Isofagomine tartrate
Glucocerebrosidase activator and chaperone / A competitive inhibitor of human lysosomal β-glucosidase, IC50 = 0.06 μM.1 Increases the activity of N370S mutant acid β-glucosidase (GlcCerase) in Gaucher fibroblasts.2 Displays a direct pharmacological chaperone effect greatly increasing the amount of newly synthesized GlcCerase that traffics out of the ER.2
Biochemicals & reagents
957230-65-8
1) Kuriyama et al. (2008), In vitro inhibition of glycogen-degrading enzymes and glycosidases by six-membered sugar mimics and their evaluation in cell culture; Bioorg. Med. Chem., 16 7330 2) Steet et al. (2006), The iminosugar isofagomine increases the activity of N370S mutant acid beta-glucosidase in Gaucher fibroblasts by several mechanisms; Proc. Natl. Acad. Sci. USA, 103 13813
-20°C
TARGET: Glycosylation; Chaperone -- PATHWAY: Carbohydrate metabolism -- RESEARCH AREA: Neuroscience; Vesicles