Progress toward in vivo use of siRNAs-II.
Progress toward in vivo use of siRNAs-II.
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DOI：
10.1038/mt.2011.263
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发表时间：
2012-03
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RNA干扰(RNAi)自10年前首次在哺乳动物细胞中应用以来，已被广泛用于活体研究。设计规则得到了改进，现在可以常规地获得抑制任何所需基因表达的试剂。与此同时，对不良副作用的分子基础的了解增加，导致了减轻这些担忧的化学修饰策略的发展。传递仍然是广泛采用体内RNAi方法的最大障碍。然而，已经取得了令人振奋的进展，正在开发的新的递送系统可能有助于克服这些障碍。这篇综述讨论了影响体内使用的RNAi生物化学和生物学的进展，并提供了展示这些原理的有趣应用的精选出版物的概述。重点放在自2006年发表本综述第一期以来发表的合成小干扰RNA(SiRNAs)的工作。
RNA interference (RNAi) has been extensively employed for in vivo research since its use was first demonstrated in mammalian cells 10 years ago. Design rules have improved, and it is now routinely possible to obtain reagents that suppress expression of any gene desired. At the same time, increased understanding of the molecular basis of unwanted side effects has led to the development of chemical modification strategies that mitigate these concerns. Delivery remains the single greatest hurdle to widespread adoption of in vivo RNAi methods. However, exciting advances have been made and new delivery systems under development may help to overcome these barriers. This review discusses advances in RNAi biochemistry and biology that impact in vivo use and provides an overview of select publications that demonstrate interesting applications of these principles. Emphasis is placed on work with synthetic, small interfering RNAs (siRNAs) published since the first installment of this review which appeared in 2006.