{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,27]],"date-time":"2026-09-27T14:19:21Z","timestamp":1790518761684,"version":"4.1.0"},"reference-count":36,"publisher":"Elsevier BV","issue":"1","license":[{"start":{"date-parts":[[2019,1,1]],"date-time":"2019-01-01T00:00:00Z","timestamp":1546300800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2019,1,1]],"date-time":"2019-01-01T00:00:00Z","timestamp":1546300800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/legal\/tdmrep-license"},{"start":{"date-parts":[[2018,10,31]],"date-time":"2018-10-31T00:00:00Z","timestamp":1540944000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100018156","name":"Sangamo Therapeutics","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100018156","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2019,1]]},"DOI":"10.1016\/j.ymthe.2018.10.018","type":"journal-article","created":{"date-parts":[[2018,11,1]],"date-time":"2018-11-01T20:28:50Z","timestamp":1541104130000},"page":"178-187","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":75,"title":["ZFN-Mediated In\u00a0Vivo Genome Editing Corrects Murine Hurler Syndrome"],"prefix":"10.1016","volume":"27","author":[{"given":"Li","family":"Ou","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Russell C.","family":"DeKelver","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michelle","family":"Rohde","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan","family":"Tom","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert","family":"Radeke","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susan J.","family":"St. Martin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yolanda","family":"Santiago","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Scott","family":"Sproul","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael J.","family":"Przybilla","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brenda L.","family":"Koniar","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kelly M.","family":"Podetz-Pedersen","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kanut","family":"Laoharawee","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Renee D.","family":"Cooksley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kathleen E.","family":"Meyer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael C.","family":"Holmes","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R. Scott","family":"McIvor","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Thomas","family":"Wechsler","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chester B.","family":"Whitley","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymthe.2018.10.018_bib1","series-title":"The Online Metabolic and Molecular Bases of Inherited Disease","author":"Neufeld","year":"2000"},{"key":"10.1016\/j.ymthe.2018.10.018_bib2","doi-asserted-by":"crossref","first-page":"19","DOI":"10.1542\/peds.2008-0416","article-title":"Mucopolysaccharidosis I: management and treatment guidelines","volume":"123","author":"Muenzer","year":"2009","journal-title":"Pediatrics"},{"key":"10.1016\/j.ymthe.2018.10.018_bib3","doi-asserted-by":"crossref","first-page":"759","DOI":"10.1038\/gim.2014.25","article-title":"The natural history of MPS I: global perspectives from the MPS I Registry","volume":"16","author":"Beck","year":"2014","journal-title":"Genet. Med."},{"key":"10.1016\/j.ymthe.2018.10.018_bib4","doi-asserted-by":"crossref","first-page":"589","DOI":"10.1590\/S1415-47572010005000093","article-title":"Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment","volume":"33","author":"Giugliani","year":"2010","journal-title":"Genet. Mol. Biol."},{"key":"10.1016\/j.ymthe.2018.10.018_bib5","doi-asserted-by":"crossref","first-page":"217","DOI":"10.1038\/nature10177","article-title":"In\u00a0vivo genome editing restores haemostasis in a mouse model of haemophilia","volume":"475","author":"Li","year":"2011","journal-title":"Nature"},{"key":"10.1016\/j.ymthe.2018.10.018_bib6","doi-asserted-by":"crossref","first-page":"3283","DOI":"10.1182\/blood-2013-04-497354","article-title":"Robust ZFN-mediated genome editing in adult hemophilic mice","volume":"122","author":"Anguela","year":"2013","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2018.10.018_bib7","doi-asserted-by":"crossref","first-page":"1777","DOI":"10.1182\/blood-2014-12-615492","article-title":"In\u00a0vivo genome editing of the albumin locus as a platform for protein replacement therapy","volume":"126","author":"Sharma","year":"2015","journal-title":"Blood"},{"key":"10.1016\/j.ymthe.2018.10.018_bib8","doi-asserted-by":"crossref","first-page":"286","DOI":"10.1097\/01.GIM.0000078027.83236.49","article-title":"Can mucopolysaccharidosis type I disease severity be predicted based on a patient\u2019s genotype? A comprehensive review of the literature","volume":"5","author":"Terlato","year":"2003","journal-title":"Genet. Med."