{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,3,12]],"date-time":"2026-03-12T00:22:21Z","timestamp":1773274941686,"version":"3.50.1"},"reference-count":37,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2013,11,1]],"date-time":"2013-11-01T00:00:00Z","timestamp":1383264000000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"}],"funder":[{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"crossref","award":["DK054481"],"award-info":[{"award-number":["DK054481"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"crossref","award":["P40 OD010939"],"award-info":[{"award-number":["P40 OD010939"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"crossref","award":["HD061879"],"award-info":[{"award-number":["HD061879"]}],"id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]},{"DOI":"10.13039\/100013927","name":"National MPS Society","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100013927","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["mgmjournal.com","elsevier.com","sciencedirect.com"],"crossmark-restriction":true},"short-container-title":["Molecular Genetics and Metabolism"],"published-print":{"date-parts":[[2013,11]]},"DOI":"10.1016\/j.ymgme.2013.06.015","type":"journal-article","created":{"date-parts":[[2013,6,25]],"date-time":"2013-06-25T13:54:28Z","timestamp":1372168468000},"page":"311-318","update-policy":"https:\/\/doi.org\/10.1016\/elsevier_cm_policy","source":"Crossref","is-referenced-by-count":12,"title":["The effect of neonatal gene therapy with a gamma retroviral vector on cardiac valve disease in mucopolysaccharidosis VII dogs after a decade"],"prefix":"10.1016","volume":"110","author":[{"given":"Paul W.","family":"Bigg","sequence":"first","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Meg M.","family":"Sleeper","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Patricia A.","family":"O'Donnell","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Yuli","family":"Liu","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Susan","family":"Wu","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Margret L.","family":"Casal","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Mark E.","family":"Haskins","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Katherine P.","family":"Ponder","sequence":"additional","affiliation":[],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"78","reference":[{"key":"10.1016\/j.ymgme.2013.06.015_bb0005","series-title":"Metabolic and Molecular Basis of Inherited Disease","first-page":"3421","article-title":"The mucopolysaccharidoses","author":"Neufeld","year":"2001"},{"key":"10.1016\/j.ymgme.2013.06.015_bb0010","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1001\/jama.281.3.249","article-title":"Prevalence of lysosomal storage disorders","volume":"281","author":"Meikle","year":"1999","journal-title":"JAMA"},{"key":"10.1016\/j.ymgme.2013.06.015_bb0015","doi-asserted-by":"crossref","first-page":"1183","DOI":"10.1007\/s10545-011-9359-8","article-title":"Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management","volume":"34","author":"Braunlin","year":"2011","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0020","doi-asserted-by":"crossref","first-page":"98","DOI":"10.1007\/BF01991908","article-title":"Mitral and aortic regurgitation in 84 patients with mucopolysaccharidoses","volume":"154","author":"Wippermann","year":"1995","journal-title":"Eur. J. Pediatr."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0025","doi-asserted-by":"crossref","first-page":"170","DOI":"10.1017\/S1047951109003576","article-title":"The natural course and the impact of therapies of cardiac involvement in the mucopolysaccharidoses","volume":"19","author":"Fesslov\u00e1","year":"2009","journal-title":"Cardiol. Young"},{"key":"10.1016\/j.ymgme.2013.06.015_bb0030","doi-asserted-by":"crossref","first-page":"534","DOI":"10.1007\/s004310050872","article-title":"Cardiovascular changes in children with mucopolysaccharide storage diseases and related disorders\u2014clinical and echocardiographic findings in 64 patients","volume":"157","author":"Dangel","year":"1998","journal-title":"Eur. J. Pediatr."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0035","doi-asserted-by":"crossref","first-page":"750","DOI":"10.1007\/s10545-007-0586-y","article-title":"Cardiac abnormalities in adults with the attenuated form of mucopolysaccharidosis type I","volume":"30","author":"Soliman","year":"2007","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0040","doi-asserted-by":"crossref","first-page":"388","DOI":"10.1016\/S0022-3476(75)80968-1","article-title":"Variation in the phenotypic expression of beta-glucuronidase deficiency","volume":"86","author":"Beaudet","year":"1975","journal-title":"J. Pediatr."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0045","doi-asserted-by":"crossref","first-page":"249","DOI":"10.1016\/S0022-3476(73)80162-3","article-title":"Beta glucuronidase deficiency: report of clinical, radiologic, and biochemical features of a new mucopolysaccharidosis","volume":"82","author":"Sly","year":"1973","journal-title":"J. Pediatr."