{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,16]],"date-time":"2026-02-16T08:22:56Z","timestamp":1771230176454,"version":"3.50.1"},"reference-count":47,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2018,3,1]],"date-time":"2018-03-01T00:00:00Z","timestamp":1519862400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2018,3,1]],"date-time":"2018-03-01T00:00:00Z","timestamp":1519862400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2019,3,7]],"date-time":"2019-03-07T00:00:00Z","timestamp":1551916800000},"content-version":"vor","delay-in-days":371,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100009429","name":"NRSA","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100009429","id-type":"DOI","asserted-by":"crossref"}]},{"name":"NIH","award":["NRSA F30NS090684"],"award-info":[{"award-number":["NRSA F30NS090684"]}]},{"name":"Legacy of Angels Foundation and Research to Prevent Blindness"},{"name":"NIH","award":["R01 NS065808"],"award-info":[{"award-number":["R01 NS065808"]}]},{"DOI":"10.13039\/100014177","name":"Legacy of Angels Foundation","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100014177","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Partners for Krabbe Disease Research"},{"DOI":"10.13039\/501100008731","name":"European Leukodystrophy Association","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100008731","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2018,3]]},"DOI":"10.1016\/j.ymthe.2018.01.009","type":"journal-article","created":{"date-parts":[[2018,1,16]],"date-time":"2018-01-16T21:35:12Z","timestamp":1516138512000},"page":"874-889","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":59,"title":["Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe\u2019s Disease after Global Gene Therapy"],"prefix":"10.1016","volume":"26","author":[{"given":"Michael S.","family":"Marshall","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yazan","family":"Issa","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Benas","family":"Jakubauskas","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Monika","family":"Stoskute","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Vince","family":"Elackattu","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jeffrey N.","family":"Marshall","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Wil","family":"Bogue","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Duc","family":"Nguyen","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Zane","family":"Hauck","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Emily","family":"Rue","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Subha","family":"Karumuthil-Melethil","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Violeta","family":"Zaric","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maarten","family":"Bosland","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Richard B.","family":"van Breemen","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maria I.","family":"Givogri","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Steven J.","family":"Gray","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Stephen J.","family":"Crocker","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Ernesto R.","family":"Bongarzone","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2018.01.009_bib1","first-page":"145","article-title":"Krabbe\u2019s globoid cell leukodystrophy: deficiency of galactocerebroside beta-galactosidase activity","volume":"30","author":"Suzuki","year":"1971","journal-title":"J.\u00a0Neuropathol. Exp. Neurol."},{"key":"10.1016\/j.ymthe.2018.01.009_bib2","doi-asserted-by":"crossref","first-page":"53","DOI":"10.1016\/S0022-2275(20)39839-4","article-title":"Krabbe disease: a galactosylsphingosine (psychosine) lipidosis","volume":"21","author":"Svennerholm","year":"1980","journal-title":"J.\u00a0Lipid Res."},{"key":"10.1016\/j.ymthe.2018.01.009_bib3","doi-asserted-by":"crossref","first-page":"753","DOI":"10.1126\/science.6719111","article-title":"Progressive accumulation of toxic metabolite in a genetic leukodystrophy","volume":"224","author":"Igisu","year":"1984","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2018.01.009_bib4","doi-asserted-by":"crossref","first-page":"307","DOI":"10.1007\/978-1-4684-7844-0_28","article-title":"The specificity of beta-galactosidase in the degradation of gangliosides","volume":"125","author":"Suzuki","year":"1980","journal-title":"Adv. Exp. Med. Biol."},{"key":"10.1016\/j.ymthe.2018.01.009_bib5","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1093\/brain\/39.1-2.74","article-title":"A new familial, infantile form of diffuse brain sclerosis","volume":"39","author":"Krabbe","year":"1916","journal-title":"Brain"},{"key":"10.1016\/j.ymthe.2018.01.009_bib6","doi-asserted-by":"crossref","first-page":"2835","DOI":"10.1002\/ajmg.a.35624","article-title":"Death rates in the U.S. due to Krabbe disease and related leukodystrophy and lysosomal storage diseases","volume":"158A","author":"Barczykowski","year":"2012","journal-title":"Am. J. Med. Genet. A."