{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,2]],"date-time":"2026-07-02T18:29:48Z","timestamp":1783016988188,"version":"3.54.6"},"reference-count":84,"publisher":"Elsevier BV","issue":"3","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":[[2025,6,5]],"date-time":"2025-06-05T00:00:00Z","timestamp":1749081600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100016036","name":"Health~Holland","doi-asserted-by":"publisher","award":["LSHM22014"],"award-info":[{"award-number":["LSHM22014"]}],"id":[{"id":"10.13039\/100016036","id-type":"DOI","asserted-by":"publisher"}]},{"name":"Start2Cure Foundation","award":["0-TI-01"],"award-info":[{"award-number":["0-TI-01"]}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy Methods &amp; Clinical Development"],"published-print":{"date-parts":[[2025,9]]},"DOI":"10.1016\/j.omtm.2025.101504","type":"journal-article","created":{"date-parts":[[2025,6,10]],"date-time":"2025-06-10T02:56:20Z","timestamp":1749524180000},"page":"101504","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":3,"title":["Developing a minimally invasive gene therapy for multiple sclerosis"],"prefix":"10.1016","volume":"33","author":[{"ORCID":"https:\/\/orcid.org\/0009-0007-1846-2675","authenticated-orcid":false,"given":"Paul J.H.","family":"Nijhuis","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maurits","family":"Romijn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roy","family":"Honing","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giselle","family":"van Zon","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Inge","family":"Huitinga","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fred","family":"de Winter","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joost","family":"Verhaagen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2025.101504_bib1","doi-asserted-by":"crossref","first-page":"511","DOI":"10.1007\/s00401-018-1818-y","article-title":"Progressive multiple sclerosis patients show substantial lesion activity that correlates with clinical disease severity and sex: a retrospective autopsy cohort analysis","volume":"135","author":"Luchetti","year":"2018","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.omtm.2025.101504_bib2","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1089\/hum.2019.367","article-title":"Postmortem analysis in a clinical trial of AAV2-NGF gene therapy for Alzheimer\u2019s disease identifies a need for improved vector delivery","volume":"31","author":"Castle","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2025.101504_bib3","doi-asserted-by":"crossref","first-page":"835","DOI":"10.1080\/14737175.2020.1776609","article-title":"Perivascular tissue resident memory T cells as therapeutic target in multiple sclerosis","volume":"20","author":"Smolders","year":"2020","journal-title":"Expert Rev. Neurother."},{"key":"10.1016\/j.omtm.2025.101504_bib4","doi-asserted-by":"crossref","first-page":"641","DOI":"10.1038\/nrd.2018.110","article-title":"Gene therapy for neurological disorders: Progress and prospects","volume":"17","author":"Deverman","year":"2018","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.omtm.2025.101504_bib5","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.omtm.2025.101504_bib6","doi-asserted-by":"crossref","first-page":"1172","DOI":"10.1038\/nn.4593","article-title":"Engineered AAVs for efficient noninvasive gene delivery to the central and peripheral nervous systems","volume":"20","author":"Chan","year":"2017","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.omtm.2025.101504_bib7","doi-asserted-by":"crossref","first-page":"579","DOI":"10.1038\/s41434-020-0171-7","article-title":"Investigation of the safety and feasibility of AAV1\/SERCA2a gene transfer in patients with chronic heart failure supported with a left ventricular assist device \u2013 the SERCA-LVAD TRIAL","volume":"27","author":"Lyon","year":"2020","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2025.101504_bib8","doi-asserted-by":"crossref","first-page":"839","DOI":"10.1016\/j.neuron.2019.02.017","article-title":"Therapeutic AAV Gene Transfer to the Nervous System: A Clinical Reality","volume":"101","author":"Hudry","year":"2019","journal-title":"Neuron (Camb., Mass.)"