{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,23]],"date-time":"2026-09-23T08:48:25Z","timestamp":1790153305971,"version":"4.0.1"},"reference-count":40,"publisher":"Elsevier BV","issue":"3","license":[{"start":{"date-parts":[[2007,3,1]],"date-time":"2007-03-01T00:00:00Z","timestamp":1172707200000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/www.elsevier.com\/tdm\/userlicense\/1.0\/"},{"start":{"date-parts":[[2016,10,7]],"date-time":"2016-10-07T00:00:00Z","timestamp":1475798400000},"content-version":"vor","delay-in-days":3508,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Molecular Therapy"],"published-print":{"date-parts":[[2007,3]]},"DOI":"10.1038\/sj.mt.6300100","type":"journal-article","created":{"date-parts":[[2007,1,23]],"date-time":"2007-01-23T15:54:00Z","timestamp":1169567640000},"page":"501-507","source":"Crossref","is-referenced-by-count":83,"title":["Physiological Correction of Pompe Disease by Systemic Delivery of Adeno-associated Virus Serotype 1 Vectors"],"prefix":"10.1016","volume":"15","author":[{"given":"Cathryn","family":"Mah","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christina A","family":"Pacak","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Kerry O","family":"Cresawn","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lara R","family":"DeRuisseau","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sean","family":"Germain","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa A","family":"Lewis","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Denise A","family":"Cloutier","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"David D","family":"Fuller","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Barry J","family":"Byrne","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"78","reference":[{"key":"10.1038\/sj.mt.6300100_bib1","first-page":"1139","article-title":"An electron microscopic and biochemical study of type II glycogenosis","volume":"13","author":"Baudhin","year":"1964","journal-title":"Lab Invest"},{"key":"10.1038\/sj.mt.6300100_bib2","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1042\/bj0860011","article-title":"Alpha-glucosidase deficiency in generalized glycogen storage disease (Pompe's disease)","volume":"86","author":"Hers","year":"1963","journal-title":"Biochem J"},{"key":"10.1038\/sj.mt.6300100_bib3","first-page":"3389","author":"Hirschhorn","year":"2000","journal-title":"Glycogen storage disease ii: acid-alpha glucosidase (acid maltase) deficiency"},{"key":"10.1038\/sj.mt.6300100_bib4","doi-asserted-by":"crossref","first-page":"145","DOI":"10.2174\/1566524024605789","article-title":"Acid alpha-glucosidase deficiency (glycogenosis type II, Pompe disease)","volume":"2","author":"Raben","year":"2002","journal-title":"Curr Mol Med"},{"key":"10.1038\/sj.mt.6300100_bib5","doi-asserted-by":"crossref","first-page":"S61","DOI":"10.1002\/mus.880181414","article-title":"Glycogenosis type II (acid maltase deficiency)","volume":"3","author":"Reuser","year":"1995","journal-title":"Muscle Nerve"},{"key":"10.1038\/sj.mt.6300100_bib6","doi-asserted-by":"crossref","first-page":"297","DOI":"10.1097\/01.gim.0000195896.04069.5f","article-title":"Electrocardiographic response to enzyme replacement therapy for Pompe disease","volume":"8","author":"Ansong","year":"2006","journal-title":"Genet Med"},{"key":"10.1038\/sj.mt.6300100_bib7","doi-asserted-by":"crossref","first-page":"289","DOI":"10.1097\/01.gim.0000204462.42910.b8","article-title":"Diagnostic challenges for Pompe disease: an under-recognized cause of floppy baby syndrome","volume":"8","author":"Howell","year":"2006","journal-title":"Genet Med"},{"key":"10.1038\/sj.mt.6300100_bib8","doi-asserted-by":"crossref","first-page":"267","DOI":"10.1097\/01.gim.0000218152.87434.f3","article-title":"Pompe disease diagnosis and management guideline","volume":"8","author":"Kishnani","year":"2006","journal-title":"Genet Med"},{"key":"10.1038\/sj.mt.6300100_bib9","doi-asserted-by":"crossref","first-page":"24","DOI":"10.1016\/j.nmd.2004.10.009","article-title":"Safety and efficacy of recombinant acid alpha-glucosidase (RhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial","volume":"15","author":"Klinge","year":"2005","journal-title":"Neuromuscul Disord"},{"key":"10.1038\/sj.mt.6300100_bib10","first-page":"1101","article-title":"Alpha-glucosidase (CHO) (genzyme)","volume":"5","author":"Lachmann","year":"2004","journal-title":"Curr Opin Invest