{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,24]],"date-time":"2026-06-24T08:38:29Z","timestamp":1782290309944,"version":"3.54.5"},"reference-count":47,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2021,3,1]],"date-time":"2021-03-01T00:00:00Z","timestamp":1614556800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2021,3,1]],"date-time":"2021-03-01T00:00:00Z","timestamp":1614556800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2020,12,31]],"date-time":"2020-12-31T00:00:00Z","timestamp":1609372800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100007338","name":"Foundation for Barnes-Jewish Hospital","doi-asserted-by":"publisher","award":["4642"],"award-info":[{"award-number":["4642"]}],"id":[{"id":"10.13039\/100007338","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100007338","name":"Foundation for Barnes-Jewish Hospital","doi-asserted-by":"publisher","award":["3770"],"award-info":[{"award-number":["3770"]}],"id":[{"id":"10.13039\/100007338","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["P20GM103620"],"award-info":[{"award-number":["P20GM103620"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["R01NS082283"],"award-info":[{"award-number":["R01NS082283"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"publisher","award":["P20GM103548"],"award-info":[{"award-number":["P20GM103548"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100011912","name":"Washington University School of Medicine in St. Louis","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100011912","id-type":"DOI","asserted-by":"publisher"}]}],"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":[[2021,3]]},"DOI":"10.1016\/j.omtm.2020.12.014","type":"journal-article","created":{"date-parts":[[2021,1,10]],"date-time":"2021-01-10T05:44:34Z","timestamp":1610257474000},"page":"497-507","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":19,"special_numbering":"C","title":["Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice"],"prefix":"10.1016","volume":"20","author":[{"given":"Katherine A.","family":"White","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hemanth R.","family":"Nelvagal","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Timothy A.","family":"Poole","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bin","family":"Lu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tyler B.","family":"Johnson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Samantha","family":"Davis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa A.","family":"Pratt","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jon","family":"Brudvig","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ana B.","family":"Assis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shibi","family":"Likhite","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathrin","family":"Meyer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian K.","family":"Kaspar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jonathan D.","family":"Cooper","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Shaomei","family":"Wang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-2504-4942","authenticated-orcid":false,"given":"Jill M.","family":"Weimer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2020.12.014_bib1","doi-asserted-by":"crossref","first-page":"80","DOI":"10.1016\/S0387-7604(88)80075-5","article-title":"Neuronal ceroid-lipofuscinoses in childhood","volume":"10","author":"Santavuori","year":"1988","journal-title":"Brain Dev."},{"key":"10.1016\/j.omtm.2020.12.014_bib2","doi-asserted-by":"crossref","first-page":"255","DOI":"10.1016\/j.ejpn.2006.08.009","article-title":"Neuronal ceroid lipofuscinoses (NCL)","volume":"10","author":"Mole","year":"2006","journal-title":"Eur. J. Paediatr. Neurol."},{"issue":"10 Pt B","key":"10.1016\/j.omtm.2020.12.014_bib3","doi-asserted-by":"crossref","first-page":"2237","DOI":"10.1016\/j.bbadis.2015.05.011","article-title":"Genetics of the neuronal ceroid lipofuscinoses (Batten disease)","volume":"1852","author":"Mole","year":"2015","journal-title":"Biochim. Biophys. Acta"},{"issue":"10 Pt B","key":"10.1016\/j.omtm.2020.12.014_bib4","doi-asserted-by":"crossref","first-page":"2242","DOI":"10.1016\/j.bbadis.2015.04.027","article-title":"Cell biology of the NCL proteins: What they do and don\u2019t do","volume":"1852","author":"C\u00e1rcel-Trullols","year":"2015","journal-title":"Biochim. Biophys. Acta"},{"key":"10.1016\/j.omtm.2020.12.014_bib5","doi-asserted-by":"crossref","first-page":"161","DOI":"10.1038\/s41582-019-0138-8","article-title":"Therapeutic landscape for Batten disease: current treatments and future prospects","volume":"15","author":"Johnson","year":"2019","journal-title":"Nat. Rev. Neurol."