Article
New insights into the pharmacological chaperone activity of c2-substituted glucoimidazoles for the treatment of Gaucher disease.
Chembiochem : a European journal of chemical biology - 8 Jul 2013
Li Zhonghua, Li Tiehai, Dai Shaoxing, Xie Xiaoli, Ma Xiaofeng, Zhao Wei, Zhang Weimin, Li Jing, Wang Peng George
Abstract excerpt
Mutations in acid β-glucocerebrosidase (GCase) lead to the accumulation of the sphingolipid glucosylceramide, thereby resulting in Gaucher disease (GD). Active-site-specific competitive GCase inhibitors are effective pharmacological chaperones (PCs) that act as folding agents for mutant GCase folding in the endoplasmic reticulum. In this study, we prepared a series of glucoimidazole C2-substituent derivatives,...
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