RNA干扰(RNA interference, RNAi) 是生物界普遍存在的一种抵御外来基因和病毒感染的保守进化机制, 其本质是siRNA与靶向mRNA特异结合、并由RISC介导其降解, 从而阻止mRNA的翻译, 导致基因沉默。因此, RNAi可以作为基因功能研究、基因治疗等的新工具。但是, 随机设计的siRNA之间沉默效应差别很大。如何针对靶基因设计特异、高效的siRNA就成了一个关键的问题。文章对siRNA设计原则的研究进展进行了总结论述。

RNA interference (RNAi), a conserved evolutionary mechanism widely found in living nature, is considered for defending the alien gene and virus infection. RNAi can induce gene silencing by prohibiting the translation of the target mRNA, which is specially recognized by the corresponding siRNA, and finally degraded by the RISC. So RNAi can be applied as a new technology tool for the functional gene research and gene therapy. But the effects of random designed siRNAs on gene silencing differ greatly from each other. The design of the special, highly efficient siRNA comes to be a crucial problem. This issue gives an overview about the new progress of the siRNA design principle.