RNAi药物研究进展

Research progress of RNAi drugs

干扰RNA(RNAi)是核酸药物的分支,也被称为小核酸药物,一般来说包括小干扰RNA(siRNA)和微小RNA(miRNA)两类。因为其具有基因靶向性强、开发周期短、候选靶标丰富等特点,而受到广泛的关注;但另外一方面,RNAi药物也具有核酸类药物的通常缺点:生物稳定性差、易被降解、给药系统设计比较困难等。这些缺点成为制约RNAi药物发展的瓶颈,也成为研究的热点。本文对RNAi药物的研究现状以及性能提高方案进行了综述和分析。

RNAi drugs are branches of nucleic acid drugs,also known as small nucleic acid drugs,generally including siRNA and miRNA.Because of its strong gene targeting,short development cycle,and abundant candidate targets,it has received widespread attention;but on the other hand,RNAi drugs also have the usual shortcomings of nucleic acid drugs:poor biological stability,easy degradation,difficult to design the drug delivery system and so on.These shortcomings have become a bottleneck restricting the development of RNAi drugs and therefore becoming a research focus.This article reviews and analyzes the current research status of RNAi drugs and performance improvement programs.