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0050","first-page":"132","article-title":"Mucopolysaccharidosis VII: postmortem biochemical and pathological findings in a young adult with beta-glucuronidase deficiency","volume":"7","author":"Vogler","year":"1994","journal-title":"Mod. Pathol."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0055","doi-asserted-by":"crossref","first-page":"87","DOI":"10.1007\/8904_2012_208","article-title":"Cardiac ultrasound findings in infants with severe (Hurler phenotype) untreated mucopolysaccharidosis (MPS) type I","volume":"10","author":"Schroeder","year":"2013","journal-title":"JIMD Rep."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0060","doi-asserted-by":"crossref","first-page":"859","DOI":"10.1016\/j.jpeds.2008.12.024","article-title":"Characterization of surgical procedures in patients with mucopolysaccharidosis type I: findings from the MPS I Registry","volume":"154","author":"Arn","year":"2009","journal-title":"J. Pediatr."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0065","doi-asserted-by":"crossref","first-page":"861","DOI":"10.1111\/j.1399-3046.2011.01595.x","article-title":"Variable disease progression after successful stem cell transplantation: prospective follow-up investigations in eight patients with Hurler syndrome","volume":"15","author":"Grigull","year":"2011","journal-title":"Pediatr. Transplant."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0070","doi-asserted-by":"crossref","first-page":"349","DOI":"10.1017\/S1047951110002015","article-title":"Aortic stenosis in a patient with Hurler's syndrome after bone marrow transplantation","volume":"21","author":"Watanabe","year":"2011","journal-title":"Cardiol. Young"},{"key":"10.1016\/j.ymgme.2013.06.015_bb0075","doi-asserted-by":"crossref","first-page":"882","DOI":"10.1016\/S0002-9149(03)00909-3","article-title":"Usefulness of bone marrow transplantation in the Hurler syndrome","volume":"92","author":"Braunlin","year":"2003","journal-title":"Am. J. Cardiol."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0080","doi-asserted-by":"crossref","first-page":"2054","DOI":"10.2460\/ajvr.1990.51.12.2054","article-title":"Cardiovascular changes after bone marrow transplantation in dogs with mucopolysaccharidosis I","volume":"51","author":"Gompf","year":"1990","journal-title":"Am. J. Vet. Res."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0085","doi-asserted-by":"crossref","first-page":"1289","DOI":"10.1038\/sj.bmt.1702448","article-title":"Effects of bone marrow transplantation on the cardiovascular abnormalities in canine mucopolysaccharidosis VII","volume":"25","author":"Sammarco","year":"2000","journal-title":"Bone Marrow Transplant."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0090","doi-asserted-by":"crossref","first-page":"416","DOI":"10.1016\/j.amjcard.2006.02.047","article-title":"Cardiac findings after enzyme replacement therapy for mucopolysaccharidosis type I","volume":"98","author":"Braunlin","year":"2006","journal-title":"Am. J. Cardiol."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0095","doi-asserted-by":"crossref","first-page":"227","DOI":"10.1007\/s10545-011-9444-z","article-title":"Mucopolysaccharidosis: cardiologic features and effects of enzyme-replacement therapy in 24 children with MPS I, II and VI","volume":"36","author":"Brands","year":"2013","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0100","doi-asserted-by":"crossref","first-page":"171","DOI":"10.1016\/j.ymgme.2006.08.007","article-title":"A follow-up study of MPS I patients treated with laronidase enzyme replacement therapy for 6years","volume":"90","author":"Sifuentes","year":"2007","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0105","doi-asserted-by":"crossref","first-page":"385","DOI":"10.1007\/s10545-012-9481-2","article-title":"Enzyme replacement therapy for mucopolysaccharidosis VI: long-term cardiac effects of galsulfase (Naglazyme(\u00ae)) therapy","volume":"36","author":"Braunlin","year":"2013","journal-title":"J. Inherit. Metab. Dis."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0110","doi-asserted-by":"crossref","first-page":"597","DOI":"10.1016\/j.ymgme.2011.08.029","article-title":"Enzyme replacement therapy attenuates disease progression in two Japanese siblings with mucopolysaccharidosis type VI","volume":"104","author":"Furujo","year":"2011","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0115","first-page":"1","article-title":"Advantages of early replacement therapy for mucopolysaccharidosis type VI: echocardiographic follow-up of siblings","author":"Leal","year":"2013","journal-title":"Cardiol. Young"},{"key":"10.1016\/j.ymgme.2013.06.015_bb0120","doi-asserted-by":"crossref","first-page":"492","DOI":"10.1111\/j.1399-0004.2009.01324.x","article-title":"Enzyme replacement therapy for mucopolysaccharidosis VI from 8weeks of age\u2014a sibling control study","volume":"77","author":"McGill","year":"2010","journal-title":"Clin. Genet."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0125","first-page":"60ra89","article-title":"Replacing the enzyme alpha-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. Total. Environ."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0130","doi-asserted-by":"crossref","first-page":"1333","DOI":"10.1517\/14712598.7.9.1333","article-title":"Gene therapy for mucopolysaccharidosis","volume":"7","author":"Ponder","year":"2007","journal-title":"Expert. Opin. Biol. Ther."