{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,1,13]],"date-time":"2026-01-13T13:13:41Z","timestamp":1768310021896,"version":"3.49.0"},"reference-count":40,"publisher":"SAGE Publications","issue":"5","license":[{"start":{"date-parts":[[2001,3,20]],"date-time":"2001-03-20T00:00:00Z","timestamp":985046400000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/www.liebertpub.com\/nv\/resources-tools\/text-and-data-mining-policy\/121\/"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Human Gene Therapy"],"published-print":{"date-parts":[[2001,3,20]]},"DOI":"10.1089\/104303401300042447","type":"journal-article","created":{"date-parts":[[2002,7,26]],"date-time":"2002-07-26T18:08:56Z","timestamp":1027706936000},"page":"527-538","source":"Crossref","is-referenced-by-count":43,"title":["Intercellular Transfer of the Virally Derived Precursor Form of Acid \u03b1-Glucosidase Corrects the Enzyme Deficiency in Inherited Cardioskeletal Myopathy Pompe Disease"],"prefix":"10.1177","volume":"12","author":[{"given":"Daniel F.","family":"Pauly","sequence":"first","affiliation":[{"name":"Peter Belfer Cardiac Laboratory and Departments of Medicine, Pediatrics, and Pathology, Johns Hopkins University School of Medicine, Baltimore, MD 21287 and Gene Therapy Center and Departments of Medicine, Pediatrics, and Molecular Genetics, University of Florida College of Medicine, Gainesville, FL 32610"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Thomas J.","family":"Fraites","sequence":"additional","affiliation":[{"name":"Gene Therapy Center and Departments of Medicine, Pediatrics, and Molecular Genetics, University of Florida College of Medicine, Gainesville, FL 32610"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Catalin","family":"Toma","sequence":"additional","affiliation":[{"name":"Peter Belfer Cardiac Laboratory and Departments of Medicine, Pediatrics, and Pathology, Johns Hopkins University School of Medicine, Baltimore, MD 21287"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Helen S.","family":"Bayes","sequence":"additional","affiliation":[{"name":"Peter Belfer Cardiac Laboratory and Departments of Medicine, Pediatrics, and Pathology, Johns Hopkins University School of Medicine, Baltimore, MD 21287"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Maryann L.","family":"Huie","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, New York University Medical Center, New York, NY 10016"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Rochelle","family":"Hirschhorn","sequence":"additional","affiliation":[{"name":"Department of Internal Medicine, New York University Medical Center, New York, NY 10016"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Paul H.","family":"Plotz","sequence":"additional","affiliation":[{"name":"Arthritis and Rheumatism Branch, National Institute of Arthritis, Musculoskeletal, and Skin Diseases, National Institutes of Health, Bethesda, MD 20892"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Nina","family":"Raben","sequence":"additional","affiliation":[{"name":"Arthritis and Rheumatism Branch, National Institute of Arthritis, Musculoskeletal, and Skin Diseases, National Institutes of Health, Bethesda, MD 20892"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Paul D.","family":"Kessler","sequence":"additional","affiliation":[{"name":"Peter Belfer Cardiac Laboratory and Departments of Medicine, Pediatrics, and Pathology, Johns Hopkins University School of Medicine, Baltimore, MD 21287"}],"role":[{"role":"author","vocabulary":"crossref"}]},{"given":"Barry J.","family":"Byrne","sequence":"additional","affiliation":[{"name":"Peter Belfer Cardiac Laboratory and Departments of Medicine, Pediatrics, and Pathology, Johns Hopkins University School of Medicine, Baltimore, MD 21287 and Gene Therapy Center and Departments of Medicine, Pediatrics, and Molecular Genetics, University of Florida College of Medicine, Gainesville, FL 32610"}],"role":[{"role":"author","vocabulary":"crossref"}]}],"member":"179","reference":[{"key":"p_79","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.96.16.8861"},{"key":"p_80","first-page":"51","volume":"1","author":"BARR E.","year":"1994","journal-title":"Gene Ther."},{"key":"p_81","first-page":"1139","volume":"13","author":"BAUDHUIN P.","year":"1964","journal-title":"Lab. Inves."