Rational design and synthesis of highly potent β-glucocerebrosidase inhibitors

被引：92

作者：

Zhu, XX

Sheth, KA

Li, SH

Chang, HH

Fan, JQ

机构：

[1] Mt Sinai Sch Med, Dept Human Genet, New York, NY 10029 USA

[2] Amicus Therapeut Inc, Cranbury, NJ 08512 USA

来源：

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION | 2005年 / 44卷 / 45期

关键词：

biological activity; drug design; enzymes; inhibitors; natural products;

D O I：

10.1002/anie.200502662

中图分类号：

O6 [化学];

学科分类号：

0703 ;

摘要：

(Chemical Equation Presented) Highly potent human β-glucocerebrosidase inhibitors have been synthesized that appear to recognize both carbohydrate and hydrophobic binding sites. The most potent inhibitor, 6-nonyl isofagomine (see formula), shows an IC50 value at subnanomolar concentrations. These compounds are potential candidates for the development of small-molecule drugs for the treatment of Gaucher disease. © 2005 Wiley-VCH Verlag GmbH & Co. KGaA.

引用

页码：7450 / 7453

页数：4

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