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