},{"key":"p_82","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/7.11.1815"},{"key":"p_83","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/8.12.2145"},{"key":"p_84","doi-asserted-by":"publisher","DOI":"10.1083\/jcb.101.1.85"},{"key":"p_85","first-page":"184","volume":"9","author":"DE BARSY T.","year":"1973","journal-title":"Birth Defects"},{"key":"p_86","doi-asserted-by":"crossref","first-page":"313","DOI":"10.3109\/10520298009067260","volume":"55","author":"FRANKLIN R.M.","year":"1980","journal-title":"Stain. Tech."},{"key":"p_87","doi-asserted-by":"publisher","DOI":"10.1111\/j.1432-1033.1995.903_a.x"},{"key":"p_88","doi-asserted-by":"crossref","first-page":"1842","DOI":"10.1128\/JVI.71.3.1842-1849.1997","volume":"71","author":"HARDY S.","year":"1997","journal-title":"J. Virol."},{"key":"p_89","doi-asserted-by":"crossref","first-page":"4946","DOI":"10.1016\/S0021-9258(19)85586-2","volume":"255","author":"HASILIK A.","year":"1980","journal-title":"J. Biol. Chem."},{"key":"p_90","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1042\/bj0860011","volume":"86","author":"HERS H.G.","year":"1963","journal-title":"Biochem. J."},{"key":"p_92","doi-asserted-by":"publisher","DOI":"10.1042\/bj2720485"},{"key":"p_93","doi-asserted-by":"publisher","DOI":"10.1006\/bbrc.1998.8255"},{"key":"p_94","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp.49.9.1011"},{"key":"p_95","first-page":"499","volume":"17","author":"KARPATI G.","year":"1994","journal-title":"Clin. Invest. Med."},{"key":"p_96","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199403313301308"},{"key":"p_97","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.93.24.14082"},{"key":"p_98","doi-asserted-by":"publisher","DOI":"10.1172\/JCI1722"},{"key":"p_99","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.94.8.4205"},{"key":"p_100","doi-asserted-by":"publisher","DOI":"10.1016\/0003-9861(84)90408-9"},{"key":"p_101","doi-asserted-by":"publisher","DOI":"10.1016\/0042-6822(88)90302-9"},{"key":"p_102","doi-asserted-by":"publisher","DOI":"10.1146\/annurev.bi.60.070191.001353"},{"key":"p_103","doi-asserted-by":"publisher","DOI":"10.1093\/hmg\/7.11.1695"},{"key":"p_104","doi-asserted-by":"publisher","DOI":"10.1126\/science.2360047"},{"key":"p_105","doi-asserted-by":"publisher","DOI":"10.1038\/sj.gt.3300609"},{"key":"p_106","doi-asserted-by":"publisher","DOI":"10.1074\/jbc.273.30.19086"},{"key":"p_107","doi-asserted-by":"publisher","DOI":"10.1016\/0014-4827(84)90779-1"},{"key":"p_108","doi-asserted-by":"crossref","first-page":"S61","DOI":"10.1002\/mus.880181414","volume":"3","author":"REUSER A.J.","year":"1995","journal-title":"Muscle Nerve"},{"key":"p_109","doi-asserted-by":"publisher","DOI":"10.1016\/0092-8674(92)90029-C"},{"key":"p_110","doi-asserted-by":"publisher","DOI":"10.1038\/nm0596-545"},{"key":"p_111","doi-asserted-by":"publisher","DOI":"10.1016\/0022-510X(87)90239-5"},{"key":"p_112","doi-asserted-by":"publisher","DOI":"10.1203\/00006450-198807000-00021"},{"key":"p_113","doi-asserted-by":"publisher","DOI":"10.1203\/00006450-199010000-00009"},{"key":"p_114","doi-asserted-by":"publisher","DOI":"10.1172\/JCI115025"},{"key":"p_115","doi-asserted-by":"publisher","DOI":"10.1073\/pnas.93.1.65"},{"key":"p_116","first-page":"385","volume":"314","author":"WATSON J.G.","year":"1986","journal-title":"N. Engl. J. Med."},{"key":"p_117","doi-asserted-by":"crossref","first-page":"2223","DOI":"10.1016\/S0021-9258(18)53985-5","volume":"268","author":"WISSELAAR H.A.","year":"1993","journal-title":"J. Biol. Chem."},{"key":"p_118","doi-asserted-by":"publisher","DOI":"10.1089\/hum.1997.8.13-1555"},{"key":"p_119","first-page":"635","volume":"49","author":"ZHONG N.","year":"1991","journal-title":"Am. J. Hum. Genet."}],"container-title":["Human Gene Therapy"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.liebertpub.com\/doi\/pdf\/10.1089\/104303401300042447","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,1,13]],"date-time":"2026-01-13T07:58:02Z","timestamp":1768291082000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/full\/10.1089\/104303401300042447"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2001,3,20]]},"references-count":40,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2001,3,20]]}},"alternative-id":["10.1089\/104303401300042447"],"URL":"https:\/\/doi.org\/10.1089\/104303401300042447","relation":{},"ISSN":["1043-0342","1557-7422"],"issn-type":[{"value":"1043-0342","type":"print"},{"value":"1557-7422","type":"electronic"}],"subject":[],"published":{"date-parts":[[2001,3,20]]}}}