Eliglustat is a potent and selective inhibitor of glucosylceramide synthase with an IC50 value of 40 nM for the reduction of glucosylceramide synthesis in K562 cells.
CAS：928659-70-5
分子式：C23H36N2O4.1/2C4H6O6
分子量：479.6
纯度：98%
存储：Store at -20°C

Background:

Eliglustat is a potent and selective inhibitor of glucosylceramide synthase with an IC50 value of 40 nM for the reduction of glucosylceramide synthesis in K562 cells. Inhibition of glucosylceramide synthase, the rate-limiting enzyme in glycosphingolipid synthesis, also reduces production of cell surface gangliosides GM1 and GM3 on K562 and B16/F10 cells, with IC50 values of 24 and 29 nM, respectively. Eliglustat is specific to glucosylceramide synthase over similar enzymes, having minimal activity (IC50 = >10 μM) at α-glucosidase I and II, lysosomal glucocerebrosidase (GBA1), non-lysosomal glucosylceramidase (GBA2), sucrase, and maltase. In vivo, eliglustat induces a dose-dependent decrease in glucosylceramide accumulation in the liver and lungs of D409V/null mice, a model of Gaucher disease. It also prevents further glucosylceramide accumulation if administered after liver and lung deposits have been established.