},{"key":"10.1016\/j.omtm.2020.12.014_bib6","doi-asserted-by":"crossref","first-page":"348","DOI":"10.1177\/0883073819895694","article-title":"Review of Cerliponase Alfa: Recombinant Human Enzyme Replacement Therapy for Late-Infantile Neuronal Ceroid Lipofuscinosis Type 2","volume":"35","author":"Lewis","year":"2020","journal-title":"J.\u00a0Child Neurol."},{"key":"10.1016\/j.omtm.2020.12.014_bib7","doi-asserted-by":"crossref","first-page":"1898","DOI":"10.1056\/NEJMoa1712649","article-title":"Study of Intraventricular Cerliponase Alfa for CLN2 Disease","volume":"378","author":"Schulz","year":"2018","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2020.12.014_bib8","doi-asserted-by":"crossref","first-page":"1836","DOI":"10.1016\/j.ymthe.2019.06.015","article-title":"Gene Therapy Corrects Brain and Behavioral Pathologies in CLN6-Batten Disease","volume":"27","author":"Cain","year":"2019","journal-title":"Mol. Ther"},{"key":"10.1016\/j.omtm.2020.12.014_bib9","doi-asserted-by":"crossref","first-page":"3867","DOI":"10.1093\/hmg\/ddz210","article-title":"Neonatal brain-directed gene therapy rescues a mouse model of neurodegenerative CLN6 Batten disease","volume":"28","author":"Kleine Holthaus","year":"2019","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2020.12.014_bib10","series-title":"Positive Interim Clinical Data from Ongoing Phase 1\/2 Study in CLN6 Batten Disease","year":"2019"},{"key":"10.1016\/j.omtm.2020.12.014_bib11","doi-asserted-by":"crossref","first-page":"42","DOI":"10.3390\/diseases6020042","article-title":"Viral Vectors in Gene Therapy","volume":"6","author":"Lundstrom","year":"2018","journal-title":"Diseases"},{"key":"10.1016\/j.omtm.2020.12.014_bib12","doi-asserted-by":"crossref","first-page":"82","DOI":"10.1016\/j.neuropharm.2012.03.004","article-title":"Adeno-associated virus (AAV) gene therapy for neurological disease","volume":"69","author":"Weinberg","year":"2013","journal-title":"Neuropharmacology"},{"key":"10.1016\/j.omtm.2020.12.014_bib13","doi-asserted-by":"crossref","first-page":"2177","DOI":"10.1172\/JCI120429","article-title":"Gene therapy and genome surgery in the retina","volume":"128","author":"DiCarlo","year":"2018","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2020.12.014_bib14","doi-asserted-by":"crossref","first-page":"e0188830","DOI":"10.1371\/journal.pone.0188830","article-title":"Cellular selectivity of AAV serotypes for gene delivery in neurons and astrocytes by neonatal intracerebroventricular injection","volume":"12","author":"Hammond","year":"2017","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2020.12.014_bib15","doi-asserted-by":"crossref","first-page":"5399","DOI":"10.1167\/iovs.17-22978","article-title":"Concepts and Strategies in Retinal Gene Therapy","volume":"58","author":"Aguirre","year":"2017","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.omtm.2020.12.014_bib16","doi-asserted-by":"crossref","first-page":"9669","DOI":"10.1523\/JNEUROSCI.1635-16.2016","article-title":"Self-Complementary AAV9 Gene Delivery Partially Corrects Pathology Associated with Juvenile Neuronal Ceroid Lipofuscinosis (CLN3)","volume":"36","author":"Bosch","year":"2016","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.omtm.2020.12.014_bib17","doi-asserted-by":"crossref","first-page":"477","DOI":"10.1038\/mt.2014.210","article-title":"Improving single injection CSF delivery of AAV9-mediated gene therapy for SMA: a dose-response study in mice and nonhuman primates","volume":"23","author":"Meyer","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.12.014_bib18","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.omtm.2020.12.014_bib19","doi-asserted-by":"crossref","first-page":"263","DOI":"10.1146\/annurev-vision-091517-034122","article-title":"Thalamocortical Circuits and Functional Architecture","volume":"4","author":"Kremkow","year":"2018","journal-title":"Annu. Rev. Vis. Sci."