{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,23]],"date-time":"2026-09-23T08:48:30Z","timestamp":1790153310760,"version":"4.0.1"},"reference-count":40,"publisher":"Elsevier BV","license":[{"start":{"date-parts":[[2017,3,1]],"date-time":"2017-03-01T00:00:00Z","timestamp":1488326400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2017,1,6]],"date-time":"2017-01-06T00:00:00Z","timestamp":1483660800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"NIH","doi-asserted-by":"publisher","award":["R01AR065873"],"award-info":[{"award-number":["R01AR065873"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/100000069","name":"National Institute of Arthritis and Musculoskeletal and Skin Diseases","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100000069","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Genethon"}],"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":[[2017,3]]},"DOI":"10.1016\/j.omtm.2016.12.010","type":"journal-article","created":{"date-parts":[[2017,1,11]],"date-time":"2017-01-11T06:04:11Z","timestamp":1484114651000},"page":"126-136","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":75,"special_numbering":"C","title":["Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction"],"prefix":"10.1016","volume":"4","author":[{"given":"Sang-oh","family":"Han","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giuseppe","family":"Ronzitti","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Benjamin","family":"Arnson","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christian","family":"Leborgne","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Songtao","family":"Li","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Federico","family":"Mingozzi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dwight","family":"Koeberl","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2016.12.010_bib1","series-title":"The Metabolic and Molecular Basis for Inherited Disease","first-page":"3389","article-title":"Chapter 135","author":"Hirschhorn","year":"2001"},{"key":"10.1016\/j.omtm.2016.12.010_bib2","doi-asserted-by":"crossref","first-page":"729","DOI":"10.1097\/GIM.0b013e3182174703","article-title":"The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: lessons learned from infantile Pompe disease","volume":"13","author":"Banugaria","year":"2011","journal-title":"Genet. Med."},{"key":"10.1016\/j.omtm.2016.12.010_bib3","doi-asserted-by":"crossref","first-page":"135","DOI":"10.1038\/gim.2011.4","article-title":"Successful immune tolerance induction to enzyme replacement therapy in CRIM-negative infantile Pompe disease","volume":"14","author":"Messinger","year":"2012","journal-title":"Genet. Med."},{"key":"10.1016\/j.omtm.2016.12.010_bib4","doi-asserted-by":"crossref","first-page":"e67052","DOI":"10.1371\/journal.pone.0067052","article-title":"Algorithm for the early diagnosis and treatment of patients with cross reactive immunologic material-negative classic infantile pompe disease: a step towards improving the efficacy of ERT","volume":"8","author":"Banugaria","year":"2013","journal-title":"PLoS ONE"},{"key":"10.1016\/j.omtm.2016.12.010_bib5","doi-asserted-by":"crossref","first-page":"1396","DOI":"10.1056\/NEJMoa0909859","article-title":"A randomized study of alglucosidase alfa in late-onset Pompe\u2019s disease","volume":"362","author":"van der Ploeg","year":"2010","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2016.12.010_bib6","doi-asserted-by":"crossref","first-page":"301","DOI":"10.1016\/j.ymgme.2012.04.027","article-title":"The impact of antibodies in late-onset Pompe disease: a case series and literature review","volume":"106","author":"Patel","year":"2012","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2016.12.010_bib7","doi-asserted-by":"crossref","first-page":"876","DOI":"10.1016\/j.ymthe.2005.04.024","article-title":"Evasion of immune responses to introduced human acid alpha-glucosidase by liver-restricted expression in glycogen storage disease type II","volume":"12","author":"Franco","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2016.12.010_bib8","doi-asserted-by":"crossref","first-page":"1042","DOI":"10.1086\/522236","article-title":"Enhanced response to enzyme replacement therapy in Pompe disease after the induction of immune tolerance","volume":"81","author":"Sun","year":"2007","journal-title":"Am. J. Hum. Genet."},{"key":"10.1016\/j.omtm.2016.12.010_bib9","doi-asserted-by":"crossref","first-page":"353","DOI":"10.1038\/mt.2009.195","article-title":"Immunomodulatory gene therapy prevents antibody formation and lethal hypersensitivity reactions in murine pompe disease","volume":"18","author":"Sun","year":"2010","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2016.12.010_bib10","doi-asserted-by":"crossref","first-page":"609","DOI":"10.1089\/hum.2008.010","article-title":"Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice","volume":"19","author":"Ziegler","year":"2008","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2016.12.010_bib11","doi-asserted-by":"crossref","first-page":"43","DOI":"10.1089\/hum.2015.103","article-title":"Copackaged AAV9 vectors promote simultaneous immune tolerance and phenotypic correction of