Drugs"},{"key":"10.1038\/sj.mt.6300100_bib11","doi-asserted-by":"crossref","first-page":"e448","DOI":"10.1542\/peds.113.5.e448","article-title":"Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk","volume":"113","author":"Van den Hout","year":"2004","journal-title":"Pediatrics"},{"key":"10.1038\/sj.mt.6300100_bib12","doi-asserted-by":"crossref","first-page":"68","DOI":"10.1089\/hum.2005.16.68","article-title":"Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II","volume":"16","author":"Cresawn","year":"2005","journal-title":"Hum Gene Ther"},{"key":"10.1038\/sj.mt.6300100_bib13","doi-asserted-by":"crossref","first-page":"571","DOI":"10.1006\/mthe.2002.0580","article-title":"Correction of the enzymatic and functional deficits in a model of Pompe disease using adeno-associated virus vectors","volume":"5","author":"Fraites","year":"2002","journal-title":"Mol Ther"},{"key":"10.1038\/sj.mt.6300100_bib14","doi-asserted-by":"crossref","first-page":"1405","DOI":"10.1038\/sj.gt.3302550","article-title":"Sustained correction of glycogen storage disease type II using adeno-associated virus serotype 1 vectors","volume":"12","author":"Mah","year":"2005","journal-title":"Gene Ther"},{"key":"10.1038\/sj.mt.6300100_bib15","doi-asserted-by":"crossref","first-page":"3007","DOI":"10.1242\/dev.01169","article-title":"Rescue of enzyme deficiency in embryonic diaphragm in a mouse model of metabolic myopathy: Pompe disease","volume":"131","author":"Rucker","year":"2004","journal-title":"Development"},{"key":"10.1038\/sj.mt.6300100_bib16","doi-asserted-by":"crossref","first-page":"889","DOI":"10.1016\/j.ymthe.2005.01.012","article-title":"Correction of glycogen storage disease type II by an adeno-associated virus vector containing a muscle-specific promoter","volume":"11","author":"Sun","year":"2005","journal-title":"Mol Ther"},{"key":"10.1038\/sj.mt.6300100_bib17","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":"10.1038\/sj.mt.6300100_bib18","doi-asserted-by":"crossref","first-page":"285","DOI":"10.2174\/1566523054065057","article-title":"New recombinant serotypes of AAV vectors","volume":"5","author":"Gao","year":"2005","journal-title":"Curr Gene Ther"},{"key":"10.1038\/sj.mt.6300100_bib19","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"},{"key":"10.1038\/sj.mt.6300100_bib20","doi-asserted-by":"crossref","first-page":"791","DOI":"10.1128\/JVI.76.2.791-801.2002","article-title":"Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity","volume":"76","author":"Rabinowitz","year":"2002","journal-title":"J Virol"},{"key":"10.1038\/sj.mt.6300100_bib21","doi-asserted-by":"crossref","first-page":"3758","DOI":"10.1073\/pnas.0508917103","article-title":"Body-wide gene therapy of duchenne muscular dystrophy in the Mdx mouse model","volume":"103","author":"Denti","year":"2006","journal-title":"Proc Natl Acad Sci USA"},{"key":"10.1038\/sj.mt.6300100_bib22","doi-asserted-by":"crossref","first-page":"828","DOI":"10.1038\/nm1085","article-title":"Systemic delivery of genes to striated muscles using adeno-associated viral vectors","volume":"10","author":"Gregorevic","year":"2004","journal-title":"Nat Med"},{"key":"10.1038\/sj.mt.6300100_bib23","series-title":"Profound cardiac and whole-body functional recovery in heart failure\/muscular dystrophy hamsters by systemic delivery of AAV8 vectors via I.V. or I.P. route. Xth Parvovirus Workshop","author":"Zhu","year":"2004"},{"key":"10.1038\/sj.mt.6300100_bib24","doi-asserted-by":"crossref","first-page":"19086","DOI":"10.1074\/jbc.273.30.19086","article-title":"Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II","volume":"273","author":"Raben","year":"1998","journal-title":"J Biol Chem"},{"key":"10.1038\/sj.mt.6300100_bib25","doi-asserted-by":"crossref","first-page":"25","DOI":"10.1007\/BF02265613","article-title":"The conduction system in Pompe's disease","volume":"2","author":"Bharati","year":"1982","journal-title":"Pediatr Cardiol"},{"key":"10.1038\/sj.mt.6300100_bib26","first-page":"622","article-title":"Electrophysiological mechanism of the short PR interval in Pompe disease","volume":"128","author":"Gillette","year":"1974","journal-title":"Am J Dis Child"},{"key":"10.1038\/sj.mt.6300100_bib27","doi-asserted-by":"crossref","first-page":"601","DOI":"10.1016\/S1525-0016(02)90716-1","article-title":"Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme","volume":"6","author":"Raben","year":"2002","journal-title":"Mol Ther"},{"key":"10.1038\/sj.mt.6300100_bib28","doi-asserted-by":"crossref","first-page":"149","DOI":"10.1007\/s10334-004-0066-4","article-title":"Current status