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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...
Autores principales: | , , , , , , , , , , |
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Formato: | Texto |
Lenguaje: | English |
Publicado: |
Public Library of Science
2010
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Materias: | |
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 |