基因编辑技术的研究进展

摘要

The most widely used approach for defining gene function is to reduce or completely disrupt its normal expression.Many gene editing tools are used widely in recent years,including RNAi technology and some increasingly developed technologies with efficient enzymes,such as zinc finger nuclease (ZFN),transcription activator-like effector nucleases (TALENs) and clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) 9 system (CRISPR/Cas9).In addition to above technologies,NgAgo is also a new gene editing tool published in 2016.For over a decade,gene editing tools represented by the CRISPR/Cas9 technique have been widely used in gene knockout research based on the cellular level and individual level.Here,the related principles and the latest research progresses of these technologies are summarized,and the principle of CRISPR/Cas9 technology and its latest progress in gene knockout animal models are highlighted.%功能缺失(loss of function)是研究基因功能及其相关表型最重要、最直接的方法和策略.基因编辑技术从开始的RNA干扰(RNAinterference,RNAi)技术到近年发展起来的高效酶技术都得到了广泛的应用.这些高效酶技术包括锌指核酸酶(zinc finger nuclease,ZFN)技术、转录激活样效应物核酸酶(transcription activator-like effector nucleases,TALENs)技术和成簇的规律间隔的短回文重复序列系统(clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas)9 system,CRISPR/Cas9).除此之外,还有2016年发表的NgAgo (Natronobacterium gregoryi Argonaute)编辑技术.近十年来,以CRISPR/Cas9为代表的基因编辑技术已经广泛应用于细胞水平和个体水平的基因功能敲除的研究.现就以上基因编辑技术的原理及最新进展进行综述,并重点介绍CRISPR/Cas9的技术原理以及该技术在建立基因敲除动物模型中的最新进展.