Intracerebroventricular dosing of N-sulfoglucosamine sulfohydrolase in mucopolysaccharidosis IIIA mice reduces markers of brain lysosomal dysfunction

Mucopolysaccharidosis type IIIA (MPS IIIA) is a lysosomal storage disorder caused by N-sulfoglucosamine sulfohydrolase (SGSH) deficiency. SGSH removes the sulfate from N-sulfoglucosamine residues on the nonreducing end of heparan sulfate (HS-NRE) within lysosomes. Enzyme deficiency results in accumu...

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Detalles Bibliográficos
Autores principales: Magat, Jenna, Jones, Samantha, Baridon, Brian, Agrawal, Vishal, Wong, Hio, Giaramita, Alexander, Mangini, Linley, Handyside, Britta, Vitelli, Catherine, Parker, Monica, Yeung, Natasha, Zhou, Yu, Pungor, Erno, Slabodkin, Ilya, Gorostiza, Olivia, Aguilera, Allora, Lo, Melanie J., Alcozie, Saida, Christianson, Terri M., Tiger, Pascale M.N., Vincelette, Jon, Fong, Sylvia, Gil, Geuncheol, Hague, Chuck, Lawrence, Roger, Wendt, Daniel J., Lebowitz, Jonathan H., Bunting, Stuart, Bullens, Sherry, Crawford, Brett E., Roy, Sushmita M., Woloszynek, Josh C.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society for Biochemistry and Molecular Biology 2022
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9694393/
https://www.ncbi.nlm.nih.gov/pubmed/36306823
http://dx.doi.org/10.1016/j.jbc.2022.102625

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