RNAi鉴定药物靶点及siRNAs治疗疾病

Using RNAi to identify drug targets and using siRNAs to cure diseases

RNAi是与靶基因序列同源的双链RNA(dsRNA)所诱导的一种特异性基因沉默现象。RNAi是生物体进化过程中抵御外来基因和病毒感染的进化保守机制,是真核生物中存在的一种抗病毒入侵、抑制转座子活动、调控基因表达的监控机制,具有重大生物学意义。RNA干扰技术的发现及其作为基因敲除工具在模式生物、哺乳动物细胞和体内的成功应用的例子确保了它在药学研究中的作用。越来越多的研究显示RNAi在药物研发中显示出极大潜力,目前已有部分RNAi药物进入临床试验阶段。本文将从两个方面来论述RNAi在药学的应用:一是RNAi在药物靶点鉴定中的运用;二是siRNAs作为药物在疾病治疗中的作用。

RNAi is a kind of peculiar gene silencing phenomenon induced by double strand RNA homologous with the sequence of target gene. It is a conservative evolution mechanism of living things （dsRNA） resisting the infections of outside genes and viruses in the process of evolution, and is a monitoring mechanism against invasion of viruses, transposition activity, and regulating gene expression. Therefore, it is of biological significance. The finding of RNAi technology and it successful application in gene knockout, mode organism, mammalian cells, in animal bodies has proved its important role in pharmaceutical researches. More and more studies show that RNAi has been demonstrated extremely large potentials in the aspect of drug researches. At present, there have been some siRNAs medicines in clinical experimental stages. This paper discusses its application in pharmacy in two aspects： one is identifying drug target, and the other is its application as medicines to treating diseases.