},{"key":"10.1016\/j.ymthe.2018.01.009_bib7","doi-asserted-by":"crossref","first-page":"2069","DOI":"10.1056\/NEJMoa042604","article-title":"Transplantation of umbilical-cord blood in babies with infantile Krabbe\u2019s disease","volume":"352","author":"Escolar","year":"2005","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.ymthe.2018.01.009_bib8","doi-asserted-by":"crossref","first-page":"1235","DOI":"10.1038\/gim.2016.35","article-title":"Clinical outcomes of children with abnormal newborn screening results for Krabbe disease in New York State","volume":"18","author":"Wasserstein","year":"2016","journal-title":"Genet. Med."},{"key":"10.1016\/j.ymthe.2018.01.009_bib9","doi-asserted-by":"crossref","first-page":"e879","DOI":"10.1542\/peds.2006-0747","article-title":"A staging system for infantile Krabbe disease to predict outcome after unrelated umbilical cord blood transplantation","volume":"118","author":"Escolar","year":"2006","journal-title":"Pediatrics"},{"key":"10.1016\/j.ymthe.2018.01.009_bib10","doi-asserted-by":"crossref","first-page":"1748","DOI":"10.1002\/jnr.22006","article-title":"Combined hematopoietic and lentiviral gene-transfer therapies in newborn Twitcher mice reveal contemporaneous neurodegeneration and demyelination in Krabbe disease","volume":"87","author":"Galbiati","year":"2009","journal-title":"J.\u00a0Neurosci. Res."},{"key":"10.1016\/j.ymthe.2018.01.009_bib11","doi-asserted-by":"crossref","first-page":"6495","DOI":"10.1523\/JNEUROSCI.4199-14.2015","article-title":"Mechanism-based combination treatment dramatically increases therapeutic efficacy in murine globoid cell leukodystrophy","volume":"35","author":"Hawkins-Salsbury","year":"2015","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.ymthe.2018.01.009_bib12","doi-asserted-by":"crossref","first-page":"1681","DOI":"10.1038\/mt.2015.145","article-title":"Long-term Improvements in Lifespan and Pathology in CNS and PNS After BMT Plus One Intravenous Injection of AAVrh10-GALC in Twitcher Mice","volume":"23","author":"Rafi","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2018.01.009_bib13","doi-asserted-by":"crossref","first-page":"1138","DOI":"10.1002\/jnr.23882","article-title":"Intrathecal administration of AAV\/GALC vectors in 10-11-day-old twitcher mice improves survival and is enhanced by bone marrow transplant","volume":"94","author":"Karumuthil-Melethil","year":"2016","journal-title":"J.\u00a0Neurosci. Res."},{"key":"10.1016\/j.ymthe.2018.01.009_bib14","doi-asserted-by":"crossref","first-page":"3372","DOI":"10.1093\/hmg\/ddv086","article-title":"Combined gene\/cell therapies provide long-term and pervasive rescue of multiple pathological symptoms in a murine model of globoid cell leukodystrophy","volume":"24","author":"Ricca","year":"2015","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.ymthe.2018.01.009_bib15","doi-asserted-by":"crossref","first-page":"44","DOI":"10.1038\/sj.mt.6300026","article-title":"Central nervous system-directed AAV2\/5-mediated gene therapy synergizes with bone marrow transplantation in the murine model of globoid-cell leukodystrophy","volume":"15","author":"Lin","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2018.01.009_bib16","doi-asserted-by":"crossref","first-page":"695","DOI":"10.1093\/brain\/103.3.695","article-title":"Hereditary leucodystrophy in the mouse: the new mutant twitcher","volume":"103","author":"Duchen","year":"1980","journal-title":"Brain"},{"key":"10.1016\/j.ymthe.2018.01.009_bib17","doi-asserted-by":"crossref","first-page":"1126","DOI":"10.1002\/jnr.23822","article-title":"Treatment for Krabbe\u2019s disease: Finding the combination","volume":"94","author":"Mikulka","year":"2016","journal-title":"J.\u00a0Neurosci. Res."},{"key":"10.1016\/j.ymthe.2018.01.009_bib18","doi-asserted-by":"crossref","first-page":"169","DOI":"10.1055\/s-0033-1364179","article-title":"Hematopoietic stem cell transplantation in the leukodystrophies: a systematic review of the literature","volume":"45","author":"Musolino","year":"2014","journal-title":"Neuropediatrics"},{"key":"10.1016\/j.ymthe.2018.01.009_bib19","doi-asserted-by":"crossref","first-page":"1365","DOI":"10.1212\/WNL.0000000000004418","article-title":"Developmental outcomes of cord blood transplantation for Krabbe disease: A\u00a015-year study","volume":"89","author":"Wright","year":"2017","journal-title":"Neurology"},{"key":"10.1016\/j.ymthe.2018.01.009_bib20","doi-asserted-by":"crossref","first-page":"35","DOI":"10.1007\/s00401-011-0814-2","article-title":"Axonopathy is a compounding factor in the pathogenesis of Krabbe disease","volume":"122","author":"Castelvetri","year":"2011","journal-title":"Acta Neuropathol."