Combining angiotensin receptor blockade and enzyme replacement therapy for vascular disease in mucopolysaccharidosis type I by Sarah C. Hurt, Moin U. Vera, Steven Q. Le, Shih-hsin Kan, Quang Bui, Patricia I. Dickson

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Genetically Corrected iPSC-Derived Neural Stem Cell Grafts Deliver Enzyme Replacement to Affect CNS Disease in Sanfilippo B Mice by Don Clarke, Yewande Pearse, Shih-hsin Kan, Steven Q. Le, Valentina Sanghez, Jonathan D. Cooper, Patricia I. Dickson, Michelina Iacovino

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Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the disease by Yewande Pearse, Don Clarke, Shih-hsin Kan, Steven Q. Le, Valentina Sanghez, Anna Luzzi, Ivy Pham, Lina R. Nih, Jonathan D. Cooper, Patricia I. Dickson, Michelina Iacovino

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A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type I by Steven Q. Le, Shih-hsin Kan, Don Clarke, Valentina Sanghez, Martin Egeland, Kristen N. Vondrak, Terence M. Doherty, Moin U. Vera, Michelina Iacovino, Jonathan D. Cooper, Mark S. Sands, Patricia I. Dickson

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A novel, long-lived, and highly engraftable immunodeficient mouse model of mucopolysaccharidosis type I by Daniel C Mendez, Alexander E Stover, Anthony D Rangel, David J Brick, Hubert E Nethercott, Marissa A Torres, Omar Khalid, Andrew MS Wong, Jonathan D Cooper, James V Jester, Edwin S Monuki, Cian McGuire, Steven Q Le, Shih-hsin Kan, Patricia I Dickson, Philip H Schwartz

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Cross-species efficacy of enzyme replacement therapy for CLN1 disease in mice and sheep by Hemanth R. Nelvagal, Samantha L. Eaton, Sophie H. Wang, Elizabeth M. Eultgen, Keigo Takahashi, Steven Q. Le, Rachel Nesbitt, Joshua T. Dearborn, Nicholas Siano, Ana C. Puhl, Patricia I. Dickson, Gerard Thompson, Fraser Murdoch, Paul M. Brennan, Mark Gray, Stephen N. Greenhalgh, Peter Tennant, Rachael Gregson, Eddie Clutton, James Nixon, Chris Proudfoot, Stefano Guido, Simon G. Lillico, C. Bruce A. Whitelaw, Jui-Yun Lu, Sandra L. Hofmann, Sean Ekins, Mark S. Sands, Thomas M. Wishart, Jonathan D. Cooper

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