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RNA干扰:新一代生物制品

RNA interference: an emerging generation of biologicals.

作者信息

Shrivastava Neeta, Srivastava Anshu

机构信息

B. V. Patel Pharmaceutical Education and Research Development (PERD) Centre, Ahmedabad, Gujarat, India.

出版信息

Biotechnol J. 2008 Mar;3(3):339-53. doi: 10.1002/biot.200700215.

Abstract

RNA interference (RNAi) is a mechanism displayed by most eukaryotic cells to rid themselves of foreign double-stranded RNA molecules. RNAi has now been demonstrated to function in mammalian cells to alter gene expression, and has been used as a means for genetic discovery as well as a possible strategy for genetic correction. RNAi was first described in animal cells by Fire and colleagues in the nematode, Caenorhabditis elegans. Knowledge of RNAi mechanism in mammalian cell in 2001 brought a storm in the field of drug discovery. During the past few years scientists all over the world are focusing on exploiting the therapeutic potential of RNAi for identifying a new class of therapeutics. The applications of RNAi in medicine are unlimited because all cells possess RNAi machinery and hence all genes can be potential targets for therapy. RNAi can be developed as an endogenous host defense mechanism against many infections and diseases. Several studies have demonstrated therapeutic benefits of small interfering RNAs and micro RNAs in animal models. This has led to the rapid advancement of the technique from research discovery to clinical trials.

摘要

RNA干扰(RNAi)是大多数真核细胞所展现出的一种机制,用于清除自身的外源双链RNA分子。现已证明RNAi在哺乳动物细胞中发挥作用以改变基因表达,并已被用作基因发现的手段以及基因校正的一种可能策略。RNAi最早由Fire及其同事在线虫秀丽隐杆线虫的动物细胞中描述。2001年对哺乳动物细胞中RNAi机制的了解在药物发现领域引发了一场风暴。在过去几年中,世界各地的科学家都在专注于开发RNAi的治疗潜力,以确定一类新的治疗方法。RNAi在医学上的应用是无限的,因为所有细胞都具有RNAi机制,因此所有基因都可能成为治疗靶点。RNAi可以发展成为一种针对许多感染和疾病的内源性宿主防御机制。多项研究已在动物模型中证明了小分子干扰RNA和微小RNA的治疗益处。这使得该技术从研究发现迅速推进到临床试验。

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