},{"key":"10.1016\/j.omtm.2025.101504_bib9","doi-asserted-by":"crossref","first-page":"458","DOI":"10.1038\/mt.2015.151","article-title":"Genome engineering using Adeno-associated virus: Basic and clinical research applications","volume":"24","author":"Gaj","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2025.101504_bib10","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. Engl. J. Med."},{"key":"10.1016\/j.omtm.2025.101504_bib11","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0225582","article-title":"Clade F AAVHSCs cross the blood brain barrier and transduce the central nervous system in addition to peripheral tissues following intravenous administration in nonhuman primates","volume":"14","author":"Ellsworth","year":"2019","journal-title":"PLoS One"},{"key":"10.1016\/j.omtm.2025.101504_bib12","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.omtm.2025.101504_bib13","doi-asserted-by":"crossref","first-page":"358","DOI":"10.1038\/s41573-019-0012-9","article-title":"Adeno-associated virus vector as a platform for gene therapy delivery","volume":"18","author":"Wang","year":"2019","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.omtm.2025.101504_bib14","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.omtm.2025.101504_bib15","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.omtm.2025.101504_bib16","doi-asserted-by":"crossref","DOI":"10.3390\/biomedicines11082273","article-title":"Deletion of Gtf2i via Systemic Administration of AAV-PHP.eB Virus Increases Social Behavior in a Mouse Model of a Neurodevelopmental Disorder","volume":"11","author":"Ophir","year":"2023","journal-title":"Biomedicines"},{"key":"10.1016\/j.omtm.2025.101504_bib17","doi-asserted-by":"crossref","first-page":"236","DOI":"10.1016\/j.omtm.2022.03.016","article-title":"AAV-PHP.eB transduces both the inner and outer retina with high efficacy in mice","volume":"25","author":"Palfi","year":"2022","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2025.101504_bib18","doi-asserted-by":"crossref","first-page":"447","DOI":"10.1016\/j.omtm.2020.10.011","article-title":"CNS Transduction Benefits of AAV-PHP.eB over AAV9 Are Dependent on Administration Route and Mouse Strain","volume":"19","author":"Mathiesen","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2025.101504_bib19","article-title":"AAV Targeting of Glial Cell Types in the Central and Peripheral Nervous System and Relevance to Human Gene Therapy","volume":"13","author":"O\u2019Carroll","year":"2020","journal-title":"Front. Mol. Neurosci."},{"key":"10.1016\/j.omtm.2025.101504_bib20","doi-asserted-by":"crossref","first-page":"198","DOI":"10.1038\/s41434-019-0075-6","article-title":"Astrocyte-selective AAV gene therapy through the endogenous GFAP promoter results in robust transduction in the rat spinal cord following injury","volume":"26","author":"Griffin","year":"2019","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2025.101504_bib21","doi-asserted-by":"crossref","first-page":"694","DOI":"10.1016\/j.expneurol.2013.03.010","article-title":"Sox10-MCS5 enhancer dynamically tracks human oligodendrocyte progenitor fate","volume":"247","author":"Pol","year":"2013","journal-title":"Exp. Neurol."},{"key":"10.1016\/j.omtm.2025.101504_bib22","doi-asserted-by":"crossref","DOI":"10.1016\/j.jneumeth.2022.109530","article-title":"Characterization of the GfaABC1D promoter to selectively target astrocytes in the rhesus macaque brain","volume":"372","author":"Heffernan","year":"2022","journal-title":"J. Neurosci. Methods"},{"key":"10.1016\/j.omtm.2025.101504_bib23","first-page":"481","article-title":"GFAP promoter elements required for region-specific and astrocyte-specific expression","volume":"56","author":"Lee","year":"2008","journal-title":"GLIA (New York, N. Y.)"},{"key":"10.1016\/j.omtm.2025.101504_bib24","doi-asserted-by":"crossref","first-page":"1114","DOI":"10.1007\/s11307-010-0440-8","article-title":"Temporal variations of skin pigmentation in C57Bl\/6 mice affect optical bioluminescence quantitation","volume":"13","author":"Curtis","year":"2011","journal-title":"Mol. Imaging Biol."