用于治疗N370S突变型戈谢病的高效药理伴侣分子的合理设计与合成。
Rational design and synthesis of highly potent pharmacological chaperones for treatment of N370S mutant Gaucher disease.
作者信息
Wang Guan-Nan, Reinkensmeier Gabriele, Zhang Si-Wei, Zhou Jian, Zhang Liang-Ren, Zhang Li-He, Butters Terry D, Ye Xin-Shan
机构信息
State Key Laboratory of Natural and Biomimetic Drugs and School of Pharmaceutical Sciences, Peking University, Xue Yuan Road No. 38, Beijing 100191, China.
出版信息
J Med Chem. 2009 May 28;52(10):3146-9. doi: 10.1021/jm801506m.
PMID:19397268
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).
摘要
通过以一锅串联反应为关键步骤的简洁路线，合理设计并合成了高效的N-取代δ-内酰胺。这些亚氨基糖对野生型β-葡萄糖脑苷脂酶的抑制作用较弱，但对突变酶（N370S）的活性有3至6倍的提升。
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