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重新设计氨基糖苷类药物以治疗由提前终止突变引起的人类遗传疾病。

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

作者信息

Nudelman Igor, Rebibo-Sabbah Annie, Shallom-Shezifi Dalia, Hainrichson Mariana, Stahl Ido, Ben-Yosef Tamar, Baasov Timor

机构信息

The Edith and Joseph Fischer Enzyme Inhibitors Laboratory, Department of Chemistry, Institute of Catalysis Science and Technology, Technion-Israel Institute of Technology, Haifa 32000, Israel.

出版信息

Bioorg Med Chem Lett. 2006 Dec 15;16(24):6310-5. doi: 10.1016/j.bmcl.2006.09.013. Epub 2006 Sep 25.

DOI:10.1016/j.bmcl.2006.09.013
PMID:16997553
Abstract

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.

摘要

设计并合成了一系列临床上使用的氨基糖苷类抗生素巴龙霉素的新衍生物,并在体外翻译系统和离体哺乳动物培养细胞中检测了它们通读提前终止密码子突变的能力。其中一种结构,即假三糖衍生物,与巴龙霉素和庆大霉素相比,在培养细胞中显示出明显更高的终止密码子通读活性。

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Redesign of aminoglycosides for treatment of human genetic diseases caused by premature stop mutations.重新设计氨基糖苷类药物以治疗由提前终止突变引起的人类遗传疾病。
Bioorg Med Chem Lett. 2006 Dec 15;16(24):6310-5. doi: 10.1016/j.bmcl.2006.09.013. Epub 2006 Sep 25.
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