The silent treatment: siRNAs as small molecule drugs

被引：269

作者：

Dykxhoorn, DM

Palliser, D

Lieberman, J

机构：

[1] Harvard Univ, Sch Med, CBR Inst Biomed Res, Boston, MA 02115 USA

[2] Harvard Univ, Sch Med, Dept Pediat, Boston, MA 02115 USA

来源：

GENE THERAPY | 2006年 / 13卷 / 06期

关键词：

RNA interference; small interfering RNA; therapy; drug development;

D O I：

10.1038/sj.gt.3302703

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：

As soon as RNA interference (RNAi) was found to work in mammalian cells, research quickly focused on harnessing this powerful endogenous and specific mechanism of gene silencing for human therapy. RNAi uses small RNAs, less than 30 nucleotides in length, to suppress expression of genes with complementary sequences. Two strategies can introduce small RNAs into the cytoplasm of cells, where they are active - a drug approach where double-stranded RNAs are administered in complexes designed for intracellular delivery and a gene therapy approach to express precursor RNAs from viral vectors. Phase I clinical studies have already begun to test the therapeutic potential of small RNA drugs that silence disease-related genes by RNAi. This review will discuss progress in developing and testing small RNAi-based drugs and potential obstacles.

引用

页码：541 / 552

页数：12

共 128 条

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