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
收藏
页码：7450 / 7453
页数：4
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