Substrate reduction augments the efficacy of enzyme therapy in a mouse model of Fabry disease.

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...

Full description

Bibliographic Details
Main Authors: John Marshall, Karen M Ashe, Dinesh Bangari, KerryAnne McEachern, Wei-Lien Chuang, Joshua Pacheco, Diane P Copeland, Robert J Desnick, James A Shayman, Ronald K Scheule, Seng H Cheng
Format: Article
Language:English
Published: Public Library of Science (PLoS) 2010-11-01
Series:PLoS ONE
Online Access:http://europepmc.org/articles/PMC2991350?pdf=render

Similar Items

Similar Items