{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,7,10]],"date-time":"2026-07-10T15:41:16Z","timestamp":1783698076055,"version":"3.55.0"},"reference-count":50,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2022,6,1]],"date-time":"2022-06-01T00:00:00Z","timestamp":1654041600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2022,6,1]],"date-time":"2022-06-01T00:00:00Z","timestamp":1654041600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2022,4,15]],"date-time":"2022-04-15T00:00:00Z","timestamp":1649980800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100004829","name":"Sichuan Province Department of Science and Technology","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100004829","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100013076","name":"National Major Science and Technology Projects of China","doi-asserted-by":"publisher","award":["2018ZX09733001-005-002"],"award-info":[{"award-number":["2018ZX09733001-005-002"]}],"id":[{"id":"10.13039\/501100013076","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100012542","name":"Sichuan Province Science and Technology Support Program","doi-asserted-by":"publisher","award":["2017SZDZX0011"],"award-info":[{"award-number":["2017SZDZX0011"]}],"id":[{"id":"10.13039\/100012542","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["U19A2002"],"award-info":[{"award-number":["U19A2002"]}],"id":[{"id":"10.13039\/501100001809","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":[[2022,6]]},"DOI":"10.1016\/j.omtm.2022.04.010","type":"journal-article","created":{"date-parts":[[2022,4,19]],"date-time":"2022-04-19T13:10:59Z","timestamp":1650373859000},"page":"370-381","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":14,"special_numbering":"C","title":["Liver-directed gene therapy corrects neurologic disease in a murine model of mucopolysaccharidosis type I-Hurler"],"prefix":"10.1016","volume":"25","author":[{"given":"Xiu","family":"Jin","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jing","family":"Su","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qinyu","family":"Zhao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ruiting","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jianlu","family":"Xiao","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Xiaomei","family":"Zhong","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Li","family":"Song","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yi","family":"Liu","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kaiqin","family":"She","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hongxin","family":"Deng","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yuquan","family":"Wei","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8190-1825","authenticated-orcid":false,"given":"Yang","family":"Yang","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2022.04.010_bib1","doi-asserted-by":"crossref","first-page":"20130467","DOI":"10.1259\/bjr.20130467","article-title":"Radiological and clinical characterization of the lysosomal storage disorders: non-lipid disorders","volume":"87","author":"Parker","year":"2014","journal-title":"Br. J. Radiol."},{"key":"10.1016\/j.omtm.2022.04.010_bib2","doi-asserted-by":"crossref","first-page":"e226","DOI":"10.1016\/j.bbmt.2019.02.012","article-title":"Hematopoietic stem cell transplantation for mucopolysaccharidoses: past, present, and future","volume":"25","author":"Taylor","year":"2019","journal-title":"Biol. Blood Marrow Transpl."},{"key":"10.1016\/j.omtm.2022.04.010_bib3","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1016\/j.ymgme.2017.10.007","article-title":"Treatment of brain disease in the mucopolysaccharidoses","volume":"122","author":"Scarpa","year":"2017","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2022.04.010_bib4","doi-asserted-by":"crossref","first-page":"100300","DOI":"10.1016\/j.jhepr.2021.100300","article-title":"Novel vectors and approaches for gene therapy in liver diseases","volume":"3","author":"Maestro","year":"2021","journal-title":"JHEP Rep."},{"key":"10.1016\/j.omtm.2022.04.010_bib5","doi-asserted-by":"crossref","first-page":"14894","DOI":"10.1073\/pnas.1413645111","article-title":"Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I","volume":"111","author":"Hinderer","year":"2014","journal-title":"Proc. Natl. Acad. Sci. U\u00a0S\u00a0A"},{"key":"10.1016\/j.omtm.2022.04.010_bib6","doi-asserted-by":"crossref","first-page":"50","DOI":"10.1016\/j.omtm.2020.05.015","article-title":"Liver-targeted AAV8 gene therapy ameliorates skeletal and cardiovascular pathology in a mucopolysaccharidosis IVA murine model","volume":"18","author":"Sawamoto","year":"2020","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2022.04.010_bib7","doi-asserted-by":"crossref","first-page":"6393","DOI":"10.1038\/s41467-021-26744-4","article-title":"Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates","volume":"12","author":"Costa-Verdera","year":"2021","journal-title":"Nat. Commun."