Generation of two induced pluripotent stem cell lines (CHOCi002-A and CHOCi003-A) from Pompe disease patients with compound heterozygous mutations in the GAA gene by Chloe Christensen, Perla Heckman, Allisandra Rha, Shih-Hsin Kan, Jerry Harb, Raymond Wang

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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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Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells by Chloe L. Christensen, Shih-Hsin Kan, Perla Andrade-Heckman, Allisandra K. Rha, Jerry F. Harb, Raymond Y. Wang

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Generation of an infantile GM1 gangliosidosis induced pluripotent stem cell line (CHOCi005-A) for disease modeling and therapeutic testing by Allisandra K. Rha, Chloe L. Christensen, Shih-Hsin Kan, Jerry F. Harb, Perla Andrade-Heckman, Raymond Y. Wang

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Antibodies to recombinant human alpha-L-iduronidase prevent disease correction in cortical bone in MPS I mice by Sarah C. Hurt, Steven Q. Le, Shih-hsin Kan, Quang D. Bui, Michael D. Brodt, Patricia I. Dickson

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Human iPSC-derived neural stem cells engraft and improve pathophysiology of MPS I mice by Caitlin C. Calhoun, Shih-Hsin Kan, Alexander E. Stover, Jerry F. Harb, Edwin S. Monuki, Raymond Y. Wang, Philip H. Schwartz

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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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CRISPR-mediated generation and characterization of a Gaa homozygous c.1935C>A (p.D645E) Pompe disease knock-in mouse model recapitulating human infantile onset-Pompe disease by Shih-hsin Kan, Jeffrey Y. Huang, Jerry Harb, Allisandra Rha, Nancy D. Dalton, Chloe Christensen, Yunghang Chan, Jeremy Davis-Turak, Jonathan Neumann, Raymond Y. Wang

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Base editing corrects the common Salla disease SLC17A5 c.115C>T variant by Jerry F. Harb, Chloe L. Christensen, Shih-Hsin Kan, Allisandra K. Rha, Perla Andrade-Heckman, Laura Pollard, Richard Steet, Jeffrey Y. Huang, Raymond Y. Wang

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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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