},{"key":"10.1016\/j.omtm.2025.101504_bib25","first-page":"2812","article-title":"Evidence for glutamine synthetase function in mouse spinal cord oligodendrocytes","volume":"69","author":"Ben Haim","year":"2021","journal-title":"GLIA (New York, N. Y.)"},{"key":"10.1016\/j.omtm.2025.101504_bib26","doi-asserted-by":"crossref","first-page":"173","DOI":"10.1016\/0165-5728(90)90088-5","article-title":"Glutamine synthetase in the central nervous system is not confined to astrocytes","volume":"26","author":"Cammer","year":"1990","journal-title":"J. Neuroimmunol."},{"key":"10.1016\/j.omtm.2025.101504_bib27","first-page":"335","article-title":"Glutamine synthetase immunoreactivity is present in oligodendroglia of various regions of the central nervous system","volume":"3","author":"D\u2019Amelio","year":"1990","journal-title":"GLIA (New York, N. Y.)"},{"key":"10.1016\/j.omtm.2025.101504_bib28","doi-asserted-by":"crossref","first-page":"2262","DOI":"10.1016\/j.celrep.2019.04.094","article-title":"Oligodendrocytes Support Neuronal Glutamatergic Transmission via Expression of Glutamine Synthetase","volume":"27","author":"Xin","year":"2019","journal-title":"Cell Rep."},{"key":"10.1016\/j.omtm.2025.101504_bib29","first-page":"99","article-title":"[Neurons in the corpus callosum of rats: expression of Cav2.2 and their connection]","volume":"33","author":"Zhang","year":"2008","journal-title":"Zhong Nan Da Xue Xue Bao Yi Xue Ban"},{"key":"10.1016\/j.omtm.2025.101504_bib30","doi-asserted-by":"crossref","DOI":"10.1186\/s13041-021-00747-0","article-title":"Region-specific distribution of Olig2-expressing astrocytes in adult mouse brain and spinal cord","volume":"14","author":"Wang","year":"2021","journal-title":"Mol. Brain"},{"key":"10.1016\/j.omtm.2025.101504_bib31","doi-asserted-by":"crossref","first-page":"331","DOI":"10.1016\/S0896-6273(00)80898-3","article-title":"Identification of a Novel Family of Oligodendrocyte Lineage-Specific Basic Helix\u2013Loop\u2013Helix Transcription Factors","volume":"25","author":"Zhou","year":"2000","journal-title":"Neuron (Camb., Mass.)"},{"key":"10.1016\/j.omtm.2025.101504_bib32","doi-asserted-by":"crossref","first-page":"317","DOI":"10.1016\/S0896-6273(00)80897-1","article-title":"Sonic hedgehog-regulated oligodendrocyte lineage genes encoding bHLH proteins in the mammalian central nervous system","volume":"25","author":"Lu","year":"2000","journal-title":"Neuron (Camb., Mass.)"},{"key":"10.1016\/j.omtm.2025.101504_bib33","doi-asserted-by":"crossref","first-page":"13","DOI":"10.3389\/fnmol.2016.00013","article-title":"Recombinant human myelin-associated glycoprotein promoter drives selective AAV-mediated transgene expression in oligodendrocytes","volume":"9","author":"von Jonquieres","year":"2016","journal-title":"Front. Mol. Neurosci."},{"key":"10.1016\/j.omtm.2025.101504_bib34","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0065646","article-title":"Glial Promoter Selectivity following AAV-Delivery to the Immature Brain","volume":"8","author":"von Jonquieres","year":"2013","journal-title":"PLoS One"},{"key":"10.1016\/j.omtm.2025.101504_bib35","doi-asserted-by":"crossref","first-page":"1637","DOI":"10.1007\/s12035-015-9122-5","article-title":"Novel Insights into NeuN: from Neuronal Marker to Splicing Regulator","volume":"53","author":"Duan","year":"2016","journal-title":"Mol. Neurobiol."},{"key":"10.1016\/j.omtm.2025.101504_bib36","doi-asserted-by":"crossref","first-page":"912","DOI":"10.1016\/j.ymthe.2019.02.013","article-title":"The GPI-Linked Protein LY6A Drives AAV-PHP.B Transport across the Blood-Brain Barrier","volume":"27","author":"Hordeaux","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2025.101504_bib37","doi-asserted-by":"crossref","first-page":"90","DOI":"10.1089\/hum.2019.186","article-title":"Ly6a Differential Expression in Blood-Brain Barrier Is Responsible for Strain Specific Central Nervous System Transduction Profile of AAV-PHP.B","volume":"31","author":"Batista","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2025.101504_bib38","doi-asserted-by":"crossref","first-page":"e0225206","DOI":"10.1371\/journal.pone.0225206","article-title":"Delivering genes across the blood-brain barrier: LY6A, a novel cellular receptor for AAV-PHP.B capsids","volume":"14","author":"Huang","year":"2019","journal-title":"PLoS One"},{"key":"10.1016\/j.omtm.2025.101504_bib39","doi-asserted-by":"crossref","first-page":"180","DOI":"10.1016\/j.jneuroim.2010.08.011","article-title":"Blood\u2013brain barrier disruption and enhanced vascular permeability in the multiple sclerosis model EAE","volume":"229","author":"Bennett","year":"2010","journal-title":"J. Neuroimmunol."