The promises and pitfalls of RNA-interference-based therapeutics

被引：985

作者：

Castanotto, Daniela ^{[1
,2
]}

Rossi, John J. ^{[1
,2
]}

机构：

[1] City Hope Natl Med Ctr, Beckman Res Inst, Dept Mol Biol, Duarte, CA 91010 USA

[2] City Hope Natl Med Ctr, Beckman Res Inst, City Hope Grad Sch Biol Sci, Duarte, CA 91010 USA

来源：

NATURE | 2009年 / 457卷 / 7228期

基金：

美国国家卫生研究院;

关键词：

DOUBLE-STRANDED-RNA; IN-VIVO DELIVERY; DICER-SUBSTRATE SIRNA; HEPATITIS-C VIRUS; MAMMALIAN-CELLS; ENDOGENOUS SIRNAS; NEUROPATHIC PAIN; SYNTHETIC SIRNA; GENE-EXPRESSION; SOMATIC-CELLS;

D O I：

10.1038/nature07758

中图分类号：

O [数理科学和化学]; P [天文学、地球科学]; Q [生物科学]; N [自然科学总论];

学科分类号：

07 ; 0710 ; 09 ;

摘要：

The discovery that gene expression can be controlled by the Watson - Crick base- pairing of small RNAs with messenger RNAs containing complementary sequence - a process known as RNA interference - has markedly advanced our understanding of eukaryotic gene regulation and function. The ability of short RNA sequences to modulate gene expression has provided a powerful tool with which to study gene function and is set to revolutionize the treatment of disease. Remarkably, despite being just one decade from its discovery, the phenomenon is already being used therapeutically in human clinical trials, and biotechnology companies that focus on RNA- interference- based therapeutics are already publicly traded.

引用

页码：426 / 433

页数：8

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