Interfering with disease: a progress report on siRNA-based therapeutics

被引：971

作者：

de Fougerolles, Antonin

Vornlocher, Hans-Peter

Maraganore, John

Lieberman, Judy

机构：

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

[2] Alnylam Europe AG, D-95326 Kulmbach, Germany

[3] CBR Inst Biomed Res, Boston, MA 02115 USA

来源：

NATURE REVIEWS DRUG DISCOVERY | 2007年 / 6卷 / 06期

关键词：

D O I：

10.1038/nrd2310

中图分类号：

Q81 [生物工程学（生物技术）]; Q93 [微生物学];

学科分类号：

071005 ; 0836 ; 090102 ; 100705 ;

摘要：

RNA interference (RNAi) quietly crept into biological research in the 1990s when unexpected gene-silencing phenomena in plants and flatworms first perplexed scientists. Following the demonstration of RNAi in mammalian cells in 2001, it was quickly realized that this highly specific mechanism of sequence-specific gene silencing might be harnessed to develop a new class of drugs that interfere with disease-causing or disease-promoting genes. Here we discuss the considerations that go into developing RNAi-based therapeutics starting from in vitro lead design and identification, to in vivo pre-clinical drug delivery and testing. We conclude by reviewing the latest clinical experience with RNAi therapeutics.

引用

收藏

页码：443 / 453

页数：11

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