},{"key":"10.1016\/j.ymthe.2018.10.018_bib9","doi-asserted-by":"crossref","first-page":"503","DOI":"10.1007\/s004390100606","article-title":"Mutational analysis of 85 mucopolysaccharidosis type I families: frequency of known mutations, identification of 17 novel mutations and in\u00a0vitro expression of missense mutations","volume":"109","author":"Beesley","year":"2001","journal-title":"Hum. Genet."},{"key":"10.1016\/j.ymthe.2018.10.018_bib10","doi-asserted-by":"crossref","first-page":"199","DOI":"10.1002\/humu.20081","article-title":"Identification and molecular characterization of alpha-L-iduronidase mutations present in mucopolysaccharidosis type I patients undergoing enzyme replacement therapy","volume":"24","author":"Yogalingam","year":"2004","journal-title":"Hum. Mutat."},{"key":"10.1016\/j.ymthe.2018.10.018_bib11","doi-asserted-by":"crossref","first-page":"637","DOI":"10.1146\/annurev-chembioeng-080615-034711","article-title":"Engineering Delivery Vehicles for Genome Editing","volume":"7","author":"Nelson","year":"2016","journal-title":"Annu. Rev. Chem. Biomol. Eng."},{"key":"10.1016\/j.ymthe.2018.10.018_bib12","doi-asserted-by":"crossref","first-page":"1902","DOI":"10.1073\/pnas.252784899","article-title":"Activated microglia in cortex of mouse models of mucopolysaccharidoses I and IIIB","volume":"100","author":"Ohmi","year":"2003","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2018.10.018_bib13","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.2018.10.018_bib14","doi-asserted-by":"crossref","first-page":"533","DOI":"10.1089\/hum.2011.183","article-title":"Hepatic gene transfer in neonatal mice by adeno-associated virus serotype 8 vector","volume":"23","author":"Wang","year":"2012","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2018.10.018_bib15","doi-asserted-by":"crossref","first-page":"1298","DOI":"10.1038\/mt.2015.99","article-title":"Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates","volume":"23","author":"Hinderer","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2018.10.018_bib16","doi-asserted-by":"crossref","first-page":"866","DOI":"10.1016\/j.ymthe.2004.03.011","article-title":"Correction of metabolic, craniofacial, and neurologic abnormalities in MPS I mice treated at birth with adeno-associated virus vector transducing the human alpha-L-iduronidase gene","volume":"9","author":"Hartung","year":"2004","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2018.10.018_bib17","doi-asserted-by":"crossref","first-page":"116","DOI":"10.1016\/j.ymgme.2013.09.008","article-title":"High-dose enzyme replacement therapy in murine Hurler syndrome","volume":"111","author":"Ou","year":"2014","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymthe.2018.10.018_bib18","doi-asserted-by":"crossref","first-page":"908","DOI":"10.1089\/hum.2009.031","article-title":"Effect of adeno-associated virus serotype and genomic structure on liver transduction and biodistribution in mice of both genders","volume":"20","author":"Pa\u00f1eda","year":"2009","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.ymthe.2018.10.018_bib19","doi-asserted-by":"crossref","first-page":"582","DOI":"10.1016\/j.biocel.2012.01.004","article-title":"Chondroitin sulfate: a key molecule in the brain matrix","volume":"44","author":"Kwok","year":"2012","journal-title":"Int. J. Biochem. Cell Biol."},{"key":"10.1016\/j.ymthe.2018.10.018_bib20","doi-asserted-by":"crossref","first-page":"126","DOI":"10.3109\/01913121003648485","article-title":"Ultrastructural analysis of dermal fibroblasts in mucopolysaccharidosis type I: Effects of enzyme replacement therapy and hematopoietic cell transplantation","volume":"34","author":"Cox-Brinkman","year":"2010","journal-title":"Ultrastruct. Pathol."},{"key":"10.1016\/j.ymthe.2018.10.018_bib21","doi-asserted-by":"crossref","first-page":"870","DOI":"10.1172\/JCI79213","article-title":"Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy","volume":"125","author":"Chandler","year":"2015","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.ymthe.2018.10.018_bib22","doi-asserted-by":"crossref","first-page":"415","DOI":"10.1126\/science.1088547","article-title":"LMO2-associated clonal T\u00a0cell proliferation in two patients after gene therapy for SCID-X1","volume":"302","author":"Hacein-Bey-Abina","year":"2003","journal-title":"Science"},{"key":"10.1016\/j.ymthe.2018.10.018_bib23","doi-asserted-by":"crossref","first-page":"581","DOI":"10.1016\/j.jpeds.2004.01.046","article-title":"Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase)","volume":"144","author":"Wraith","year":"2004","journal-title":"J.