first-page":"1052","DOI":"10.1089\/hum.2018.189","article-title":"An improved adeno-associated virus vector for neurological correction of the mouse model of mucopolysaccharidosis IIIA","volume":"30","author":"Gray","year":"2019","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib169","doi-asserted-by":"crossref","first-page":"eaam6375","DOI":"10.1126\/scitranslmed.aam6375","article-title":"Rescue of Pompe disease in mice by AAV-mediated liver delivery of secretable acid alpha-glucosidase","volume":"9","author":"Puzzo","year":"2017","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2022.11.015_bib170","doi-asserted-by":"crossref","first-page":"213","DOI":"10.1016\/j.ymgme.2012.05.009","article-title":"Intravenous high-dose enzyme replacement therapy with recombinant palmitoyl-protein thioesterase reduces visceral lysosomal storage and modestly prolongs survival in a preclinical mouse model of infantile neuronal ceroid lipofuscinosis","volume":"107","author":"Hu","year":"2012","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib171","doi-asserted-by":"crossref","first-page":"60ra89","DOI":"10.1126\/scitranslmed.3001380","article-title":"Replacing the enzyme alpha-L-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I","volume":"2","author":"Dierenfeld","year":"2010","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2022.11.015_bib172","doi-asserted-by":"crossref","first-page":"611","DOI":"10.1007\/s10545-010-9158-7","article-title":"Early initiation of enzyme replacement therapy improves metabolic correction in the brain tissue of aspartylglycosaminuria mice","volume":"33","author":"Dunder","year":"2010","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.ymthe.2022.11.015_bib173","doi-asserted-by":"crossref","first-page":"2233","DOI":"10.1016\/j.bbmt.2018.06.020","article-title":"Long-term functional outcomes after hematopoietic stem cell transplant for early infantile Krabbe disease","volume":"24","author":"Allewelt","year":"2018","journal-title":"Biol. Blood Marrow Transpl."},{"key":"10.1016\/j.ymthe.2022.11.015_bib174","first-page":"81","article-title":"Effectiveness of early hematopoietic stem cell transplantation in preventing neurocognitive decline in mucopolysaccharidosis type II: a case series","volume":"41","author":"Selvanathan","year":"2018","journal-title":"JIMD Rep."},{"key":"10.1016\/j.ymthe.2022.11.015_bib175","doi-asserted-by":"crossref","first-page":"157","DOI":"10.1016\/j.ymgme.2017.05.001","article-title":"Favourable effect of early versus late start of enzyme replacement therapy on plasma globotriaosylsphingosine levels in men with classical Fabry disease","volume":"121","author":"Arends","year":"2017","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib176","first-page":"62","article-title":"Early hematopoietic stem cell transplantation in a patient with severe mucopolysaccharidosis II: a 7 years follow-up","volume":"12","author":"Barth","year":"2017","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.ymthe.2022.11.015_bib177","doi-asserted-by":"crossref","first-page":"63","DOI":"10.1016\/j.ymgme.2013.11.015","article-title":"Early initiation of enzyme replacement therapy for the mucopolysaccharidoses","volume":"111","author":"Muenzer","year":"2014","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib178","doi-asserted-by":"crossref","first-page":"eaay8980","DOI":"10.1126\/scitranslmed.aay8980","article-title":"Tolerance induction and microglial engraftment after fetal therapy without conditioning in mice with Mucopolysaccharidosis type VII","volume":"12","author":"Nguyen","year":"2020","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.ymthe.2022.11.015_bib179","doi-asserted-by":"crossref","first-page":"1","DOI":"10.52793\/JSCR.2021.2(2)-S5","article-title":"In utero hematopoietic stem cell transplantation","volume":"2","author":"Vogel","year":"2021","journal-title":"J.\u00a0Stem Cell Res."},{"key":"10.1016\/j.ymthe.2022.11.015_bib180","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.ymgme.2012.02.003","article-title":"Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I","volume":"106","author":"Dickson","year":"2012","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2022.11.015_bib181","doi-asserted-by":"crossref","first-page":"977","DOI":"10.1038\/mt.2010.40","article-title":"AAV vectors avoid inflammatory signals necessary to render transduced hepatocyte targets for destructive T\u00a0cells","volume":"18","author":"Somanathan","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib182","doi-asserted-by":"crossref","first-page":"3038","DOI":"10.1182\/blood-2011-09-382317","article-title":"Factor IX expression in skeletal muscle of a severe hemophilia B patient 10 years after AAV-mediated gene transfer","volume":"119","author":"Buchlis","year":"2012","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2022.11.015_bib183","doi-asserted-by":"crossref","first-page":"1994","DOI":"10.1056\/NEJMoa1407309","article-title":"Long-term safety and efficacy of factor IX gene therapy in hemophilia B","volume":"371","author":"Nathwani","year":"2014","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2022.11.015_bib184","doi-asserted-by":"crossref","first-page":"346","DOI":"10.1038\/nrg1066","article-title":"Progress and problems with the use of viral vectors for gene therapy","volume":"4","author":"Thomas","year":"2003","journal-title":"Nat. Rev. Genet."