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Gene therapy for lysosomal storage disorders

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Pages 857-867 | Published online: 23 Feb 2005

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Marta A Tarczyluk & Jonathan D Cooper. (2015) Investigative and emerging treatments for Batten disease. Expert Opinion on Orphan Drugs 3:9, pages 1031-1045.
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Christopher J. Folts, Nicole Scott-Hewitt, Christoph Pröschel, Margot Mayer-Pröschel & Mark Noble. (2016) Lysosomal Re-acidification Prevents Lysosphingolipid-Induced Lysosomal Impairment and Cellular Toxicity. PLOS Biology 14:12, pages e1002583.
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Kim M. Hemsley & John J. Hopwood. (2010) Lessons learnt from animal models: pathophysiology of neuropathic lysosomal storage disorders. Journal of Inherited Metabolic Disease 33:4, pages 363-371.
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Kerry Anne McEachern, Jennifer B. Nietupski, Wei-Lien Chuang, Donna Armentano, Jennifer Johnson, Elizabeth Hutto, Gregory A. Grabowski, Seng H. Cheng & John Marshall. (2006) AAV8-mediated expression of glucocerebrosidase ameliorates the storage pathology in the visceral organs of a mouse model of Gaucher disease. The Journal of Gene Medicine 8:6, pages 719-729.
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Ronald J. Mandel, Fredric P. Manfredsson, Kevin D. Foust, Aaron Rising, Sharon Reimsnider, Kevin Nash & Corinna Burger. (2006) Recombinant adeno-associated viral vectors as therapeutic agents to treat neurological disorders. Molecular Therapy 13:3, pages 463-483.
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Eun Young Kim, Young Bin Hong, Zhennan Lai, Youl-Hee Cho, Roscoe O. Brady & Sung-Chul Jung. (2005) Long-term expression of the human glucocerebrosidase genein vivo after transplantation of bone-marrow-derived cells transformed with a lentivirus vector. The Journal of Gene Medicine 7:7, pages 878-887.
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Young Bin Hong, Eun Young Kim, Han-Wook Yoo & Sung-Chul Jung. (2004) Feasibility of gene therapy in Gaucher disease using an adeno-associated virus vector. Journal of Human Genetics 49:10, pages 536-543.
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Eun Young Kim, Young Bin Hong, Zhennan Lai, Hyon J Kim, Youl-Hee Cho, Roscoe O Brady & Sung-Chul Jung. (2004) Expression and secretion of human glucocerebrosidase mediated by recombinant lentivirus vectors in vitro and in vivo: implications for gene therapy of Gaucher disease. Biochemical and Biophysical Research Communications 318:2, pages 381-390.
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N. Matthew Ellinwood, Charles H. Vite & Mark E. Haskins. (2004) Gene therapy for lysosomal storage diseases: the lessons and promise of animal models. The Journal of Gene Medicine 6:5, pages 481-506.
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Frances M. Platt, Mylvaganam Jeyakumar, Ulrika Andersson, Tanya Heare, Raymond A. Dwek & Terry D. Butters. (2003) Substrate reduction therapy in mouse models of the glycosphingolipidoses. Philosophical Transactions of the Royal Society of London. Series B: Biological Sciences 358:1433, pages 947-954.
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Aubrey D.N.J de Grey. (2002) Bioremediation meets biomedicine: therapeutic translation of microbial catabolism to the lysosome. Trends in Biotechnology 20:11, pages 452-455.
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M. Jeyakumar, T. D. Butters, R. A. Dwek & F. M. Platt. (2002) Glycosphingolipid lysosomal storage diseases: therapy and pathogenesis. Neuropathology and Applied Neurobiology 28:5, pages 343-357.
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John Marshall, Kerry Anne McEachern, Julie A.Cavanagh Kyros, Jennifer B Nietupski, Tracey L Budzinski, Robin J Ziegler, Nelson S Yew, Jennifer Sullivan, Abraham Scaria, Nico van Rooijen, John A Barranger & Seng H Cheng. (2002) Demonstration of Feasibility of In Vivo Gene Therapy for Gaucher Disease Using a Chemically Induced Mouse Model. Molecular Therapy 6:2, pages 179-189.
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