},{"key":"10.1016\/j.omtm.2022.04.010_bib8","doi-asserted-by":"crossref","first-page":"675","DOI":"10.1002\/emmm.201202083","article-title":"A highly secreted sulphamidase engineered to cross the blood-brain barrier corrects brain lesions of mice with mucopolysaccharidoses type IIIA","volume":"5","author":"Sorrentino","year":"2013","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.omtm.2022.04.010_bib9","doi-asserted-by":"crossref","first-page":"2018","DOI":"10.1038\/mt.2014.135","article-title":"Intrathecal gene therapy corrects CNS pathology in a feline model of mucopolysaccharidosis I","volume":"22","author":"Hinderer","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.04.010_bib10","doi-asserted-by":"crossref","first-page":"275","DOI":"10.1038\/nrd.2015.21","article-title":"From blood-brain barrier to blood-brain interface: new opportunities for CNS drug delivery","volume":"15","author":"Banks","year":"2016","journal-title":"Nat. Rev. Drug Discov."},{"key":"10.1016\/j.omtm.2022.04.010_bib11","doi-asserted-by":"crossref","first-page":"1342","DOI":"10.1021\/mp200136x","article-title":"Reversal of lysosomal storage in brain of adult MPS-I mice with intravenous Trojan horse-iduronidase fusion protein","volume":"8","author":"Boado","year":"2011","journal-title":"Mol. Pharm."},{"key":"10.1016\/j.omtm.2022.04.010_bib12","doi-asserted-by":"crossref","first-page":"110","DOI":"10.1186\/s13023-018-0849-8","article-title":"Neurocognitive and somatic stabilization in pediatric patients with severe Mucopolysaccharidosis Type I after 52\u00a0weeks of intravenous brain-penetrating insulin receptor antibody-iduronidase fusion protein (valanafusp alpha): an open label phase 1-2 trial","volume":"13","author":"Giugliani","year":"2018","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.omtm.2022.04.010_bib13","doi-asserted-by":"crossref","first-page":"2028","DOI":"10.1038\/mt.2014.152","article-title":"Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase","volume":"22","author":"El-Amouri","year":"2014","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.04.010_bib14","doi-asserted-by":"crossref","first-page":"e2469","DOI":"10.1371\/journal.pone.0002469","article-title":"A unique carrier for delivery of therapeutic compounds beyond the blood-brain barrier","volume":"3","author":"Karkan","year":"2008","journal-title":"PLoS One"},{"key":"10.1016\/j.omtm.2022.04.010_bib15","doi-asserted-by":"crossref","first-page":"2930","DOI":"10.1007\/s11095-016-2015-0","article-title":"Anti-cancer antibody trastuzumab-melanotransferrin conjugate (BT2111) for the treatment of metastatic HER2+ breast cancer tumors in the brain: an in-vivo study","volume":"33","author":"Nounou","year":"2016","journal-title":"Pharm. Res."},{"key":"10.1016\/j.omtm.2022.04.010_bib16","doi-asserted-by":"crossref","first-page":"596976","DOI":"10.3389\/fnins.2021.596976","article-title":"Discovery of a highly conserved peptide in the iron transporter melanotransferrin that traverses an intact blood brain barrier and localized in neural cells","volume":"15","author":"Singh","year":"2021","journal-title":"Front. Neurosci."},{"key":"10.1016\/j.omtm.2022.04.010_bib17","doi-asserted-by":"crossref","first-page":"2074","DOI":"10.1177\/0271678X18772998","article-title":"A peptide derived from melanotransferrin delivers a protein-based interleukin 1 receptor antagonist across the BBB and ameliorates neuropathic pain in a preclinical model","volume":"39","author":"Thom","year":"2019","journal-title":"J.\u00a0Cereb. Blood Flow Metab."},{"key":"10.1016\/j.omtm.2022.04.010_bib18","doi-asserted-by":"crossref","first-page":"611367","DOI":"10.3389\/fmolb.2021.611367","article-title":"A nanomule peptide carrier delivers siRNA across the intact blood-brain barrier to attenuate ischemic stroke","volume":"8","author":"Eyford","year":"2021","journal-title":"Front. Mol. Biosci."},{"key":"10.1016\/j.omtm.2022.04.010_bib19","doi-asserted-by":"crossref","first-page":"483","DOI":"10.1146\/annurev.cb.05.110189.002411","article-title":"The biogenesis of lysosomes","volume":"5","author":"Kornfeld","year":"1989","journal-title":"Annu. Rev. Cell Biol."},{"key":"10.1016\/j.omtm.2022.04.010_bib20","doi-asserted-by":"crossref","first-page":"148","DOI":"10.1097\/00005072-199802000-00006","article-title":"Caprine mucopolysaccharidosis-IIID: clinical, biochemical, morphological and immunohistochemical characteristics","volume":"57","author":"Jones","year":"1998","journal-title":"J.\u00a0Neuropathol. Exp. Neurol."