Potent aminocyclitol glucocerebrosidase inhibitors are subnanomolar pharmacological chaperones for treating gaucher disease.

Amino-myo-inositol derivatives have been found to be potent inhibitors of glucocerebrosidase (GCase), the β-glucosidase enzyme deficient in Gaucher disease (GD). When tested using lymphoblasts derived from patients with GD homozygous for N370S or L444P mutations, the compounds enhanced GCase activit...

詳細記述

書誌詳細
主要な著者: Trapero, A, González-Bulnes, P, Butters, T, Llebaria, A
フォーマット: Journal article
言語:English
出版事項: 2012