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

Authors
Volume
52
Issue
10
Identifiers
DOI: 10.1021/jm801506m

Abstract

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 wildtype β-glucocerebrosidase but 3- to 6-fold increases in mutant enzyme activity (N370S). © 2009 American Chemical Society.

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