},{"key":"10.1016\/j.omtm.2020.12.014_bib20","doi-asserted-by":"crossref","first-page":"284","DOI":"10.1016\/j.nbd.2005.11.008","article-title":"Visual deficits in a mouse model of Batten disease are the result of optic nerve degeneration and loss of dorsal lateral geniculate thalamic neurons","volume":"22","author":"Weimer","year":"2006","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.omtm.2020.12.014_bib21","doi-asserted-by":"crossref","first-page":"10","DOI":"10.3389\/fncir.2018.00010","article-title":"The Mouse Superior Colliculus: An Emerging Model for Studying Circuit Formation and Function","volume":"12","author":"Ito","year":"2018","journal-title":"Front. Neural Circuits"},{"key":"10.1016\/j.omtm.2020.12.014_bib22","doi-asserted-by":"crossref","first-page":"e54146","DOI":"10.1371\/journal.pone.0054146","article-title":"Tropisms of AAV for subretinal delivery to the neonatal mouse retina and its application for in\u00a0vivo rescue of developmental photoreceptor disorders","volume":"8","author":"Watanabe","year":"2013","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2020.12.014_bib23","doi-asserted-by":"crossref","first-page":"677","DOI":"10.1017\/S0952523805225166","article-title":"Independent visual threshold measurements in the two eyes of freely moving rats and mice using a virtual-reality optokinetic system","volume":"22","author":"Douglas","year":"2005","journal-title":"Vis. Neurosci."},{"key":"10.1016\/j.omtm.2020.12.014_bib24","doi-asserted-by":"crossref","first-page":"150","DOI":"10.1016\/j.nbd.2006.09.001","article-title":"Successive neuron loss in the thalamus and cortex in a mouse model of infantile neuronal ceroid lipofuscinosis","volume":"25","author":"Kielar","year":"2007","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.omtm.2020.12.014_bib25","doi-asserted-by":"crossref","first-page":"823","DOI":"10.1016\/j.nbd.2005.05.018","article-title":"Thalamocortical neuron loss and localized astrocytosis in the Cln3Deltaex7\/8 knock-in mouse model of Batten disease","volume":"20","author":"Pontikis","year":"2005","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.omtm.2020.12.014_bib26","doi-asserted-by":"crossref","first-page":"e78694","DOI":"10.1371\/journal.pone.0078694","article-title":"A murine model of variant late infantile ceroid lipofuscinosis recapitulates behavioral and pathological phenotypes of human disease","volume":"8","author":"Morgan","year":"2013","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2020.12.014_bib27","doi-asserted-by":"crossref","first-page":"4784","DOI":"10.1167\/iovs.17-22290","article-title":"Evidence for Cerebrospinal Fluid Entry Into the Optic Nerve via a Glymphatic Pathway","volume":"58","author":"Mathieu","year":"2017","journal-title":"Invest. Ophthalmol. Vis. Sci."},{"key":"10.1016\/j.omtm.2020.12.014_bib28","doi-asserted-by":"crossref","first-page":"1343","DOI":"10.1016\/j.ymthe.2018.02.027","article-title":"Prevention of Photoreceptor Cell Loss in a Cln6nclf Mouse Model of Batten Disease Requires CLN6 Gene Transfer to Bipolar Cells","volume":"26","author":"Kleine Holthaus","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.12.014_bib29","doi-asserted-by":"crossref","first-page":"413","DOI":"10.1016\/j.ymthe.2005.04.018","article-title":"AAV2-mediated ocular gene therapy for infantile neuronal ceroid lipofuscinosis","volume":"12","author":"Griffey","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.12.014_bib30","doi-asserted-by":"crossref","first-page":"108135","DOI":"10.1016\/j.exer.2020.108135","article-title":"Intravitreal enzyme replacement inhibits progression of retinal degeneration in canine CLN2 neuronal ceroid lipofuscinosis","volume":"198","author":"Whiting","year":"2020","journal-title":"Exp. Eye Res."},{"key":"10.1016\/j.omtm.2020.12.014_bib31","article-title":"Experimental therapeutic approaches for the treatment of retinal dystrophy in neuronal ceroid lipofuscinosis","author":"Bartsch","year":"2020","journal-title":"Ophthalmologe"},{"key":"10.1016\/j.omtm.2020.12.014_bib32","doi-asserted-by":"crossref","first-page":"709","DOI":"10.1089\/hum.2020.038","article-title":"Gene Therapy Targeting the Inner Retina Rescues the Retinal Phenotype in a Mouse Model of CLN3 Batten Disease","volume":"31","author":"Kleine Holthaus","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2020.12.014_bib33","doi-asserted-by":"crossref","first-page":"3415","DOI":"10.1038\/s41467-019-11321-7","article-title":"Standard screening methods underreport AAV-mediated transduction and gene editing","volume":"10","author":"Lang","year":"2019","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2020.12.014_bib34","doi-asserted-by":"crossref","first-page":"13104","DOI":"10.1073\/pnas.0702778104","article-title":"Existence of transient functional double-stranded DNA intermediates during recombinant AAV transduction","volume":"104","author":"Wang","year":"2007","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2020.12.014_bib35","doi-asserted-by":"crossref","first-page":"1235","DOI":"10.1089\/hum.2018.144","article-title":"Dosage Thresholds and Influence of Transgene Cassette in Adeno-Associated Virus-Related Toxicity","volume":"29","author":"Khabou","year":"2018","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2020.12.014_bib36","doi-asserted-by":"crossref","first-page":"2648","DOI":"10.1016\/j.ymthe.2017.08.018","article-title":"AAV8 