},{"key":"10.1016\/j.omtm.2025.101504_bib40","doi-asserted-by":"crossref","DOI":"10.1371\/journal.pone.0118550","article-title":"Analysis of Neurogenesis during Experimental Autoimmune Encephalomyelitis Reveals Pitfalls of Bioluminescence Imaging","volume":"10","author":"Ayzenberg","year":"2015","journal-title":"PLoS One"},{"key":"10.1016\/j.omtm.2025.101504_bib41","doi-asserted-by":"crossref","first-page":"106","DOI":"10.1038\/s41593-021-00969-4","article-title":"AAV capsid variants with brain-wide transgene expression and decreased liver targeting after intravenous delivery in mouse and marmoset","volume":"25","author":"Goertsen","year":"2022","journal-title":"Nat. Neurosci."},{"key":"10.1016\/j.omtm.2025.101504_bib42","doi-asserted-by":"crossref","first-page":"3570","DOI":"10.1016\/j.ymthe.2022.11.001","article-title":"Vector genome loss and epigenetic modifications mediate decline in transgene expression of AAV5 vectors produced in mammalian and insect cells","volume":"30","author":"Handyside","year":"2022","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2025.101504_bib43","doi-asserted-by":"crossref","DOI":"10.1038\/s41467-023-38106-3","article-title":"The AAV capsid can influence the epigenetic marking of rAAV delivered episomal genomes in a species dependent manner","volume":"14","author":"Gonzalez-Sandoval","year":"2023","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2025.101504_bib44","doi-asserted-by":"crossref","first-page":"278","DOI":"10.1038\/s41586-018-0750-6","article-title":"NP220 mediates silencing of unintegrated retroviral DNA","volume":"564","author":"Zhu","year":"2018","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2025.101504_bib45","doi-asserted-by":"crossref","DOI":"10.4238\/gmr.15038560","article-title":"Promoter methylation and histone modifications affect the expression of the exogenous DsRed gene in transgenic goats","volume":"15","author":"Nuo","year":"2016","journal-title":"Genet. Mol. Res."},{"key":"10.1016\/j.omtm.2025.101504_bib46","doi-asserted-by":"crossref","first-page":"950","DOI":"10.1016\/j.cels.2022.11.005","article-title":"The sound of silence: Transgene silencing in mammalian cell engineering","volume":"13","author":"Cabrera","year":"2022","journal-title":"Cell Syst."},{"key":"10.1016\/j.omtm.2025.101504_bib47","doi-asserted-by":"crossref","DOI":"10.1212\/WNL.0000000000202555","article-title":"Intravenous and Intrathecal Onasemnogene Abeparvovec Gene Therapy in Symptomatic and Presymptomatic Spinal Muscular Atrophy: Long-Term Follow-Up Study (S34.002)","volume":"100","author":"Darras","year":"2023","journal-title":"Neurology"},{"key":"10.1016\/j.omtm.2025.101504_bib48","doi-asserted-by":"crossref","first-page":"S122","DOI":"10.1016\/j.nmd.2020.08.260","article-title":"SMA \u2013 THERAPY: P.261 Long-term follow-up of onasemnogene abeparvovec gene therapy in spinal muscular atrophy type 1 (SMA1)","volume":"30","author":"Mendell","year":"2020","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.omtm.2025.101504_bib49","doi-asserted-by":"crossref","DOI":"10.3389\/fneur.2021.685802","article-title":"Toward Development of Neuron Specific Transduction After Systemic Delivery of Viral Vectors","volume":"12","author":"Finneran","year":"2021","journal-title":"Front. Neurol."},{"key":"10.1016\/j.omtm.2025.101504_bib50","doi-asserted-by":"crossref","first-page":"560","DOI":"10.1016\/j.jhep.2020.11.001","article-title":"Hepatotoxicity following administration of onasemnogene abeparvovec (AVXS-101) for the treatment of spinal muscular atrophy","volume":"74","author":"Chand","year":"2021","journal-title":"J. Hepatol."