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 (AC₅₀

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 (AC₅₀ = 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.

Specifications

• Color: Light Orange
• Description: 98% by HPLC
• Target Proteins: Glucocerebrosidase (Gcase)
• Molecular Formula: C₁₇H₁₄N₄O
• Solubility: DMSO
• Content And Storage: Protect from light
• Form: Powder
• Quantity: 10 mg