Rational design and synthesis of highly potent pharmacological chaperones for treatment of N370S mutant Gaucher disease.

J Med Chem

State Key Laboratory of Natural and Biomimetic Drugs and School of Pharmaceutical Sciences, Peking University, Xue Yuan Road No. 38, Beijing 100191, China.

Published: May 2009

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Article Abstract

Highly potent N-substituted delta-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 beta-glucocerebrosidase but 3- to 6-fold increases in mutant enzyme activity (N370S).

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http://dx.doi.org/10.1021/jm801506mDOI Listing

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