未登录 Progression of vertebral bone disease in mucopolysaccharidosis VII dogs from birth to skeletal maturity Peck, Sun H.; Lau, Yian Khai; Kang, Jennifer L.; Lin, Megan; Arginteanu, Toren; Matalon, Dena R.; Bendigo, Justin R.; O'Donnell, Patricia; Haskins, Mark E.; Casal, Margret L.; Smith, Lachlan J. 分享 收藏
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Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model Hinderer, Christian; Bell, Peter; Louboutin, Jean-Pierre; Katz, Nathan; Zhu, Yanqing; Lin, Gloria; Choa, Ruth; Bagel, Jessica; O'Donnell, Patricia; Fitzgerald, Caitlin A.; Langan, Therese; Wang, Ping; Casal, Margret L.; Haskins, Mark E.; Wilson, James M. 分享 收藏
Urinary glycosaminoglycan concentration as a biomarker for effectiveness of enzyme replacement and gene therapy in large animal models of mucopolysaccharidoses Casal, Margret L.; Wang, Ping; ODonnell, Patricia A.; Sikora, Tracey; Langan, Therese; O'Malley, Thomas; Ferla, Rita; Auricchio, Alberto; Wilson, James M.; Gao, Guangping; Ponder, Katherine; Haskins, Mark E. 分享 收藏
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Pharmacodynamics, pharmacokinetics and biodistribution of recombinant human N-acetylgalactosamine 4-sulfatase after 6 months of therapy in cats using different IV infusion durations Ruane, Therese; Haskins, Mark; Cheng, Alphonsus; Wang, Ping; Aguirre, Gustavo; Knox, Van W. (Rafe); Qi, Yulan; Tompkins, Troy; O'Neill, Charles A. 分享 收藏
Evaluation of AAV-mediated Gene Therapy for Central Nervous System Disease in Canine Mucopolysaccharidosis VII Gurda, Brittney L.; De Lataillade, Adrien De Guilhem; Bell, Peter; Zhu, Yanqing; Yu, Hongwei; Wang, Ping; Bagel, Jessica; Vite, Charles H.; Sikora, Tracey; Hinderer, Christian; Calcedo, Roberto; Yox, Alexander D.; Steet, Richard A.; Ruane, Therese; O'Donnell, Patricia; Gao, Guangping; Wilson, James M.; Casal, Margret; Ponder, Katherine P.; Haskins, Mark E. 分享 收藏
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Neonatal Systemic AAV Induces Tolerance to CNS Gene Therapy in MPS I Dogs and Nonhuman Primates Hinderer, Christian; Bell, Peter; Louboutin, Jean-Pierre; Zhu, Yanqing; Yu, Hongwei; Lin, Gloria; Choa, Ruth; Gurda, Brittney L.; Bagel, Jessica; O'Donnell, Patricia; Sikora, Tracey; Ruane, Therese; Wang, Ping; Tarantal, Alice F.; Casal, Margret L.; Haskins, Mark E.; Wilson, James M. 分享 收藏
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Pentosan polysulfate: New mechanistic insights and treatment of the mucopolysaccharidoses Simonaro, Calogera M.; Tomatsu, Shunji; Ge, Yi; Meng, Fanli; Frohbergh, Michael; Haskins, Mark E.; Ruane, Therese; Wang, Raymond Y.; Vera, Moin; Solyom, Alexander; Schuchman, Edward H. 分享 收藏
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Adeno-associated virus vector-mediated gene therapy can effectively treat CNS and cardiac lesions and induce immune tolerance to the therapeutic enzyme in large animal models of mucopolysaccharidosis type I Wilson, James M.; Hinderer, Christian; Bell, Peter; Gurda, Brittney L.; Wang, Qiang; Louboutin, Jean-Pierre; Zhu, Yanqing; Choa, Ruth; Lin, Gloria; Bagel, Jessica; O'Donnell, Patricia; Sikora, Tracey; Ruane, Therese; Wang, Ping; Haskins, Mark E. 分享 收藏
Intrathecal Gene Therapy Corrects CNS Pathology in a Feline Model of Mucopolysaccharidosis I Hinderer, Christian; Bell, Peter; Gurda, Brittney L.; Wang, Qiang; Louboutin, Jean-Pierre; Zhu, Yanqing; Bagel, Jessica; O'Donnell, Patricia; Sikora, Tracey; Ruane, Therese; Wang, Ping; Haskins, Mark E.; Wilson, James M. 分享 收藏
Liver-directed gene therapy corrects cardiovascular lesions in feline mucopolysaccharidosis type I Hinderer, Christian; Bell, Peter; Gurda, Brittney L.; Wang, Qiang; Louboutin, Jean-Pierre; Zhu, Yanqing; Bagel, Jessica; O'Donnell, Patricia; Sikora, Tracey; Ruane, Therese; Wang, Ping; Haskins, Mark E.; Wilson, James M. 分享 收藏