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Substrate Reduction Augments the Efficacy of Enzyme Therapy in a Mouse Model of Fabry Disease

Fabry disease is an X-linked glycosphingolipid storage disorder caused by a deficiency in the activity of the lysosomal hydrolase α-galactosidase A (α-gal). This deficiency results in accumulation of the glycosphingolipid globotriaosylceramide (GL-3) in lysosomes. Endothelial cell storage of GL-3 fr...

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Detalles Bibliográficos
Autores principales:	Marshall, John, Ashe, Karen M., Bangari, Dinesh, McEachern, KerryAnne, Chuang, Wei-Lien, Pacheco, Joshua, Copeland, Diane P., Desnick, Robert J., Shayman, James A., Scheule, Ronald K., Cheng, Seng H.
Formato:	Texto
Lenguaje:	English
Publicado:	Public Library of Science 2010
Materias:	Research Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC2991350/ https://www.ncbi.nlm.nih.gov/pubmed/21124789 http://dx.doi.org/10.1371/journal.pone.0015033

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