Title : Substrate Reduction Therapy for Sandhoff Disease through Inhibition of Glucosylceramide Synthase Activity.

Pub. Date : 2019 Aug 7

PMID : 31208914






1 Functional Relationships(s)
Download
Sentence
Compound Name
Protein Name
Organism
1 Here, we evaluated the potential of substrate reduction therapy (SRT) using an inhibitor of glucosylceramide synthase (GCS) to decrease the synthesis of glucosylceramide (GL1) and related glycosphingolipids. Glucosylceramides UDP-glucose ceramide glucosyltransferase Mus musculus