Department of Molecular, Cellular, and Developmental Biology, Yale University, P. O. Box 208103, New Haven, CT 06520, USA.
Int J Biochem Cell Biol. 2010 Aug;42(8):1243-51. doi: 10.1016/j.biocel.2009.04.023. Epub 2009 May 13.
Not many scientific breakthroughs bring significant advances simultaneously in both basic research and translational applications like the discovery of RNA interference. Along with the elucidation of the RNA interference pathway and the discovery of its participation in crucial biological events, a branch of science has grown to utilize the RNA interference pathway as a biotechnology for both basic and applied research. Small interference RNA, plasmid-, and virus-encoded short-hairpin RNA are now regular reagents in the tool box of biologists to knockdown the expression of specific genes posttranscriptionally. Efforts have also been made to develop RNA interference based therapeutics into reality. Many concerns about the RNA interference technique have now been answered through research and development, although hurdles are still present. In this review, the RNA interference/microRNA pathway is briefly introduced followed with a detailed summary about the design and application of the RNA interference experiments, along with examples of the utilization of the RNA interference technology in animal cells and model organisms. Recent progresses and current concerns are also highlighted. Two techniques, namely morpholino and external guide sequence, are discussed as complementary gene knockdown technology. RNA interference technology, along with several other alternative gene knockdown techniques, is now indispensable to modern biological and medical research.
没有多少科学突破能像 RNA 干扰的发现那样,同时在基础研究和转化应用方面带来重大进展。随着 RNA 干扰途径的阐明以及其参与关键生物事件的发现,一个分支科学已经发展起来,将 RNA 干扰途径用作基础和应用研究的生物技术。小干扰 RNA、质粒和病毒编码的短发夹 RNA 现在已成为生物学家工具箱中的常规试剂,可在后转录水平下调特定基因的表达。也已经努力将基于 RNA 干扰的疗法变为现实。尽管仍然存在障碍,但通过研究和开发,现在已经回答了许多关于 RNA 干扰技术的担忧。在这篇综述中,简要介绍了 RNA 干扰/miRNA 途径,详细总结了 RNA 干扰实验的设计和应用,以及在动物细胞和模式生物中利用 RNA 干扰技术的实例。还强调了最近的进展和当前的关注点。讨论了两种技术,即 morpholino 和外部引导序列,作为互补的基因敲低技术。RNA 干扰技术以及其他几种替代基因敲低技术,现在是现代生物和医学研究不可或缺的。