RNA干扰(RNA interference,RNAi)是近年发展起来的一种新技术。RNAi是指通过外源性或内源性的双链RNA在体内诱导靶基因mR_NA产生特异性降解,进而引起不同水平的基因沉默。其效应分子主要是小干扰RNA(siRNA)。siRNA是生物界普遍存在的一种抵御外来基因和病毒感染的基因调控方式,也是一种重要的研究工具。大量的研究工作致力于设计合理的siRNA片段用于基因功能研究,并将其作为一种治疗方法用于肿瘤、病毒性疾病等基因治疗以及药物靶向研究。因此本文对siRNA的作用机制、设计原则及其在临床应用中的缺点和解决方法进行综述。
: RNA interference (RNAi) is a newly developed technology. It is the different levels of gene silencing induced by specific degradation of targeted genes in vivo, and both exogenous and endogenous double- stranded RNAs could induce the specific degradation, which was realized mainly by Small interfering RNA (siRNA). siRNA was commonly exists in the nature as a gene regulation way to prevent alien gene and viral infection. And SiRNA is becoming an important investigating tool. A lot of studies were devoted to the effective sequence design of siRNA to study gene's function, SiRNA is regarded as an effective therapy for cancer, viral disease and study for drug targeting. This paper focuses on the interference mechanisms, design regulations, shortcomings in clinical treatment and solutions of siRNA.