迈向基于RNA干扰的持久抗HIV基因疗法。
Toward a durable anti-HIV gene therapy based on RNA interference.
作者信息
Berkhout Ben
机构信息
Laboratory of Experimental Virology, Academic Medical Center, University of Amsterdam, the Netherlands.
出版信息
Ann N Y Acad Sci. 2009 Sep;1175(1):3-14. doi: 10.1111/j.1749-6632.2009.04972.x.
Abstract
Basic research in the field of molecular biology led to the discovery of the mechanism of RNA interference (RNAi) in Caenorhabditis elegans in 1998. RNAi is now widely appreciated as an important gene control mechanism in mammals, and several RNAi-based gene-silencing applications have already been used in clinical trials. In this review I will discuss RNAi approaches to inhibit the pathogenic human immunodeficiency virus type 1 (HIV-1), which establishes a chronic infection that would most likely require a durable gene therapy approach. Viruses, such as HIV-1, are particularly difficult targets for RNAi attack because they mutate frequently, which allows viral escape by mutation of the RNAi target sequence. Combinatorial RNAi strategies are required to prevent viral escape.
摘要
分子生物学领域的基础研究在1998年促成了秀丽隐杆线虫中RNA干扰（RNAi）机制的发现。如今，RNAi作为哺乳动物中一种重要的基因调控机制已得到广泛认可，并且一些基于RNAi的基因沉默应用已在临床试验中得到应用。在这篇综述中，我将讨论用于抑制致病性人类免疫缺陷病毒1型（HIV-1）的RNAi方法，HIV-1会引发慢性感染，很可能需要持久的基因治疗方法。诸如HIV-1之类的病毒是RNAi攻击的特别困难的目标，因为它们频繁突变，这使得病毒能够通过RNAi靶序列的突变而逃逸。需要采用组合RNAi策略来防止病毒逃逸。
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