},{"key":"10.1016\/j.omtm.2025.101504_bib51","doi-asserted-by":"crossref","first-page":"806","DOI":"10.1002\/lt.21436","article-title":"Glial fibrillary acidic protein as an early marker of hepatic stellate cell activation in chronic and posttransplant recurrent hepatitis C","volume":"14","author":"Carotti","year":"2008","journal-title":"Liver Transpl."},{"key":"10.1016\/j.omtm.2025.101504_bib52","doi-asserted-by":"crossref","DOI":"10.1016\/j.lfs.2024.122547","article-title":"The evidence-based multifaceted roles of hepatic stellate cells in liver diseases: A concise review","volume":"344","author":"Kim","year":"2024","journal-title":"Life Sci."},{"key":"10.1016\/j.omtm.2025.101504_bib53","doi-asserted-by":"crossref","first-page":"376","DOI":"10.1016\/S1016-8478(23)17595-9","article-title":"Glial Fibrillary Acidic Protein Splice Variants in Hepatic Stellate Cells - Expression and Regulation","volume":"25","author":"Lim","year":"2008","journal-title":"Mol. Cells"},{"key":"10.1016\/j.omtm.2025.101504_bib54","doi-asserted-by":"crossref","first-page":"56","DOI":"10.1038\/s41434-020-0169-1","article-title":"Optimization of adeno-associated viral vector-mediated transduction of the corticospinal tract: comparison of four promoters","volume":"28","author":"Nieuwenhuis","year":"2021","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2025.101504_bib55","doi-asserted-by":"crossref","first-page":"504","DOI":"10.1016\/j.omtm.2023.05.013","article-title":"Strong ubiquitous micro-promoters for recombinant adeno-associated viral vectors","volume":"29","author":"Chai","year":"2023","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2025.101504_bib56","doi-asserted-by":"crossref","first-page":"2","DOI":"10.3389\/fnana.2017.00002","article-title":"Adeno-associated viral vectors serotype 8 for cell-specific delivery of therapeutic genes in the central nervous system","volume":"11","author":"Pignataro","year":"2017","journal-title":"Front. Neuroanat."},{"key":"10.1016\/j.omtm.2025.101504_bib57","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1113\/expphysiol.2004.028159","article-title":"Promoters and serotypes: targeting of adeno-associated virus vectors for gene transfer in the rat central nervous system in vitro and in vivo","volume":"90","author":"Shevtsova","year":"2005","journal-title":"Exp. Physiol."},{"key":"10.1016\/j.omtm.2025.101504_bib58","doi-asserted-by":"crossref","first-page":"4080","DOI":"10.1523\/JNEUROSCI.3386-15.2016","article-title":"Re-Establishment of Cortical Motor Output Maps and Spontaneous Functional Recovery via Spared Dorsolaterally Projecting Corticospinal Neurons after Dorsal Column Spinal Cord Injury in Adult Mice","volume":"36","author":"Hilton","year":"2016","journal-title":"J. Neurosci."},{"key":"10.1016\/j.omtm.2025.101504_bib59","doi-asserted-by":"crossref","first-page":"872","DOI":"10.1038\/sj.gt.3302924","article-title":"Efficient gene transduction of neurons by lentivirus with enhanced neuron-specific promoters","volume":"14","author":"Hioki","year":"2007","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2025.101504_bib60","doi-asserted-by":"crossref","first-page":"3431","DOI":"10.1073\/pnas.88.8.3431","article-title":"Characterization of tissue-specific transcription by the human synapsin I gene promoter","volume":"88","author":"Thiel","year":"1991","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2025.101504_bib61","doi-asserted-by":"crossref","first-page":"73","DOI":"10.1016\/j.neulet.2014.05.044","article-title":"Widespread neuron-specific transgene expression in brain and spinal cord following synapsin promoter-driven AAV9 neonatal intracerebroventricular injection","volume":"576","author":"McLean","year":"2014","journal-title":"Neurosci. Lett."