Title : Iminosugar-based inhibitors of glucosylceramide synthase increase brain glycosphingolipids and survival in a mouse model of Sandhoff disease.

Pub. Date : 2011

PMID : 21738789






1 Functional Relationships(s)
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1 A potential approach is substrate reduction therapy using inhibitors of glucosylceramide synthase (GCS) to decrease the synthesis of glucosylceramide and related glycosphingolipids that accumulate in the lysosomes. Glucosylceramides UDP-glucose ceramide glucosyltransferase Mus musculus