{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2025,11,1]],"date-time":"2025-11-01T13:48:40Z","timestamp":1762004920592},"reference-count":25,"publisher":"Elsevier BV","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":[[2017,10,3]],"date-time":"2017-10-03T00:00:00Z","timestamp":1506988800000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"name":"Eunice Kennedy Shriver Institute for Child Health and Human Development"},{"DOI":"10.13039\/100000065","name":"National Institute for Neurological Disorders and Stroke","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100000065","id-type":"DOI","asserted-by":"crossref"}]},{"name":"NIH","award":["R03 HD074907","R01 NS085381","R01\u00a0NS043205"],"award-info":[{"award-number":["R03 HD074907","R01 NS085381","R01\u00a0NS043205"]}]},{"DOI":"10.13039\/100000968","name":"American Heart Association","doi-asserted-by":"crossref","award":["12SDG9260007"],"award-info":[{"award-number":["12SDG9260007"]}],"id":[{"id":"10.13039\/100000968","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Los Angeles Biomedical Research Institute and the Department of Pediatrics at Harbor-UCLA Medical Center"},{"name":"UCLA Medical Genetics Training Program","award":["GM008243"],"award-info":[{"award-number":["GM008243"]}]}],"content-domain":{"domain":["cell.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Therapy - Methods & Clinical Development"],"published-print":{"date-parts":[[2018,3]]},"DOI":"10.1016\/j.omtm.2017.09.008","type":"journal-article","created":{"date-parts":[[2017,10,11]],"date-time":"2017-10-11T14:16:07Z","timestamp":1507731367000},"page":"42-51","update-policy":"http:\/\/dx.doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":15,"special_numbering":"C","title":["A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I"],"prefix":"10.1016","volume":"8","author":[{"given":"Steven Q.","family":"Le","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Shih-hsin","family":"Kan","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Don","family":"Clarke","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Valentina","family":"Sanghez","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Martin","family":"Egeland","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Kristen N.","family":"Vondrak","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Terence M.","family":"Doherty","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Moin U.","family":"Vera","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Michelina","family":"Iacovino","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Jonathan D.","family":"Cooper","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mark S.","family":"Sands","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Patricia I.","family":"Dickson","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.omtm.2017.09.008_bib1","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.2017.09.008_bib2","doi-asserted-by":"crossref","first-page":"847","DOI":"10.1016\/j.jpeds.2013.03.002","article-title":"B-Cell depletion and immunomodulation before initiation of enzyme replacement therapy blocks the immune response to acid alpha-glucosidase in infantile-onset Pompe disease","volume":"163","author":"Elder","year":"2013","journal-title":"J.\u00a0Pediatr."},{"key":"10.1016\/j.omtm.2017.09.008_bib3","doi-asserted-by":"crossref","first-page":"13","DOI":"10.1016\/j.ymgme.2008.10.009","article-title":"A dose-optimization trial of laronidase (Aldurazyme) in patients with mucopolysaccharidosis I","volume":"96","author":"Giugliani","year":"2009","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2017.09.008_bib4","doi-asserted-by":"crossref","first-page":"901","DOI":"10.1038\/nbt.1484","article-title":"Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment","volume":"26","author":"Wang","year":"2008","journal-title":"Nat. Biotechnol."},{"key":"10.1016\/j.omtm.2017.09.008_bib5","first-page":"2868","article-title":"Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I","volume":"118","author":"Dickson","year":"2008","journal-title":"J.\u00a0Clin. Invest."},{"key":"10.1016\/j.omtm.2017.09.008_bib6","doi-asserted-by":"crossref","first-page":"43160","DOI":"10.1074\/jbc.M107778200","article-title":"Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated \u03b2-glucuronidase in the murine model of mucopolysaccharidosis VII","volume":"276","author":"Sands","year":"2001","journal-title":"J.\u00a0Biol. Chem."},{"key":"10.1016\/j.omtm.2017.09.008_bib7","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1016\/j.ymgme.2012.02.003","article-title":"Specific antibody titer alters the effectiveness of intrathecal enzyme replacement therapy in canine mucopolysaccharidosis I","volume":"106","author":"Dickson","year":"2012","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2017.09.008_bib8","doi-asserted-by":"crossref","first-page":"391","DOI":"10.1002\/bab.52","article-title":"Biochemical characterization of fluorescent-labeled recombinant human alpha-L-iduronidase in\u00a0vitro","volume":"58","author":"Tippin","year":"2011","journal-title":"Biotechnol. Appl. Biochem."},{"key":"10.1016\/j.omtm.2017.09.008_bib9","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1006\/mgme.2000.2979","article-title":"Enzyme replacement therapy in mucopolysaccharidosis I: altered distribution and targeting of alpha-L-iduronidase in immunized rats","volume":"69","author":"Turner","year":"2000","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2017.09.008_bib10","doi-asserted-by":"crossref","first-page":"30","DOI":"10.1159\/000448968","article-title":"Long term treatment with enzyme replacement therapy in patients with Fabry disease","volume":"134","author":"Oder","year":"2016","journal-title":"Nephron"},{"key":"10.1016\/j.omtm.2017.09.008_bib11","doi-asserted-by":"crossref","first-page":"912","DOI":"10.1038\/gim.2015.6","article-title":"CRIM-negative infantile Pompe disease: characterization of immune responses in patients treated with ERT monotherapy","volume":"17","author":"Berrier","year":"2015","journal-title":"Genet. Med."