Redesign of aminoglycosides for treatment of human genetic diseases caused by premature stop mutations

被引：65
|
作者：
Nudelman, Igor
Rebibo-Sabbah, Annie
Shallom-Shezifi, Dalia
Hainrichson, Mariana
Stahl, Ido
Ben-Yosef, Tamar [1 ]
Baasov, Timor
机构：
[1] Technion Israel Inst Technol, Dept Genet, Fac Med, IL-31096 Haifa, Israel
[2] Technion Israel Inst Technol, Rappaport Family Inst Res Med Sci, IL-31096 Haifa, Israel
[3] Technion Israel Inst Technol, Edith & Joseph Fischer enzyme Inhibitors Lab, Dept Chem, Inst Catalysis Sci & Technol, IL-32000 Haifa, Israel
来源：
BIOORGANIC & MEDICINAL CHEMISTRY LETTERS | 2006年 / 16卷 / 24期
基金：
以色列科学基金会;
关键词：
aminoglycosides; cystic fibrosis; drug design; stop mutation suppression; Usher syndrome;
D O I：
10.1016/j.bmcl.2006.09.013
中图分类号：
R914 [药物化学];
学科分类号：
100701 ;
摘要：
A series of new derivatives of the clinically used aminoglycoside antibiotic paromomycin were designed, synthesized, and their ability to read-through premature stop codon mutations was examined in both in vitro translation system and ex vivo mammalian cultured cells. One of these structures, a pseudo-trisaccharide derivative, showed notably higher stop codon read-through activity in cultured cells compared to those of paromomycin and gentamicin. (c) 2006 Elsevier Ltd. All rights reserved.
引用
收藏
页码：6310 / 6315
页数：6
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