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We found that hepatic gene transfer not only generated therapeutic levels of circulating enzyme, but in most cases also resulted in complete resolution of storage lesions in the cardiac valves, a tissue that is refractory to currently available therapies and responsible for much of the residual morbidity and mortality in treated patients.<\/jats:p>","DOI":"10.1073\/pnas.1413645111","type":"journal-article","created":{"date-parts":[[2014,9,30]],"date-time":"2014-09-30T11:28:38Z","timestamp":1412076518000},"page":"14894-14899","update-policy":"https:\/\/doi.org\/10.1073\/pnas.cm10313","source":"Crossref","is-referenced-by-count":41,"title":["Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I"],"prefix":"10.1073","volume":"111","author":[{"given":"Christian","family":"Hinderer","sequence":"first","affiliation":[{"name":"Gene Therapy Program, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Bell","sequence":"additional","affiliation":[{"name":"Gene Therapy Program, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brittney L.","family":"Gurda","sequence":"additional","affiliation":[{"name":"Gene Therapy Program, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qiang","family":"Wang","sequence":"additional","affiliation":[{"name":"Gene Therapy Program, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jean-Pierre","family":"Louboutin","sequence":"additional","affiliation":[{"name":"Section of Anatomy, Department of Basic Medical Sciences, Mona Campus, University of the West Indies, Kingston, Jamaica"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yanqing","family":"Zhu","sequence":"additional","affiliation":[{"name":"Gene Therapy Program, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jessica","family":"Bagel","sequence":"additional","affiliation":[{"name":"Departments of Pathobiology and Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Patricia","family":"O\u2019Donnell","sequence":"additional","affiliation":[{"name":"Departments of Pathobiology and Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tracey","family":"Sikora","sequence":"additional","affiliation":[{"name":"Departments of Pathobiology and Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Therese","family":"Ruane","sequence":"additional","affiliation":[{"name":"Departments of Pathobiology and Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ping","family":"Wang","sequence":"additional","affiliation":[{"name":"Departments of Pathobiology and Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mark E.","family":"Haskins","sequence":"additional","affiliation":[{"name":"Departments of Pathobiology and Clinical Studies, School of Veterinary Medicine, University of Pennsylvania, Philadelphia, PA 19104; and"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"James M.","family":"Wilson","sequence":"additional","affiliation":[{"name":"Gene Therapy Program, Department of Pathology and Laboratory Medicine, Perelman School of Medicine, and"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"341","published-online":{"date-parts":[[2014,9,29]]},"reference":[{"key":"e_1_3_4_1_2","doi-asserted-by":"crossref","first-page":"20130467","DOI":"10.1259\/bjr.20130467","article-title":"Radiological and clinical characterization of the lysosomal storage disorders: Non-lipid disorders","volume":"87","author":"Parker EI","year":"2014","unstructured":"EI Parker, M Xing, A Moreno-De-Luca, E Harmouche, MR Terk, Radiological and clinical characterization of the lysosomal storage disorders: Non-lipid disorders. 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