},{"key":"10.1016\/j.omtm.2022.04.010_bib21","doi-asserted-by":"crossref","first-page":"62","DOI":"10.1016\/j.ymgme.2009.08.002","article-title":"Characterization of an MPS I-H knock-in mouse that carries a nonsense mutation analogous to the human IDUA-W402X mutation","volume":"99","author":"Wang","year":"2010","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2022.04.010_bib22","doi-asserted-by":"crossref","first-page":"315","DOI":"10.1111\/j.1365-2990.1988.tb00891.x","article-title":"Focal dendritic swellings in Purkinje cells in mucopolysaccharidoses types I, II and III. A Golgi and ultrastructural study","volume":"14","author":"Ferrer","year":"1988","journal-title":"Neuropathol. Appl. Neurobiol."},{"key":"10.1016\/j.omtm.2022.04.010_bib23","doi-asserted-by":"crossref","first-page":"1838","DOI":"10.3390\/cells9081838","article-title":"Mucopolysaccharidosis type I: a review of the natural history and molecular pathology","volume":"9","author":"Hampe","year":"2020","journal-title":"Cells"},{"key":"10.1016\/j.omtm.2022.04.010_bib24","doi-asserted-by":"crossref","first-page":"241","DOI":"10.1016\/j.brainres.2007.10.036","article-title":"Progression of multiple behavioral deficits with various ages of onset in a murine model of Hurler syndrome","volume":"1188","author":"Pan","year":"2008","journal-title":"Brain Res."},{"key":"10.1016\/j.omtm.2022.04.010_bib25","doi-asserted-by":"crossref","first-page":"e2389","DOI":"10.21769\/BioProtoc.2389","article-title":"Delayed-matching-to-place task in a dry maze to measure spatial working memory in mice","volume":"7","author":"Feng","year":"2017","journal-title":"Bio Protoc."},{"key":"10.1016\/j.omtm.2022.04.010_bib26","doi-asserted-by":"crossref","first-page":"1103","DOI":"10.1007\/s40265-019-01147-4","article-title":"Therapeutic options for mucopolysaccharidoses: current and emerging treatments","volume":"79","author":"Sawamoto","year":"2019","journal-title":"Drugs"},{"key":"10.1016\/j.omtm.2022.04.010_bib27","first-page":"3","article-title":"Characterization of the MPS I-H knock-in mouse reveals increased femoral biomechanical integrity with compromised material strength and altered bone geometry","volume":"5","author":"Oestreich","year":"2015","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.omtm.2022.04.010_bib28","doi-asserted-by":"crossref","first-page":"374","DOI":"10.1016\/j.ymgme.2013.12.007","article-title":"Long-term nonsense suppression therapy moderates MPS I-H disease progression","volume":"111","author":"Gunn","year":"2014","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2022.04.010_bib29","doi-asserted-by":"crossref","first-page":"1261","DOI":"10.1038\/mt.2008.84","article-title":"Mannose 6-phosphate receptor-mediated transport of sulfamidase across the blood-brain barrier in the newborn mouse","volume":"16","author":"Urayama","year":"2008","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.04.010_bib30","doi-asserted-by":"crossref","first-page":"3437","DOI":"10.1093\/hmg\/ddn237","article-title":"Reversal of peripheral and central neural storage and ataxia after recombinant enzyme replacement therapy in alpha-mannosidosis mice","volume":"17","author":"Blanz","year":"2008","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2022.04.010_bib31","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.omtm.2022.04.010_bib32","doi-asserted-by":"crossref","first-page":"178","DOI":"10.1016\/j.ymthe.2018.10.018","article-title":"ZFN-mediated in\u00a0vivo genome editing corrects murine Hurler syndrome","volume":"27","author":"Ou","year":"2019","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2022.04.010_bib33","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.omtm.2022.04.010_bib34","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. U\u00a0S\u00a0A"},{"key":"10.1016\/j.omtm.2022.04.010_bib35","doi-asserted-by":"crossref","first-page":"105","DOI":"10.1016\/j.ymgme.2017.11.013","article-title":"RTB lectin-mediated delivery of lysosomal \u03b1-l-iduronidase mitigates disease manifestations systemically including the central nervous system","volume":"123","author":"Ou","year":"2018","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2022.04.010_bib36","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.omtm.2022.04.010_bib37","doi-asserted-by":"crossref","first-page":"576","DOI":"10.1089\/hum.2017.187","article-title":"Intranasal adeno-associated virus mediated gene delivery and expression of human iduronidase in the central nervous system: a noninvasive and effective approach for prevention of neurologic disease in mucopolysaccharidosis type I","volume":"28","author":"Belur","year":"2017","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2022.04.010_bib38","doi-asserted-by":"crossref","first-page":"618360","DOI":"10.3389\/fnmol.2021.618360","article-title":"Comparative effectiveness of intracerebroventricular, intrathecal, and intranasal routes of AAV9 vector administration for genetic therapy of neurologic disease in murine mucopolysaccharidosis type I","volume":"14","author":"Belur","year":"2021","journal-title":"Front. Mol. Neurosci."