\u00a0Pediatr."},{"key":"10.1016\/j.ymthe.2018.10.018_bib24","series-title":"Stocker and Dehner\u2019s Pediatric Pathology","author":"Stocker","year":"2012"},{"key":"10.1016\/j.ymthe.2018.10.018_bib25","doi-asserted-by":"crossref","first-page":"673","DOI":"10.1096\/fasebj.10.7.8635684","article-title":"Analysis of liver development, regeneration, and carcinogenesis by genetic marking studies","volume":"10","author":"Ponder","year":"1996","journal-title":"FASEB J."},{"key":"10.1016\/j.ymthe.2018.10.018_bib26","doi-asserted-by":"crossref","first-page":"466","DOI":"10.1053\/j.gastro.2009.05.044","article-title":"Stem cells and liver regeneration","volume":"137","author":"Duncan","year":"2009","journal-title":"Gastroenterology"},{"key":"10.1016\/j.ymthe.2018.10.018_bib27","doi-asserted-by":"crossref","first-page":"692","DOI":"10.1016\/j.jhep.2012.04.016","article-title":"Liver regeneration","volume":"57","author":"Fausto","year":"2012","journal-title":"J.\u00a0Hepatol."},{"key":"10.1016\/j.ymthe.2018.10.018_bib28","doi-asserted-by":"crossref","first-page":"1127","DOI":"10.1016\/j.ymthe.2018.03.002","article-title":"Dose-Dependent Prevention of Metabolic and Neurologic Disease in Murine MPS II by ZFN-Mediated In\u00a0Vivo Genome Editing","volume":"26","author":"Laoharawee","year":"2018","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymthe.2018.10.018_bib29","doi-asserted-by":"crossref","first-page":"1450","DOI":"10.1016\/j.cell.2018.07.002","article-title":"Heparan Sulfate Organizes Neuronal Synapses through Neurexin Partnerships","volume":"174","author":"Zhang","year":"2018","journal-title":"Cell"},{"key":"10.1016\/j.ymthe.2018.10.018_bib30","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1186\/s13023-015-0356-0","article-title":"Effect of systemic high dose enzyme replacement therapy on the improvement of CNS defects in a mouse model of mucopolysaccharidosis type II","volume":"10","author":"Cho","year":"2015","journal-title":"Orphanet J. Rare Dis."},{"key":"10.1016\/j.ymthe.2018.10.018_bib31","doi-asserted-by":"crossref","first-page":"14777","DOI":"10.1073\/pnas.0506892102","article-title":"Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII","volume":"102","author":"Vogler","year":"2005","journal-title":"Proc. Natl. Acad. Sci. USA"},{"key":"10.1016\/j.ymthe.2018.10.018_bib32","doi-asserted-by":"crossref","first-page":"265","DOI":"10.1042\/cs0570265","article-title":"Biochemical discrimination of Hurler and Scheie syndromes","volume":"57","author":"Hopwood","year":"1979","journal-title":"Clin. Sci. (Lond.)"},{"key":"10.1016\/j.ymthe.2018.10.018_bib33","doi-asserted-by":"crossref","first-page":"403","DOI":"10.1002\/jgm.1028","article-title":"Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses","volume":"9","author":"Aronovich","year":"2007","journal-title":"J.\u00a0Gene Med."},{"key":"10.1016\/j.ymthe.2018.10.018_bib34","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1038\/nmeth.1539","article-title":"Enhancing zinc-finger-nuclease activity with improved obligate heterodimeric architectures","volume":"8","author":"Doyon","year":"2011","journal-title":"Nat. Methods"},{"key":"10.1016\/j.ymthe.2018.10.018_bib35","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1016\/0022-2836(70)90057-4","article-title":"A general method applicable to the search for similarities in the amino acid sequence of two proteins","volume":"48","author":"Needleman","year":"1970","journal-title":"J.\u00a0Mol. Biol."},{"key":"10.1016\/j.ymthe.2018.10.018_bib36","doi-asserted-by":"crossref","first-page":"113","DOI":"10.1016\/j.ymgme.2013.11.009","article-title":"Standardization of \u03b1-L-iduronidase enzyme assay with Michaelis-Menten kinetics","volume":"111","author":"Ou","year":"2014","journal-title":"Mol. Genet. Metab."}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001618305252?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001618305252?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2025,10,26]],"date-time":"2025-10-26T17:54:10Z","timestamp":1761501250000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001618305252"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2019,1]]},"references-count":36,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2019,1]]}},"alternative-id":["S1525001618305252"],"URL":"https:\/\/doi.org\/10.1016\/j.ymthe.2018.10.018","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2019,1]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"ZFN-Mediated In\u00a0Vivo Genome Editing Corrects Murine Hurler Syndrome","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymthe.2018.10.018","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2018 The Author(s).","name":"copyright","label":"Copyright"}]}}