Gene silencing by chemically modified siRNAs

被引：30

作者：

Engels, Joachim W. ^{[1
]}

机构：

[1] Goethe Univ Frankfurt, Inst Organ Chem & Chem Biol, D-60438 Frankfurt, Germany

来源：

NEW BIOTECHNOLOGY | 2013年 / 30卷 / 03期

关键词：

DOUBLE-STRANDED-RNA; INTERFERING RNAS; MAMMALIAN-CELLS; NUCLEIC-ACID; EXPRESSION; STABILITY; POTENT; THERAPEUTICS; ACTIVATION; ANTISENSE;

D O I：

10.1016/j.nbt.2012.07.002

中图分类号：

Q5 [生物化学];

学科分类号：

071010 ; 081704 ;

摘要：

RNA interference (RNAi) has not only already risen as a gold standard for validating gene function in basic science studies, but also holds great promise as a new therapeutic paradigm. Advantages of RNAi-based therapeutics include relatively fast initial screening and the ability to target proteins not yet addressable by traditional drug design strategies. In this review we describe the development of chemically modified small inhibiting siRNAs and their application as potential therapeutics during the past decade. Focus is on proper siRNA design, choice of chemical modification and how to circumvent immunogenicity as well as off-target effects.

引用

页码：302 / 307

页数：6

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