},{"key":"10.1016\/j.omtm.2022.04.010_bib39","doi-asserted-by":"crossref","first-page":"49","DOI":"10.1146\/annurev.psych.093008.100523","article-title":"Hippocampal-neocortical interactions in memory formation, consolidation, and reconsolidation","volume":"61","author":"Wang","year":"2010","journal-title":"Annu. Rev. Psychol."},{"key":"10.1016\/j.omtm.2022.04.010_bib40","doi-asserted-by":"crossref","first-page":"95","DOI":"10.1515\/REVNEURO.2000.11.2-3.95","article-title":"Sensory and memory properties of hippocampal place cells","volume":"11","author":"Poucet","year":"2000","journal-title":"Rev. Neurosci."},{"key":"10.1016\/j.omtm.2022.04.010_bib41","doi-asserted-by":"crossref","first-page":"359","DOI":"10.1016\/S0079-6123(08)62623-0","article-title":"Chapter 40 Spatial memory deficit resulting from ischemia-induced damage to the hippocampus is ameliorated by intra-hippocampal transplants of fetal hippocampal neurons","volume":"82","author":"Onifer","year":"1990","journal-title":"Prog. Brain Res."},{"key":"10.1016\/j.omtm.2022.04.010_bib42","first-page":"100604","article-title":"Intravenous delivery for treatment of mucopolysaccharidosis type I: a comparison of AAV serotypes 9 and rh10","volume":"24","author":"Belur","year":"2020","journal-title":"Mol. Genet. Metab. Rep."},{"key":"10.1016\/j.omtm.2022.04.010_bib43","doi-asserted-by":"crossref","first-page":"491","DOI":"10.1182\/blood-2018-99-118334","article-title":"Adeno-associated mediated gene transfer for hemophilia B:8 Year follow up and impact of removing \u201cempty viral particles\u201d on safety and efficacy of gene transfer","volume":"132","author":"Nathwani","year":"2018","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2022.04.010_bib44","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.omtm.2022.04.010_bib45","doi-asserted-by":"crossref","first-page":"1961","DOI":"10.1056\/NEJMoa2104205","article-title":"Multiyear factor VIII expression after AAV gene transfer for hemophilia A","volume":"385","author":"George","year":"2021","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2022.04.010_bib46","doi-asserted-by":"crossref","first-page":"862","DOI":"10.1177\/0271678X16630991","article-title":"In\u00a0vitro models of the blood-brain barrier: an overview of commonly used brain endothelial cell culture models and guidelines for their use","volume":"36","author":"Helms","year":"2016","journal-title":"J.\u00a0Cereb. Blood Flow Metab."},{"key":"10.1016\/j.omtm.2022.04.010_bib47","doi-asserted-by":"crossref","first-page":"652","DOI":"10.1016\/j.omtm.2021.02.005","article-title":"In\u00a0vivo PCSK9 gene editing using an all-in-one self-cleavage AAV-CRISPR system","volume":"20","author":"Li","year":"2021","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2022.04.010_bib48","doi-asserted-by":"crossref","first-page":"115","DOI":"10.1089\/hgtb.2013.131","article-title":"Absolute determination of single-stranded and self-complementary adeno-associated viral vector genome titers by droplet digital PCR","volume":"25","author":"Lock","year":"2014","journal-title":"Hum. Gene Ther. Methods"},{"key":"10.1016\/j.omtm.2022.04.010_bib49","doi-asserted-by":"crossref","first-page":"334","DOI":"10.1038\/nbt.3469","article-title":"A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice","volume":"34","author":"Yang","year":"2016","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2022.04.010_bib50","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1037\/h0077579","article-title":"Memory deficits associated with senescence: a neurophysiological and behavioral study in the rat","volume":"93","author":"Barnes","year":"1979","journal-title":"J.\u00a0Comp. Physiol. Psychol."}],"container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050122000584?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050122000584?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,11,6]],"date-time":"2025-11-06T09:27:53Z","timestamp":1762421273000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050122000584"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2022,6]]},"references-count":50,"alternative-id":["S2329050122000584"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2022.04.010","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2022,6]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Liver-directed gene therapy corrects neurologic disease in a murine model of mucopolysaccharidosis type I-Hurler","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2022.04.010","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2022 The Author(s).","name":"copyright","label":"Copyright"}]}}