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The Journal of Biological Chemistry|October 28, 2022
Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunctionJenna Magat, Samantha Jones, Brian Baridon, et al.Proceedings of the National Academy of Sciences of the United States of America|October 1, 2014
Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIBShih-Hsin Kan, Mika Aoyagi-Scharber, Steven Q Le, et al.Biorxiv : the Preprint Server for Biology|October 1, 2025
A Common PD-Risk <i>GBA1</i> Variant Disrupts LIMP2 Interaction, Impairs Glucocerebrosidase Function, and Drives Lysosomal and Mitochondrial DysfunctionOliver B Davis, Jennifer E Kung, Sonnet S Davis, et al.The Journal of Pharmacology and Experimental Therapeutics|June 18, 2022
Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIBN Matthew Ellinwood, Bethann N Valentine, Andrew S Hess, et al.Pageof 6