},{"key":"10.1016\/j.ymthe.2022.11.015_bib185","doi-asserted-by":"crossref","first-page":"3631","DOI":"10.1038\/s41598-019-40071-1","article-title":"Vector uncoating limits adeno-associated viral vector-mediated transduction of human dendritic cells and vector immunogenicity","volume":"9","author":"Rossi","year":"2019","journal-title":"Sci. Rep."},{"key":"10.1016\/j.ymthe.2022.11.015_bib186","doi-asserted-by":"crossref","first-page":"6381","DOI":"10.1128\/JVI.78.12.6381-6388.2004","article-title":"Clades of Adeno-associated viruses are widely disseminated in human tissues","volume":"78","author":"Gao","year":"2004","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib187","doi-asserted-by":"crossref","first-page":"704","DOI":"10.1089\/hum.2009.182","article-title":"Prevalence of serum IgG and neutralizing factors against adeno-associated virus (AAV) types 1, 2, 5, 6, 8, and 9 in the healthy population: implications for gene therapy using AAV vectors","volume":"21","author":"Boutin","year":"2010","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib188","doi-asserted-by":"crossref","first-page":"59","DOI":"10.1089\/hgtb.2012.243","article-title":"Pre-existing anti-adeno-associated virus antibodies as a challenge in AAV gene therapy","volume":"24","author":"Louis Jeune","year":"2013","journal-title":"Hum. Gene Ther. Methods"},{"key":"10.1016\/j.ymthe.2022.11.015_bib189","doi-asserted-by":"crossref","first-page":"723","DOI":"10.1016\/j.ymthe.2019.12.010","article-title":"AAV vector immunogenicity in humans: a long journey to successful gene transfer","volume":"28","author":"Verdera","year":"2020","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib190","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."},{"key":"10.1016\/j.ymthe.2022.11.015_bib191","doi-asserted-by":"crossref","first-page":"803","DOI":"10.1007\/s40291-021-00555-1","article-title":"Autotemcel: first approval","volume":"25","author":"Keam","year":"2021","journal-title":"Mol. Diagn. Ther."},{"key":"10.1016\/j.ymthe.2022.11.015_bib192","unstructured":"https:\/\/investor.bluebirdbio.com\/news-releases\/news-release-details\/bluebird-bio-receives-fda-accelerated-approval-skysonar-gene. (bluebird bio)."},{"key":"10.1016\/j.ymthe.2022.11.015_bib193","doi-asserted-by":"crossref","first-page":"51","DOI":"10.1186\/s12962-022-00369-w","article-title":"A systematic review of economic evaluations of enzyme replacement therapy in Lysosomal storage diseases","volume":"20","author":"Katsigianni","year":"2022","journal-title":"Cost Eff. Resour. Alloc."},{"key":"10.1016\/j.ymthe.2022.11.015_bib194","doi-asserted-by":"crossref","first-page":"1311","DOI":"10.1038\/s41591-021-01481-9","article-title":"Gene therapies should be for all","volume":"27","year":"2021","journal-title":"Nat. Med."},{"key":"10.1016\/j.ymthe.2022.11.015_bib195","first-page":"674","article-title":"Gene therapy may not be as\u00a0expensive as people think: challenges in assessing the value of single and short-term\u00a0therapies","volume":"27","author":"Garrison","year":"2021","journal-title":"J.\u00a0Manag. Care Spec. Pharm."},{"key":"10.1016\/j.ymthe.2022.11.015_bib196","doi-asserted-by":"crossref","first-page":"885639","DOI":"10.3389\/fonc.2022.885639","article-title":"New indications for hematopoietic stem cell gene therapy in lysosomal storage disorders","volume":"12","author":"Rossini","year":"2022","journal-title":"Front. Oncol."},{"key":"10.1016\/j.ymthe.2022.11.015_bib197","first-page":"639","article-title":"Combination therapies for lysosomal storage diseases: a complex answer to a simple problem","volume":"13","author":"Macauley","year":"2016","journal-title":"Pediatr. Endocrinol. Rev."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001622006736?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001622006736?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,26]],"date-time":"2025-10-26T18:07:53Z","timestamp":1761502073000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001622006736"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2023,3]]},"references-count":197,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2023,3]]}},"alternative-id":["S1525001622006736"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2022.11.015","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2023,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Targeting the central nervous system in lysosomal storage diseases: Strategies to deliver therapeutics across the blood-brain barrier","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2022.11.015","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2022 The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}