},{"key":"10.1016\/j.ymthe.2018.01.009_bib21","doi-asserted-by":"crossref","first-page":"10048","DOI":"10.1523\/JNEUROSCI.0217-13.2013","article-title":"The sphingolipid psychosine inhibits fast axonal transport in Krabbe disease by activation of GSK3\u03b2 and deregulation of molecular motors","volume":"33","author":"Cantuti Castelvetri","year":"2013","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.ymthe.2018.01.009_bib22","doi-asserted-by":"crossref","first-page":"92","DOI":"10.1016\/j.nbd.2014.02.012","article-title":"Early axonal loss accompanied by impaired endocytosis, abnormal axonal transport, and decreased microtubule stability occur in the model of Krabbe\u2019s disease","volume":"66","author":"Teixeira","year":"2014","journal-title":"Neurobiol. Dis."},{"key":"10.1016\/j.ymthe.2018.01.009_bib23","doi-asserted-by":"crossref","first-page":"2031","DOI":"10.1038\/mt.2012.153","article-title":"Extended normal life after AAVrh10-mediated gene therapy in the mouse model of Krabbe disease","volume":"20","author":"Rafi","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2018.01.009_bib24","doi-asserted-by":"crossref","first-page":"69","DOI":"10.1038\/gt.2011.216","article-title":"Systemic delivery of scAAV9 in fetal macaques facilitates neuronal transduction of the central and peripheral nervous systems","volume":"20","author":"Mattar","year":"2013","journal-title":"Gene Ther."},{"key":"10.1016\/j.ymthe.2018.01.009_bib25","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.2018.01.009_bib26","doi-asserted-by":"crossref","first-page":"459","DOI":"10.1016\/j.ymgme.2014.12.300","article-title":"Intravenous injection of AAVrh10-GALC after the neonatal period in twitcher mice results in significant expression in the central and peripheral nervous systems and improvement of clinical features","volume":"114","author":"Rafi","year":"2015","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2018.01.009_bib27","doi-asserted-by":"crossref","first-page":"1187","DOI":"10.1038\/ng.3389","article-title":"Recurrent AAV2-related insertional mutagenesis in human hepatocellular carcinomas","volume":"47","author":"Nault","year":"2015","journal-title":"Nat. Genet."},{"key":"10.1016\/j.ymthe.2018.01.009_bib28","doi-asserted-by":"crossref","first-page":"352","DOI":"10.1002\/jnr.22564","article-title":"Persistence of psychosine in brain lipid rafts is a limiting factor in the therapeutic recovery of a mouse model for Krabbe disease","volume":"89","author":"White","year":"2011","journal-title":"J.\u00a0Neurosci. Res."},{"key":"10.1016\/j.ymthe.2018.01.009_bib29","doi-asserted-by":"crossref","first-page":"44945","DOI":"10.1074\/jbc.M407986200","article-title":"Association of gamma-secretase with lipid rafts in post-Golgi and endosome membranes","volume":"279","author":"Vetrivel","year":"2004","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.ymthe.2018.01.009_bib30","doi-asserted-by":"crossref","first-page":"6068","DOI":"10.1523\/JNEUROSCI.5597-08.2009","article-title":"Psychosine accumulates in membrane microdomains in the brain of krabbe patients, disrupting the raft architecture","volume":"29","author":"White","year":"2009","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.ymthe.2018.01.009_bib31","doi-asserted-by":"crossref","first-page":"285","DOI":"10.1016\/0022-510X(82)90127-7","article-title":"The pathogenesis of globoid cell leucodystrophy in peripheral nerve of the mouse mutant twitcher","volume":"55","author":"Jacobs","year":"1982","journal-title":"J.\u00a0Neurol. Sci."},{"key":"10.1016\/j.ymthe.2018.01.009_bib32","doi-asserted-by":"crossref","first-page":"347","DOI":"10.1016\/0006-8993(89)91198-0","article-title":"The twitcher mouse: accumulation of galactosylsphingosine and pathology of the central nervous system","volume":"482","author":"Tanaka","year":"1989","journal-title":"Brain Res."},{"key":"10.1016\/j.ymthe.2018.01.009_bib33","first-page":"308","article-title":"The twitcher mouse. An alteration of the unmyelinated fibers in the PNS","volume":"131","author":"Kobayashi","year":"1988","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.ymthe.2018.01.009_bib34","first-page":"227","article-title":"Expression of glial fibrillary acidic protein in the CNS and PNS of murine globoid cell leukodystrophy, the twitcher","volume":"125","author":"Kobayashi","year":"1986","journal-title":"Am. J. Pathol."