},{"key":"10.1016\/j.omtm.2025.101504_bib62","doi-asserted-by":"crossref","first-page":"337","DOI":"10.1038\/sj.gt.3301905","article-title":"Human synapsin 1 gene promoter confers highly neuron-specific long-term transgene expression from an adenoviral vector in the adult rat brain depending on the transduced area","volume":"10","author":"K\u00fcgler","year":"2003","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2025.101504_bib63","doi-asserted-by":"crossref","first-page":"47","DOI":"10.3389\/fncir.2017.00047","article-title":"Focal stroke in the developing rat motor cortex induces age- and experience-dependent maladaptive plasticity of corticospinal system","volume":"11","author":"Gennaro","year":"2017","journal-title":"Front. Neural Circuits"},{"key":"10.1016\/j.omtm.2025.101504_bib64","doi-asserted-by":"crossref","first-page":"144","DOI":"10.1016\/j.neures.2014.09.002","article-title":"Comparative analyses of adeno-associated viral vector serotypes 1, 2, 5, 8 and 9 in marmoset, mouse and macaque cerebral cortex","volume":"93","author":"Watakabe","year":"2015","journal-title":"Neurosci. Res."},{"key":"10.1016\/j.omtm.2025.101504_bib65","doi-asserted-by":"crossref","first-page":"354","DOI":"10.1002\/dneu.22459","article-title":"Application of viral vectors to the study of neural connectivities and neural circuits in the marmoset brain","volume":"77","author":"Watakabe","year":"2017","journal-title":"Dev. Neurobiol."},{"key":"10.1016\/j.omtm.2025.101504_bib66","doi-asserted-by":"crossref","first-page":"3453","DOI":"10.1523\/JNEUROSCI.3693-15.2016","article-title":"Dysfunctional Calcium and Glutamate Signaling in Striatal Astrocytes from Huntington\u2019s Disease Model Mice","volume":"36","author":"Jiang","year":"2016","journal-title":"J. Neurosci."},{"key":"10.1016\/j.omtm.2025.101504_bib67","doi-asserted-by":"crossref","first-page":"1280","DOI":"10.1016\/j.cell.2019.03.019","article-title":"Hyperactivity with Disrupted Attention by Activation of an Astrocyte Synaptogenic Cue","volume":"177","author":"Nagai","year":"2019","journal-title":"Cell"},{"key":"10.1016\/j.omtm.2025.101504_bib68","doi-asserted-by":"crossref","first-page":"1170","DOI":"10.1016\/j.neuron.2018.08.015","article-title":"Reducing astrocyte calcium signaling in vivo alters striatal microcircuits and causes repetitive behavior","volume":"99","author":"Yu","year":"2018","journal-title":"Neuron (Camb., Mass.)"},{"key":"10.1016\/j.omtm.2025.101504_bib69","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1016\/j.neuron.2018.03.003","article-title":"An optical neuron-astrocyte proximity assay at synaptic distance scales","volume":"98","author":"Octeau","year":"2018","journal-title":"Neuron (Camb., Mass.)"},{"key":"10.1016\/j.omtm.2025.101504_bib70","doi-asserted-by":"crossref","first-page":"992","DOI":"10.1016\/j.neuroscience.2010.08.034","article-title":"Specific Disruption of Astrocytic Ca2+ Signaling Pathway in vivo by Adeno-Associated Viral Transduction","volume":"170","author":"Xie","year":"2010","journal-title":"Neuroscience"},{"key":"10.1016\/j.omtm.2025.101504_bib71","doi-asserted-by":"crossref","first-page":"E2675","DOI":"10.1073\/pnas.1520759113","article-title":"Astrocytes regulate cortical state switching in vivo","volume":"113","author":"Poskanzer","year":"2016","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2025.101504_bib72","doi-asserted-by":"crossref","first-page":"1030","DOI":"10.1523\/JNEUROSCI.14-03-01030.1994","article-title":"GFAP promoter directs astrocyte-specific expression in transgenic mice","volume":"14","author":"Brenner","year":"1994","journal-title":"J. Neurosci."},{"key":"10.1016\/j.omtm.2025.101504_bib73","doi-asserted-by":"crossref","first-page":"1085","DOI":"10.1111\/j.1471-4159.1988.tb03072.x","article-title":"Glial Fibrillary Acidic Protein Increases in the Spinal Cord of Lewis Rats with Acute Experimental Autoimmune Encephalomyelitis","volume":"51","author":"Aquino","year":"1988","journal-title":"J. Neurochem."},{"key":"10.1016\/j.omtm.2025.101504_bib74","doi-asserted-by":"crossref","first-page":"1398","DOI":"10.1111\/j.1471-4159.1990.tb01975.x","article-title":"Expression of glial fibrillary acidic protein and neurofilament mRNA in gliosis induced by experimental autoimmune encephalomyelitis","volume":"54","author":"Aquino","year":"1990","journal-title":"J. Neurochem."},{"key":"10.1016\/j.omtm.2025.101504_bib75","doi-asserted-by":"crossref","first-page":"1280","DOI":"10.1093\/nar\/gkq951","article-title":"Olig2 regulates Sox10 expression in oligodendrocyte precursors through an evolutionary conserved distal enhancer","volume":"39","author":"K\u00fcspert","year":"2011","journal-title":"Nucleic Acids Res."