Pompe disease","volume":"27","author":"Doerfler","year":"2016","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2016.12.010_bib12","doi-asserted-by":"crossref","first-page":"460","DOI":"10.1089\/hum.2011.063","article-title":"Immunodominant liver-specific expression suppresses transgene-directed immune responses in murine pompe disease","volume":"23","author":"Zhang","year":"2012","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2016.12.010_bib13","doi-asserted-by":"crossref","first-page":"159","DOI":"10.1016\/j.ymgme.2003.08.022","article-title":"Enzyme replacement therapy in the mouse model of Pompe disease","volume":"80","author":"Raben","year":"2003","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2016.12.010_bib14","doi-asserted-by":"crossref","first-page":"48","DOI":"10.1016\/j.ymthe.2004.09.017","article-title":"Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers","volume":"11","author":"Raben","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2016.12.010_bib15","doi-asserted-by":"crossref","first-page":"321","DOI":"10.1016\/j.nmd.2014.12.004","article-title":"Safety and efficacy of alternative alglucosidase alfa regimens in Pompe disease","volume":"25","author":"Case","year":"2015","journal-title":"Neuromuscul. Disord."},{"key":"10.1016\/j.omtm.2016.12.010_bib16","doi-asserted-by":"crossref","first-page":"14018","DOI":"10.1038\/mtm.2014.18","article-title":"Assessment of toxicity and biodistribution of recombinant AAV8 vector-mediated immunomodulatory gene therapy in mice with Pompe disease","volume":"1","author":"Wang","year":"2014","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2016.12.010_bib17","doi-asserted-by":"crossref","first-page":"1347","DOI":"10.1172\/JCI200316887","article-title":"Induction of immune tolerance to coagulation factor IX antigen by in\u00a0vivo hepatic gene transfer","volume":"111","author":"Mingozzi","year":"2003","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2016.12.010_bib18","doi-asserted-by":"crossref","first-page":"15007","DOI":"10.1038\/mtm.2015.7","article-title":"Comparative impact of AAV and enzyme replacement therapy on respiratory and cardiac function in adult Pompe mice","volume":"2","author":"Falk","year":"2015","journal-title":"Mol. Ther. Methods Clin. Dev."},{"key":"10.1016\/j.omtm.2016.12.010_bib19","doi-asserted-by":"crossref","first-page":"34","DOI":"10.1096\/fj.12-207472","article-title":"Adjunctive \u03b22-agonists reverse neuromuscular involvement in murine Pompe disease","volume":"27","author":"Li","year":"2013","journal-title":"FASEB J."},{"key":"10.1016\/j.omtm.2016.12.010_bib20","doi-asserted-by":"crossref","first-page":"5842","DOI":"10.1182\/blood-2010-06-288001","article-title":"Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy","volume":"116","author":"Finn","year":"2010","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2016.12.010_bib21","doi-asserted-by":"crossref","first-page":"1698","DOI":"10.1002\/emmm.201302859","article-title":"Effective gene therapy for haemophilic mice with pathogenic factor IX antibodies","volume":"5","author":"Markusic","year":"2013","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.omtm.2016.12.010_bib22","doi-asserted-by":"crossref","first-page":"1684","DOI":"10.1002\/emmm.201302857","article-title":"Liver gene therapy by lentiviral vectors reverses anti-factor IX pre-existing immunity in haemophilic mice","volume":"5","author":"Annoni","year":"2013","journal-title":"EMBO Mol. Med."},{"key":"10.1016\/j.omtm.2016.12.010_bib23","doi-asserted-by":"crossref","first-page":"1553","DOI":"10.1182\/blood-2014-07-588194","article-title":"AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice","volume":"125","author":"Crudele","year":"2015","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2016.12.010_bib24","doi-asserted-by":"crossref","first-page":"57","DOI":"10.1016\/j.ymthe.2004.10.004","article-title":"Efficacy of an adeno-associated virus 8-pseudotyped vector in glycogen storage disease type II","volume":"11","author":"Sun","year":"2005","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2016.12.010_bib25","doi-asserted-by":"crossref","first-page":"480","DOI":"10.1182\/blood-2002-09-2889","article-title":"Sex significantly influences transduction of murine liver by recombinant adeno-associated viral vectors through an androgen-dependent pathway","volume":"102","author":"Davidoff","year":"2003","journal-title":"Blood"},{"key":"10.1016\/j.omtm.2016.12.010_bib26","doi-asserted-by":"crossref","first-page":"67","DOI":"10.1016\/j.ymthe.2005.09.003","article-title":"High levels of persistent expression of alpha1-antitrypsin mediated by the nonhuman primate serotype rh.10 adeno-associated virus despite preexisting immunity to common human adeno-associated viruses","volume":"13","author":"De","year":"2006","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2016.12.010_bib27","doi-asserted-by":"crossref","first-page":"91","DOI":"10.1016\/j.ymgme.2008.10.017","article-title":"Long-term inhibition of glycosphingolipid accumulation in Fabry model mice by a single systemic injection of AAV1 vector in the neonatal period","volume":"96","author":"Ogawa","year":"2009","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2016.12.010_bib28","doi-asserted-by":"crossref","first-page":"1007","DOI":"10.1089\/hum.2013.166","article-title":"Safety and liver transduction efficacy of rAAV5-cohPBGD in nonhuman primates: a potential therapy for acute intermittent porphyria","volume":"24","author":"Pa\u00f1eda","year":"2013","journal-title":"Hum. Gene Ther."