},{"key":"10.1016\/j.ymthe.2018.01.009_bib35","doi-asserted-by":"crossref","first-page":"18670","DOI":"10.1073\/pnas.0506473102","article-title":"Galactocerebrosidase-deficient oligodendrocytes maintain stable central myelin by exogenous replacement of the missing enzyme in mice","volume":"102","author":"Kondo","year":"2005","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2018.01.009_bib36","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.2018.01.009_bib37","doi-asserted-by":"crossref","first-page":"9945","DOI":"10.1523\/JNEUROSCI.1802-11.2011","article-title":"Bone marrow transplantation augments the effect of brain- and spinal cord-directed adeno-associated virus 2\/5 gene therapy by altering inflammation in the murine model of globoid-cell leukodystrophy","volume":"31","author":"Reddy","year":"2011","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.ymthe.2018.01.009_bib38","doi-asserted-by":"crossref","first-page":"628","DOI":"10.1093\/jnen\/59.7.628","article-title":"Infiltration of hematogenous lineage cells into the demyelinating central nervous system of twitcher mice","volume":"59","author":"Wu","year":"2000","journal-title":"J.\u00a0Neuropathol. Exp. Neurol."},{"key":"10.1016\/j.ymthe.2018.01.009_bib39","doi-asserted-by":"crossref","first-page":"146","DOI":"10.1016\/j.brainres.2009.09.017","article-title":"Effects of treatments on inflammatory and apoptotic markers in the CNS of mice with globoid cell leukodystrophy","volume":"1300","author":"Luzi","year":"2009","journal-title":"Brain Res."},{"key":"10.1016\/j.ymthe.2018.01.009_bib40","doi-asserted-by":"crossref","first-page":"4383","DOI":"10.1523\/JNEUROSCI.4531-05.2006","article-title":"Prostaglandin D2-mediated microglia\/astrocyte interaction enhances astrogliosis and demyelination in twitcher","volume":"26","author":"Mohri","year":"2006","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.ymthe.2018.01.009_bib41","doi-asserted-by":"crossref","first-page":"6969","DOI":"10.1128\/JVI.75.15.6969-6976.2001","article-title":"Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in\u00a0vivo","volume":"75","author":"Nakai","year":"2001","journal-title":"J.\u00a0Virol."},{"key":"10.1016\/j.ymthe.2018.01.009_bib42","doi-asserted-by":"crossref","first-page":"13730","DOI":"10.1523\/JNEUROSCI.3379-07.2007","article-title":"Autonomic denervation of lymphoid organs leads to epigenetic immune atrophy in a mouse model of Krabbe disease","volume":"27","author":"Galbiati","year":"2007","journal-title":"J.\u00a0Neurosci."},{"key":"10.1016\/j.ymthe.2018.01.009_bib43","doi-asserted-by":"crossref","first-page":"1118","DOI":"10.1046\/j.1471-4159.1996.66031118.x","article-title":"Molecular cloning and expression of cDNA for murine galactocerebrosidase and mutation analysis of the twitcher mouse, a model of Krabbe\u2019s disease","volume":"66","author":"Sakai","year":"1996","journal-title":"J.\u00a0Neurochem."},{"key":"10.1016\/j.ymthe.2018.01.009_bib44","doi-asserted-by":"crossref","first-page":"422","DOI":"10.1016\/j.ymthe.2005.04.019","article-title":"AAV2\/5 vector expressing galactocerebrosidase ameliorates CNS disease in the murine model of globoid-cell leukodystrophy more efficiently than AAV2","volume":"12","author":"Lin","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2018.01.009_bib45","doi-asserted-by":"crossref","first-page":"287","DOI":"10.1038\/mt.2015.187","article-title":"Production of Recombinant Adeno-associated Virus Vectors Using Suspension HEK293 Cells and Continuous Harvest of Vector From the Culture Media for GMP FIX and FLT1 Clinical Vector","volume":"24","author":"Grieger","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2018.01.009_bib46","doi-asserted-by":"crossref","first-page":"509","DOI":"10.1002\/path.4328","article-title":"Neuronal inclusions of \u03b1-synuclein contribute to the pathogenesis of Krabbe disease","volume":"232","author":"Smith","year":"2014","journal-title":"J.\u00a0Pathol."},{"key":"10.1016\/j.ymthe.2018.01.009_bib47","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."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001618300169?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001618300169?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,26]],"date-time":"2025-10-26T17:52:30Z","timestamp":1761501150000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001618300169"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,3]]},"references-count":47,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2018,3]]}},"alternative-id":["S1525001618300169"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2018.01.009","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2018,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Long-Term Improvement of Neurological Signs and Metabolic Dysfunction in a Mouse Model of Krabbe\u2019s Disease after Global Gene Therapy","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2018.01.009","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2018 The American Society of Gene and Cell Therapy.","name":"copyright","label":"Copyright"}]}}