of cardiac MRI in small animals","volume":"17","author":"Vallee","year":"2004","journal-title":"MAGMA"},{"key":"10.1038\/sj.mt.6300100_bib29","doi-asserted-by":"crossref","first-page":"H652","DOI":"10.1152\/ajpheart.2000.278.2.H652","article-title":"Developmental changes of cardiac function and mass assessed with MRI in neonatal, juvenile, and adult mice","volume":"278","author":"Wiesmann","year":"2000","journal-title":"Am J Physiol Heart Circ Physiol"},{"key":"10.1038\/sj.mt.6300100_bib30","doi-asserted-by":"crossref","first-page":"581","DOI":"10.1212\/01.wnl.0000198776.53007.2c","article-title":"Course of disability and respiratory function in untreated late-onset Pompe disease","volume":"66","author":"Hagemans","year":"2006","journal-title":"Neurology"},{"key":"10.1038\/sj.mt.6300100_bib31","doi-asserted-by":"crossref","first-page":"1465","DOI":"10.1212\/01.WNL.0000158682.85052.C0","article-title":"Respiratory failure in Pompe disease: treatment with noninvasive ventilation","volume":"64","author":"Mellies","year":"2005","journal-title":"Neurology"},{"key":"10.1038\/sj.mt.6300100_bib32","doi-asserted-by":"crossref","first-page":"1","DOI":"10.1016\/S1056-8719(02)00191-0","article-title":"Pulmonary function assessment by whole-body plethysmography in restrained versus unrestrained mice","volume":"47","author":"DeLorme","year":"2002","journal-title":"J Pharmacol Toxicol Methods"},{"key":"10.1038\/sj.mt.6300100_bib33","first-page":"11","article-title":"Ultrastructure of the atrioventricular junctional area in the heart of Molossus Molossus Pallas 1766 (Chiroptera: Molossidae)","volume":"82","author":"Da Silva","year":"1998","journal-title":"Morphologie"},{"key":"10.1038\/sj.mt.6300100_bib34","doi-asserted-by":"crossref","first-page":"1024","DOI":"10.1183\/09031936.05.00020005","article-title":"Respiratory insufficiency and limb muscle weakness in adults with Pompe's disease","volume":"26","author":"Pellegrini","year":"2005","journal-title":"Eur Respir J"},{"key":"10.1038\/sj.mt.6300100_bib35","doi-asserted-by":"crossref","first-page":"412","DOI":"10.1097\/00005072-197107000-00008","article-title":"Nervous system in Pompe's disease. Ultrastructure and biochemistry","volume":"30","author":"Gambetti","year":"1971","journal-title":"J Neuropathol Exp Neurol"},{"key":"10.1038\/sj.mt.6300100_bib36","doi-asserted-by":"crossref","first-page":"85","DOI":"10.1097\/00005072-196501000-00008","article-title":"Pompe's disease (diffuse glycogenosis) with neuronal storage","volume":"24","author":"Mancall","year":"1965","journal-title":"J Neuropathol Exp Neurol"},{"key":"10.1038\/sj.mt.6300100_bib37","doi-asserted-by":"crossref","first-page":"158","DOI":"10.1016\/S1046-2023(02)00220-7","article-title":"Production and purification of serotype 1, 2, and 5 recombinant adeno-associated viral vectors","volume":"28","author":"Zolotukhin","year":"2002","journal-title":"Methods"},{"key":"10.1038\/sj.mt.6300100_bib38","first-page":"328","article-title":"Percutaneous intravenous injection in neonatal mice","volume":"49","author":"Sands","year":"1999","journal-title":"Lab Anim Sci"},{"key":"10.1038\/sj.mt.6300100_bib39","doi-asserted-by":"crossref","first-page":"81","DOI":"10.1542\/peds.16.1.81","article-title":"A barometric method for measuring ventilation in newborn infants","volume":"16","author":"Drorbaugh","year":"1955","journal-title":"Pediatrics"},{"key":"10.1038\/sj.mt.6300100_bib40","author":"DeRuisseau","year":"2007","journal-title":"Neural deficits contribute to respiratory insufficiency in a mouse model of Pompe disease"}],"container-title":["Molecular Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616313090?httpAccept=text\/plain","content-type":"text\/plain","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/api.elsevier.com\/content\/article\/PII:S1525001616313090?httpAccept=text\/xml","content-type":"text\/xml","content-version":"vor","intended-application":"text-mining"}],"deposited":{"date-parts":[[2023,5,10]],"date-time":"2023-05-10T13:53:32Z","timestamp":1683726812000},"score":1,"resource":{"primary":{"URL":"https:\/\/linkinghub.elsevier.com\/retrieve\/pii\/S1525001616313090"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2007,3]]},"references-count":40,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2007,3]]}},"alternative-id":["S1525001616313090"],"URL":"https:\/\/doi.org\/10.1038\/sj.mt.6300100","relation":{},"ISSN":["1525-0016"],"issn-type":[{"value":"1525-0016","type":"print"}],"subject":[],"published":{"date-parts":[[2007,3]]}}}