Isofagomine Tartrate

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].

References:

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