Kim Daniel, Rossi John
Division of Molecular Biology, Beckman Research Institute of the City of Hope, Duarte, CA, USA.
Biotechniques. 2008 Apr;44(5):613-6. doi: 10.2144/000112792.
Within the past two decades we have become increasingly aware of the roles that RNAs play in regulation of gene expression. The RNA world was given a booster shot with the discovery of RNA interference (RNAi), a compendium of mechanisms involving small RNAs (less than 30 bases long) that regulate the expression of genes in a variety of eukaryotic organisms. Rapid progress in our understanding of RNAi-based mechanisms has led to applications of this powerful process in studies of gene function as well as in therapeutic applications for the treatment of disease. RNAi-based therapies involve two-dimensional drug designs using only identification of good Watson-Crick base pairing between the RNAi guide strand and the target, thereby resulting in rapid design and testing of RNAi triggers. To date there are several clinical trials using RNAi, and we should expect the list of new applications to grow at a phenomenal rate. This article summarizes our current knowledge about the mechanisms and applications of RNAi.
在过去二十年中，我们越来越意识到RNA在基因表达调控中所起的作用。随着RNA干扰（RNAi）的发现，RNA世界得到了进一步推动，RNA干扰是一系列涉及小RNA（长度小于30个碱基）的机制，这些小RNA在多种真核生物中调节基因表达。我们对基于RNAi的机制的理解取得了快速进展，这使得这一强大过程在基因功能研究以及疾病治疗应用中得到了应用。基于RNAi的疗法涉及二维药物设计，仅通过识别RNAi引导链与靶标之间良好的沃森-克里克碱基配对，从而实现RNAi触发物的快速设计和测试。迄今为止，有几项使用RNAi的临床试验，我们预计新应用的清单将以惊人的速度增长。本文总结了我们目前对RNAi机制和应用的认识。
