American Chemical Society
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Rational Design and Synthesis of Highly Potent Pharmacological Chaperones for Treatment of N370S Mutant Gaucher Disease

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journal contribution
posted on 2009-05-28, 00:00 authored by Guan-Nan Wang, Gabriele Reinkensmeier, Si-Wei Zhang, Jian Zhou, Liang-Ren Zhang, Li-He Zhang, Terry D. Butters, Xin-Shan Ye
Highly potent N-substituted δ-lactams have been rationally designed and synthesized by a concise route with a one-pot tandem reaction as key step. These iminosugars show weak inhibition of wild-type β-glucocerebrosidase but 3- to 6-fold increases in mutant enzyme activity (N370S).