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RNA干扰:潜在的治疗靶点。

RNA interference: potential therapeutic targets.

作者信息

Jana S, Chakraborty C, Nandi S, Deb J K

机构信息

Department of Biochemical Engineering and Biotechnology, Indian Institute of Technology--Delhi, Hauz Khas, New Delhi, 110016, India.

出版信息

Appl Microbiol Biotechnol. 2004 Nov;65(6):649-57. doi: 10.1007/s00253-004-1732-1. Epub 2004 Sep 15.

Abstract

One of the most exciting findings in recent years has been the discovery of RNA interference (RNAi). RNAi methodologies hold the promise to selectively inhibit gene expression in mammals. RNAi is an innate cellular process activated when a double-stranded RNA (dsRNA) molecule of greater than 19 duplex nucleotides enters the cell, causing the degradation of not only the invading dsRNA molecule, but also single-stranded (ssRNAs) RNAs of identical sequences, including endogenous mRNAs. The use of RNAi for genetic-based therapies has been widely studied, especially in viral infections, cancers, and inherited genetic disorders. As such, RNAi technology is a potentially useful method to develop highly specific dsRNA-based gene-silencing therapeutics.

摘要

近年来最令人兴奋的发现之一是RNA干扰(RNAi)的发现。RNAi方法有望在哺乳动物中选择性抑制基因表达。RNAi是一种天然的细胞过程,当大于19个双链核苷酸的双链RNA(dsRNA)分子进入细胞时被激活,这不仅会导致入侵的dsRNA分子降解,还会导致相同序列的单链(ssRNAs)RNA降解,包括内源性mRNA。RNAi在基于基因的治疗中的应用已得到广泛研究,尤其是在病毒感染、癌症和遗传性遗传疾病方面。因此,RNAi技术是开发基于dsRNA的高度特异性基因沉默疗法的一种潜在有用方法。

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