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ML-198 is a chemical chaperone of glucocerebrosidase.1 It activates hydrolysis of the fluorescent glucocerebrosidase substrate 4-methylumbelliferyl-β-D-glucopyranoside (4-MUG; Item No. 20948) in spleen homogenate isolated from patients with Gaucher disease with the homozygous N370S mutant of glucocerebrosidase (glucocerebrosidaseN370S; AC50 = 0.36 µM). ML-198 (5 µM) increases the translocation of glucocerebrosidaseN370S to lysosomes in dermal fibroblasts isolated from patients with Gaucher disease.
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1. Discovery, structure-