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0135","doi-asserted-by":"crossref","first-page":"13102","DOI":"10.1073\/pnas.192353499","article-title":"Therapeutic neonatal hepatic gene therapy in mucopolysaccharidosis VII dogs","volume":"99","author":"Ponder","year":"2002","journal-title":"Proc. Natl. Acad. Sci. U. S. A."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0140","doi-asserted-by":"crossref","first-page":"145","DOI":"10.1016\/j.ymgme.2012.03.013","article-title":"Effect of neonatal gene therapy on lumbar spine disease in mucopolysaccharidosis VII dogs","volume":"107","author":"Smith","year":"2012","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0145","doi-asserted-by":"crossref","first-page":"141","DOI":"10.1006\/mthe.2002.0527","article-title":"Transduction of hepatocytes after neonatal delivery of a Moloney murine leukemia virus based retroviral vector results in long-term expression of beta-glucuronidase in mucopolysaccharidosis VII dogs","volume":"5","author":"Xu","year":"2002","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0150","doi-asserted-by":"crossref","first-page":"815","DOI":"10.1161\/01.CIR.0000138747.82487.4B","article-title":"Gene therapy ameliorates cardiovascular disease in dogs with mucopolysaccharidosis VII","volume":"110","author":"Sleeper","year":"2004","journal-title":"Circulation"},{"key":"10.1016\/j.ymgme.2013.06.015_bb0155","doi-asserted-by":"crossref","first-page":"396","DOI":"10.1016\/j.ymgme.2009.12.003","article-title":"Upregulation of elastase activity in aorta in mucopolysaccharidosis I and VII dogs may be due to increased cytokine expression","volume":"99","author":"Metcalf","year":"2010","journal-title":"Mol. Genet. Metab."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0160","doi-asserted-by":"crossref","first-page":"898","DOI":"10.1038\/mt.2012.9","article-title":"Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats","volume":"20","author":"Ponder","year":"2012","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0165","doi-asserted-by":"crossref","first-page":"1423","DOI":"10.1038\/sj.mt.6300201","article-title":"Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy","volume":"15","author":"Traas","year":"2007","journal-title":"Mol. Ther."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0170","doi-asserted-by":"crossref","first-page":"248","DOI":"10.1006\/geno.1997.5189","article-title":"Cloning of the canine beta-glucuronidase cDNA, mutation identification in canine MPS VII, and retroviral vector-mediated correction of MPS VII cells","volume":"48","author":"Ray","year":"1998","journal-title":"Genomics"},{"key":"10.1016\/j.ymgme.2013.06.015_bb0175","doi-asserted-by":"crossref","first-page":"103","DOI":"10.1016\/j.jvc.2012.02.001","article-title":"Pathology of myxomatous mitral valve disease in the dog","volume":"14","author":"Fox","year":"2012","journal-title":"J. Vet. Cardiol."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0180","doi-asserted-by":"crossref","first-page":"651","DOI":"10.1016\/j.cvsm.2010.03.008","article-title":"Canine degenerative myxomatous mitral valve disease: natural history, clinical presentation and therapy","volume":"40","author":"Borgarelli","year":"2010","journal-title":"Vet. Clin. North Am. Small Anim. Pract."},{"key":"10.1016\/j.ymgme.2013.06.015_bb0185","doi-asserted-by":"crossref","first-page":"487","DOI":"10.1016\/0002-9149(76)90468-9","article-title":"The heart in the Hurler syndrome: gross, histologic and ultrastructural observations in five necropsy cases","volume":"38","author":"Renteria","year":"1976","journal-title":"Am. J. Cardiol."}],"container-title":["Molecular Genetics and Metabolism"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719213002175?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1096719213002175?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2024,5,12]],"date-time":"2024-05-12T23:00:18Z","timestamp":1715554818000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1096719213002175"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2013,11]]},"references-count":37,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2013,11]]}},"alternative-id":["S1096719213002175"],"URL":"https:\/\/doi.org\/10.1016\/j.ymgme.2013.06.015","relation":{},"ISSN":["1096-7192"],"issn-type":[{"value":"1096-7192","type":"print"}],"subject":[],"published":{"date-parts":[[2013,11]]},"assertion":[{"value":"Elsevier","name":"publisher","label":"This article is maintained by"},{"value":"The effect of neonatal gene therapy with a gamma retroviral vector on cardiac valve disease in mucopolysaccharidosis VII dogs after a decade","name":"articletitle","label":"Article Title"},{"value":"Molecular Genetics and Metabolism","name":"journaltitle","label":"Journal Title"},{"value":"https:\/\/doi.org\/10.1016\/j.ymgme.2013.06.015","name":"articlelink","label":"CrossRef DOI link to publisher maintained version"},{"value":"article","name":"content_type","label":"Content Type"},{"value":"Copyright \u00a9 2013 Elsevier Inc. All rights reserved.","name":"copyright","label":"Copyright"}]}}