},{"key":"10.1016\/j.omtm.2017.09.008_bib12","doi-asserted-by":"crossref","first-page":"36182","DOI":"10.1038\/srep36182","article-title":"Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients","volume":"6","author":"Masat","year":"2016","journal-title":"Sci. Rep."},{"key":"10.1016\/j.omtm.2017.09.008_bib13","doi-asserted-by":"crossref","first-page":"1924","DOI":"10.1038\/mt.2012.133","article-title":"Administration of anti-CD3 antibodies modulates the immune response to an infusion of \u03b1-glucosidase in mice","volume":"20","author":"Ohashi","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.omtm.2017.09.008_bib14","doi-asserted-by":"crossref","first-page":"66","DOI":"10.1016\/j.ymgme.2015.11.001","article-title":"Immune response to enzyme replacement therapies in lysosomal storage diseases and the role of immune tolerance induction","volume":"117","author":"Kishnani","year":"2016","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.omtm.2017.09.008_bib15","doi-asserted-by":"crossref","first-page":"1589","DOI":"10.1111\/j.1523-1755.2004.00924.x","article-title":"Enzyme therapy for Fabry disease: neutralizing antibodies toward agalsidase alpha and beta","volume":"66","author":"Linthorst","year":"2004","journal-title":"Kidney Int."},{"key":"10.1016\/j.omtm.2017.09.008_bib16","doi-asserted-by":"crossref","first-page":"229","DOI":"10.1542\/peds.2007-3847","article-title":"Long-term efficacy and safety of laronidase in the treatment of mucopolysaccharidosis I","volume":"123","author":"Clarke","year":"2009","journal-title":"Pediatrics"},{"key":"10.1016\/j.omtm.2017.09.008_bib17","doi-asserted-by":"crossref","first-page":"41","DOI":"10.1016\/j.jim.2016.10.006","article-title":"Antibodies that neutralize cellular\u00a0uptake of elosulfase alfa are not associated with reduced efficacy or pharmacodynamic effect in individuals with Morquio A syndrome","volume":"440","author":"Melton","year":"2017","journal-title":"J.\u00a0Immunol. Methods"},{"key":"10.1016\/j.omtm.2017.09.008_bib18","doi-asserted-by":"crossref","first-page":"499","DOI":"10.1007\/s10545-016-9917-1","article-title":"The impact of the immune system on the safety and efficiency of enzyme replacement therapy in lysosomal storage disorders","volume":"39","author":"Broomfield","year":"2016","journal-title":"J.\u00a0Inherit. Metab. Dis."},{"key":"10.1016\/j.omtm.2017.09.008_bib19","doi-asserted-by":"crossref","first-page":"482","DOI":"10.1016\/j.it.2007.07.011","article-title":"Immunogenicity of protein therapeutics","volume":"28","author":"De Groot","year":"2007","journal-title":"Trends Immunol."},{"key":"10.1016\/j.omtm.2017.09.008_bib20","doi-asserted-by":"crossref","first-page":"119","DOI":"10.1038\/ni.2511","article-title":"Innate B cell helpers reveal novel types of antibody responses","volume":"14","author":"Vinuesa","year":"2013","journal-title":"Nat. Immunol."},{"key":"10.1016\/j.omtm.2017.09.008_bib21","doi-asserted-by":"crossref","first-page":"503","DOI":"10.1093\/hmg\/6.4.503","article-title":"Murine mucopolysaccharidosis type I: targeted disruption of the murine alpha-L-iduronidase gene","volume":"6","author":"Clarke","year":"1997","journal-title":"Hum. Mol. Genet."},{"key":"10.1016\/j.omtm.2017.09.008_bib22","doi-asserted-by":"crossref","first-page":"60ra89","DOI":"10.1126\/scitranslmed.3001380","article-title":"Replacing the enzyme \u03b1-l-iduronidase at birth ameliorates symptoms in the brain and periphery of dogs with mucopolysaccharidosis type I","volume":"2","author":"Dierenfeld","year":"2010","journal-title":"Sci. Transl. Med."},{"key":"10.1016\/j.omtm.2017.09.008_bib23","doi-asserted-by":"crossref","first-page":"156","DOI":"10.1006\/bmme.1996.0044","article-title":"Long-term and high-dose trials of enzyme replacement therapy in the canine model of mucopolysaccharidosis I","volume":"58","author":"Kakkis","year":"1996","journal-title":"Biochem. Mol. Med."},{"key":"10.1016\/j.omtm.2017.09.008_bib24","first-page":"17.9.1","article-title":"Direct chemical analysis of glycoconjugates for carbohydrates","volume":"32","author":"Manzi","year":"2001","journal-title":"Curr. Protoc. Mol. Biol."},{"key":"10.1016\/j.omtm.2017.09.008_bib25","doi-asserted-by":"crossref","first-page":"14.1.1","DOI":"10.1002\/0471142735.im1401s83","article-title":"The isolation and characterization of murine macrophages","volume":"83","author":"Zhang","year":"2008","journal-title":"Curr. Protoc. Immunol."}],"container-title":["Molecular Therapy - Methods & Clinical Development"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050117301079?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S2329050117301079?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2022,8,4]],"date-time":"2022-08-04T08:07:29Z","timestamp":1659600449000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S2329050117301079"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2018,3]]},"references-count":25,"alternative-id":["S2329050117301079"],"URL":"https:\/\/doi.org\/10.1016\/j.omtm.2017.09.008","relation":{},"ISSN":["2329-0501"],"issn-type":[{"value":"2329-0501","type":"print"}],"subject":[],"published":{"date-parts":[[2018,3]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I","name":"articletitle","label":"Article Title"},{"value":"Molecular Therapy - Methods & Clinical Development","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.omtm.2017.09.008","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"\u00a9 2017 The Authors.","name":"copyright","label":"Copyright"}]}}