},{"key":"10.1016\/j.omtm.2025.101504_bib76","doi-asserted-by":"crossref","first-page":"476","DOI":"10.1016\/S1044-7431(03)00210-0","article-title":"NG2-expressing glial progenitor cells: an abundant and widespread population of cycling cells in the adult rat CNS","volume":"24","author":"Dawson","year":"2003","journal-title":"Mol. Cell. Neurosci."},{"key":"10.1016\/j.omtm.2025.101504_bib77","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/j.bbi.2016.01.005","article-title":"Adult oligodendrocyte progenitor cells \u2013 Multifaceted regulators of the CNS in health and disease","volume":"57","author":"Fernandez-Castaneda","year":"2016","journal-title":"Brain Behav. Immun."},{"key":"10.1016\/j.omtm.2025.101504_bib78","doi-asserted-by":"crossref","DOI":"10.3389\/fbioe.2021.679483","article-title":"PhP.B Enhanced Adeno-Associated Virus Mediated-Expression Following Systemic Delivery or Direct Brain Administration","volume":"9","author":"Pietersz","year":"2021","journal-title":"Front. Bioeng. Biotechnol."},{"key":"10.1016\/j.omtm.2025.101504_bib79","doi-asserted-by":"crossref","first-page":"382","DOI":"10.1089\/hum.2011.200","article-title":"Adeno-associated virus serotype 9 transduction in the central nervous system of nonhuman primates","volume":"23","author":"Samaranch","year":"2012","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2025.101504_bib80","doi-asserted-by":"crossref","first-page":"807","DOI":"10.1038\/gt.2016.62","article-title":"Characterization of a novel adeno-associated viral vector with preferential oligodendrocyte tropism","volume":"23","author":"Powell","year":"2016","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2025.101504_bib81","doi-asserted-by":"crossref","first-page":"744","DOI":"10.1002\/jnr.20776","article-title":"Increased glia-specific transgene expression with glial fibrillary acidic protein promoters containing multiple enhancer elements","volume":"83","author":"De Leeuw","year":"2006","journal-title":"J. Neurosci. Res."},{"key":"10.1016\/j.omtm.2025.101504_bib82","first-page":"89","article-title":"Differential transgene expression in brain cells in vivo and in vitro from AAV-2 vectors with small transcriptional control units","volume":"311","author":"K\u00fcgler","year":"2003","journal-title":"Virology (New York, N. Y.)"},{"key":"10.1016\/j.omtm.2025.101504_bib83","first-page":"1","article-title":"Comparison of AAV Serotypes for Gene Delivery to Dopaminergic Neurons in the Substantia Nigra","volume":"2","author":"Korecka","year":"2011","journal-title":"Viral Gene Therapy"},{"key":"10.1016\/j.omtm.2025.101504_bib84","article-title":"Author Spotlight: Advancing Gene Therapy with High-Yield AAV Vectors Through HEK293 Suspension Cell Cultivation","volume":"2024","author":"Pietersz","year":"2024","journal-title":"J. Vis. Exp."}],"updated-by":[{"DOI":"10.1016\/j.omtm.2025.101543","type":"erratum","label":"Erratum","source":"publisher","updated":{"date-parts":[[2025,9,11]],"date-time":"2025-09-11T00:00:00Z","timestamp":1757548800000}}],"container-title":["Molecular Therapy Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050125000993?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050125000993?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,8]],"date-time":"2025-11-08T15:19:16Z","timestamp":1762615156000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050125000993"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,9]]},"references-count":84,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2025,9]]}},"alternative-id":["S2329050125000993"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2025.101504","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2025,9]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Developing a minimally invasive gene therapy for multiple sclerosis","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2025.101504","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 Inc. on behalf of The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}],"article-number":"101504"}}