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Molecular Genetics and Metabolism|April 11, 2021
Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndromeShih-Hsin Kan, Ibrahim Elsharkawi, Steven Q Le, et al.
The Biochemical Journal|November 26, 2013
Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblastsShih-hsin Kan, Larisa A Troitskaya, Carolyn S Sinow, et al.
Molecular Therapy. Methods & Clinical Development|November 24, 2022
Brain transplantation of genetically corrected Sanfilippo type B neural stem cells induces partial cross-correction of the diseaseYewande Pearse, Don Clarke, Shih-Hsin Kan, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 12, 2026
First-in-human intracisternal dosing of RGX-111 in severe MPS I is well-tolerated and generates sustained neurodevelopment without HSCTRaymond Y Wang, Nina Movsesyan, Shih-Hsin Kan, et al.
Advances in Cell and Gene Therapy|August 29, 2025
Intra-Articular AAV9 α-l-Iduronidase Gene Replacement in the Canine Model of Mucopolysaccharidosis Type IRaymond Yu-Jeang Wang, Shih-Hsin Kan, Haoyue Zhang, et al.
Disease Models & Mechanisms|February 2, 2026
Longitudinal characterization of Gaac.1826dupA mouse reveals cardiac, myopathic, biochemical phenotypes of Pompe diseaseJerry F Harb, Shih-Hsin Kan, Chloe L Christensen, et al.
Molecular Pharmaceutics|December 15, 2020
Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-N-Acetylglucosamine-6-Sulfatase in Neonatal MiceFeng Wang, Derek R Moen, Chelsee Sauni, et al.
Protein Science : a Publication of the Protein Society|November 12, 2021
Discovery of antimicrobial agent targeting tryptophan synthaseYuliana K Bosken, Rizi Ai, Eduardo Hilario, et al.
Molecular Therapy. Methods & Clinical Development|November 22, 2017
A Humoral Immune Response Alters the Distribution of Enzyme Replacement Therapy in Murine Mucopolysaccharidosis Type ISteven Q Le, Shih-Hsin Kan, Don Clarke, et al.
Molecular Genetics and Metabolism|June 22, 2014
Intra-articular enzyme replacement therapy with rhIDUA is safe, well-tolerated, and reduces articular GAG storage in the canine model of mucopolysaccharidosis type IRaymond Y Wang, Afshin Aminian, Michael F McEntee, et al.
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