Can Induce Innate and Adaptive Immune Response in the Primate Eye","volume":"25","author":"Reichel","year":"2017","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2020.12.014_bib37","doi-asserted-by":"crossref","first-page":"5785","DOI":"10.1073\/pnas.1821000116","article-title":"AAV cis-regulatory sequences are correlated with ocular toxicity","volume":"116","author":"Xiong","year":"2019","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.omtm.2020.12.014_bib38","doi-asserted-by":"crossref","first-page":"313ra180","DOI":"10.1126\/scitranslmed.aac6191","article-title":"AAV gene transfer delays disease onset in a TPP1-deficient canine model of the late infantile form of Batten disease","volume":"7","author":"Katz","year":"2015","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2020.12.014_bib39","doi-asserted-by":"crossref","first-page":"1155","DOI":"10.1089\/hum.2020.196","article-title":"Adeno-Associated Virus Capsid-Promoter Interactions in the Brain Translate from Rat to the Nonhuman Primate","volume":"31","author":"Bohlen","year":"2020","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2020.12.014_bib40","doi-asserted-by":"crossref","first-page":"55","DOI":"10.1016\/j.omtm.2018.11.008","article-title":"Adeno-Associated Viral Vector 2 and 9 Transduction Is Enhanced in Streptozotocin-Induced Diabetic Mouse Retina","volume":"13","author":"Lee","year":"2018","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2020.12.014_bib41","doi-asserted-by":"crossref","first-page":"520","DOI":"10.1038\/gt.2016.24","article-title":"Axonal transport of AAV9 in nonhuman primate brain","volume":"23","author":"Green","year":"2016","journal-title":"Gene Ther."},{"key":"10.1016\/j.omtm.2020.12.014_bib42","doi-asserted-by":"crossref","first-page":"705","DOI":"10.1089\/hum.2013.189","article-title":"Adeno-associated virus serotypes 1, 8, and 9 share conserved mechanisms for anterograde and retrograde axonal transport","volume":"25","author":"Castle","year":"2014","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2020.12.014_bib43","doi-asserted-by":"crossref","first-page":"3302","DOI":"10.1523\/JNEUROSCI.23-08-03302.2003","article-title":"Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice","volume":"23","author":"Hennig","year":"2003","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.omtm.2020.12.014_bib44","doi-asserted-by":"crossref","first-page":"eaaz7423","DOI":"10.1126\/scitranslmed.aaz7423","article-title":"Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy","volume":"12","author":"Yu-Wai-Man","year":"2020","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2020.12.014_bib45","doi-asserted-by":"crossref","first-page":"346","DOI":"10.1016\/j.nbd.2004.02.010","article-title":"Regional and cellular neuropathology in the palmitoyl protein thioesterase-1 null mutant mouse model of infantile neuronal ceroid lipofuscinosis","volume":"16","author":"Bible","year":"2004","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.omtm.2020.12.014_bib46","doi-asserted-by":"crossref","first-page":"1083","DOI":"10.1093\/brain\/awt020","article-title":"Immune cells perturb axons and impair neuronal survival in a mouse model of infantile neuronal ceroid lipofuscinosis","volume":"136","author":"Groh","year":"2013","journal-title":"Brain"},{"key":"10.1016\/j.omtm.2020.12.014_bib47","doi-asserted-by":"crossref","first-page":"482","DOI":"10.1002\/ar.1092310411","article-title":"Unbiased stereological estimation of the total number of neurons in thesubdivisions of the rat hippocampus using the optical fractionator","volume":"231","author":"West","year":"1991","journal-title":"Anat. Rec."}],"container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S232905012030262X?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S232905012030262X?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2026,5,15]],"date-time":"2026-05-15T17:52:38Z","timestamp":1778867558000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S232905012030262X"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2021,3]]},"references-count":47,"alternative-id":["S232905012030262X"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2020.12.014","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2021,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Intracranial delivery of AAV9 gene therapy partially prevents retinal degeneration and visual deficits in CLN6-Batten disease mice","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2020.12.014","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2021 The Authors.","name":"copyright","label":"Copyright"}]}}