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

页码：6310 / 6315

页数：6

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