MilliporeSigma™ Calbiochem™ GCase Activator, NCGC00188758

Description

• A cell-permeable pyrazolopyrimidine-carboxamide compound that directly binds to glucocerebrosidase (GCase; K d ∽ 9μM for NT-467 fluorescent labeled GCase) and enhances its activity and serves as a chaperone for its translocation to the lysosomal compartment in fibroblasts of patients with Gaucher disease (AC 50 = 5.2 and 6.5μM for wild type and mutant N370S, respectively)
• Also shown to be effective in increasing GCase activity in macrophages from Gaucher's disease patients as well as in iPSC-derived macrophages (10.7 and 3.2 fold, respectively)
• Normalizes reactive oxygen species production and improves chemotaxis in Gaucher disease macrophages
• Shown to permeate the blood-brain barrier
• Exhibits desirable pharmacokinetic properties with plasma t 1/2 = 19.3 h and C max of 1.03μM (following a single injection of 50mg/kg in mice)
• Please note that the molecular weight for this compound is batch-specific due to variable water content.