《生命科学》 2016, 28(8): 867-870

基于CRISPR-Cas9新型基因编辑技术研究

王皓毅1*，李劲松2，李　伟1

(1 中国科学院动物研究所，干细胞与生殖生物学国家重点实验室，北京 100190；2 中国科学院上海生命科学研究院生物化学与细胞生物学研究所，上海 200031)

摘　要：摘　要：高效、特异的目标基因组位点修饰一直是基因工程研究的重点和挑战。靶向基因编辑技术不仅能够有效地用于建立动物和细胞疾病模型、培育动植物新品种，并具有治疗诸多疾病的重大潜力。近年来靶向核酸酶技术的研究取得了重大进展，且逐渐成为基因编辑的主流工具，特别是规律成簇间隔短回文重复序列(CRISPR-Cas9) 技术因其靶向编辑目的基因的特异性、高效性和设计的简便性等诸多优点，得到更为广泛的应用。在中国科学院干细胞先导专项的支持下，基因编辑技术攻关团队在进一步改造和应用CRISPR-Cas9 技术方面取得了一系列成果，就此进行全方面的总结。

Application and improvement of CRISPR-Cas9 system for genome editing

WANG Hao-Yi1*， LI Jin-Song2， LI Wei1

(1 Institute of Zoology， Chinese Academy of Sciences， Beijing 100101， China; 2 Institute of Biochemistry and Cell Biology， Shanghai Institutes for Biological Sciences， Chinese Academy of Sciences， Shanghai 200031， China)

Abstract: Abstract: Genome editing technologies are invaluable tools for understanding the function of genes in development and disease. In recent years， the programmable site-specific DNA endonucleases， including zinc finger nucleases (ZFNs)， transcription activator-like effector nucleases (TALENs) and the clustered， regularly interspaced short palindromic-repeat (CRISPR) system， have gained tremendous popularity and become widely used. In particular due to its simplicity and robustness， the CRISPR-Cas9 systems has swiftly become the most commonly used tool for efficient genome editing of bacteria， plants， cell lines， primary cells and animal species ranging from Drosophila to primates. Supported by “Strategic Priority Research Program” of the Chinese Academy of Sciences (CAS)，research teams at CAS have made a series of breakthroughs in the application and improvement of CRISPR-Cas9 system. Here we review these progresses.