Rational Design and Synthesis of Highly Potent Pharmacological Chaperones for Treatment of N370S Mutant Gaucher Disease
Journal of Medicinal Chemistry2009Vol. 52(10), pp. 3146–3149
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Guan‐Nan Wang, Gabriele Reinkensmeier, Siwei Zhang, Jian Zhou, Liangren Zhang, Lihe Zhang, Terry D. Butters, Xin‐Shan Ye
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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