RNAi therapeutics: a potential new class of pharmaceutical drugs

被引：873

作者：

Bumcrot, David ^{[1
]}

Manoharan, Muthiah ^{[1
]}

Koteliansky, Victor ^{[1
]}

Sah, Dinah W. Y. ^{[1
]}

机构：

[1] Alnylam Pharmaceut Inc, Cambridge, MA 02142 USA

来源：

NATURE CHEMICAL BIOLOGY | 2006年 / 2卷 / 12期

关键词：

D O I：

10.1038/nchembio839

中图分类号：

Q5 [生物化学]; Q7 [分子生物学];

学科分类号：

071010 ; 081704 ;

摘要：

The rapid identification of highly specific and potent drug candidates continues to be a substantial challenge with traditional pharmaceutical approaches. Moreover, many targets have proven to be intractable to traditional small-molecule and protein approaches. Therapeutics based on RNA interference (RNAi) offer a powerful method for rapidly identifying specific and potent inhibitors of disease targets from all molecular classes. Numerous proof-of-concept studies in animal models of human disease demonstrate the broad potential application of RNAi therapeutics. The major challenge for successful drug development is identifying delivery strategies that can be translated to the clinic. With advances in this area and the commencement of multiple clinical trials with RNAi therapeutic candidates, a transformation in modern medicine may soon be realized.

引用

页码：711 / 719

页数：9

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