RNAi技术在病毒性疾病和肿瘤治疗中的应用前景

Clinical prospects of RNAi in viral diseases and tumor treatment

RNA干扰是内源性或外源性双链RNA诱发的mRNA水平上的基因沉默机制 ,具有高度特异性。 2 1nt干扰性小RNA在哺乳动物细胞中可以诱导强有力的特异性RNAi作用 ,这一突破性研究进展开创了人类疾病治疗的新天地 ,RNAi技术用于人类疾病治疗已受到极大关注 ,尤其在AIDS、乙型肝炎和丙型肝炎等病毒感染性疾病和肿瘤治疗中的应用研究最为活跃 ,并取得了令人鼓舞的成果。本文对siRNA的设计和制备的基本思路 ,及其在病毒性疾病和肿瘤治疗中的研究进展作一综述。

RNA interference (RNAi) is the process of highly sequence-specific post transcriptional gene silencing mechanism, induced by endogenous or extragenous double-stranded RNA (dsRNA).Twenty one nucleotide small interfering RNAs (siRNA) can specifically induces potent silencing effect of gene expression in mammalian cells. This remarkable study provides a new tool for the treatment of many human diseases including AIDS, hepatitis and tumors, etc. Here, the strategy of siRNA design and preparation, and its clinical prospects in the treatment of AIDs and tumor diseases are reviewed.