Inhibition of substrate synthesis as a strategy for glycolipid lysosomal storage disease therapy
- 1 April 2001
- journal article
- review article
- Published by Wiley in Journal of Inherited Metabolic Disease
- Vol. 24 (2) , 275-290
- https://doi.org/10.1023/a:1010335505357
Abstract
The glycosphingolipid (GSL) lysosomal storage diseases are caused by mutations in the genes encoding the glycohydrolases that catabolize GSLs within lysosomes. In these diseases the substrate for the...Keywords
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