},{"key":"10.1016\/j.omtm.2016.12.010_bib29","doi-asserted-by":"crossref","first-page":"776","DOI":"10.1016\/j.jhep.2016.05.012","article-title":"Phase I open label liver-directed gene therapy clinical trial for acute intermittent porphyria","volume":"65","author":"D\u2019Avola","year":"2016","journal-title":"J.\u00a0Hepatol."},{"key":"10.1016\/j.omtm.2016.12.010_bib30","doi-asserted-by":"crossref","first-page":"2357","DOI":"10.1056\/NEJMoa1108046","article-title":"Adenovirus-associated virus vector-mediated gene transfer in hemophilia B","volume":"365","author":"Nathwani","year":"2011","journal-title":"N.\u00a0Engl. J. Med."},{"key":"10.1016\/j.omtm.2016.12.010_bib31","doi-asserted-by":"crossref","first-page":"1042","DOI":"10.1038\/mt.2016.61","article-title":"Superior in\u00a0vivo transduction of human hepatocytes using engineered AAV3 capsid","volume":"24","author":"Vercauteren","year":"2016","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2016.12.010_bib32","doi-asserted-by":"crossref","first-page":"382","DOI":"10.1038\/nature12875","article-title":"Selection and evaluation of clinically relevant AAV variants in a xenograft liver model","volume":"506","author":"Lisowski","year":"2014","journal-title":"Nature"},{"key":"10.1016\/j.omtm.2016.12.010_bib33","doi-asserted-by":"crossref","first-page":"1877","DOI":"10.1038\/mt.2015.179","article-title":"Comparative study of liver gene transfer with AAV vectors based on natural and engineered AAV capsids","volume":"23","author":"Wang","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2016.12.010_bib34","doi-asserted-by":"crossref","first-page":"1867","DOI":"10.1038\/mt.2015.174","article-title":"Efficient and targeted transduction of nonhuman primate liver with systemically delivered optimized AAV3B vectors","volume":"23","author":"Li","year":"2015","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2016.12.010_bib35","first-page":"2686","article-title":"Immune response hinders therapy for lysosomal storage diseases","volume":"118","author":"Ponder","year":"2008","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2016.12.010_bib36","doi-asserted-by":"crossref","first-page":"183","DOI":"10.2147\/JBM.S6885","article-title":"Treatment of hemophilia: a review of current advances and ongoing issues","volume":"1","author":"Coppola","year":"2010","journal-title":"J.\u00a0Blood Med."},{"key":"10.1016\/j.omtm.2016.12.010_bib37","first-page":"CD008185","article-title":"Enzyme replacement therapy with idursulfase for mucopolysaccharidosis type II (Hunter syndrome)","volume":"11","author":"da Silva","year":"2011","journal-title":"Cochrane Database Syst. Rev."},{"key":"10.1016\/j.omtm.2016.12.010_bib38","doi-asserted-by":"crossref","first-page":"443","DOI":"10.1016\/j.ymgme.2011.12.006","article-title":"Anti-\u03b1-galactosidase A antibody response to agalsidase beta treatment: data from the Fabry Registry","volume":"105","author":"Wilcox","year":"2012","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2016.12.010_bib39","first-page":"CD009354","article-title":"Enzyme replacement therapy with laronidase (Aldurazyme(\u00ae)) for treating mucopolysaccharidosis type I","volume":"11","author":"Jameson","year":"2013","journal-title":"Cochrane Database Syst. Rev."},{"key":"10.1016\/j.omtm.2016.12.010_bib40","doi-asserted-by":"crossref","first-page":"11854","DOI":"10.1073\/pnas.182412299","article-title":"Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy","volume":"99","author":"Gao","year":"2002","journal-title":"Proc. Natl. Acad. Sci. USA"}],"updated-by":[{"DOI":"10.1016\/j.omtm.2019.04.005","type":"erratum","label":"Erratum","source":"publisher","updated":{"date-parts":[[2019,6,14]],"date-time":"2019-06-14T00:00:00Z","timestamp":1560470400000}}],"container-title":["Molecular Therapy - Methods &amp; Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050117300049?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050117300049?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2018,9,5]],"date-time":"2018-09-05T04:54:00Z","timestamp":1536123240000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050117300049"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2017,3]]},"references-count":40,"alternative-id":["S2329050117300049"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2016.12.010","relation":{"erratum":[{"id-type":"doi","id":"10.1016\/j.omtm.2019.04.005","asserted-by":"object"}]},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2017,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"Low-Dose Liver-Targeted Gene Therapy for Pompe Disease Enhances Therapeutic Efficacy of ERT via Immune Tolerance Induction","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2016.12.010","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2017 The Author(s).","name":"copyright","label":"Copyright"}]}}