中文摘要：

RNA干涉（RNA interference,RNAi）是一种非常保守的细胞现象,在基础研究和疾病治疗方面具有重大的应用前景。尤其引人注目的是,新的RNAi方法学与已建立的转基因策略相结合,有效地将组织特异性RNAi导入到患者体内,有望治疗人类疾病。本文综述了RNAi的机制与在其临床前的实验研究,简要介绍了RNAi的新用途,讨论了RNAi基因治疗存在的问题,展望了RNAi基因治疗的应用前景。

英文摘要：

RNA interference （RNAi） is a conserved cellular phenomenon. It holds great prospects in basic research and therapeutic applications. Remarkably, the new RNAi methodology, in combination with the well established transgene technology, can introduce tissue specific RNAi to patients effectively to cure diseases. This paper mainly summarized the mechnism of RNAi and its preclinical applications. The new usage of RNAi and its prospects of therapeutic application were also introduced.