《生命科学》 2018, 30(9): 1003-1009

同源重组及非同源末端连接修复途径介导的基因编辑：CRISPR技术的认知、应用及展望

朱娉慧1#，罗　群1#，王曜峰1，2，冯　波1，2*

(1 中国科学院广州生物医药与健康研究院，华南干细胞与再生医学研究所，广州 510530；
    2 香港中文大学生物医学学院，中国香港)

摘　要：摘　要：CRISPR 系统具有精确识别及剪切特异性DNA 序列功能而被开发成一种高效的基因编辑工具。它以成本低廉、操作简便、效率高及通用性广等优势，成为新一代最具代表性的基因编辑技术。在应用中，CRISPR 系统可在特定靶点形成DNA 双链断裂，继而诱导同源重组(HDR) 或非同源末端连接修复(NHEJ)，为基因组定向改造与调控带来了革命性的突破。该文将对近年来生物科学领域中发展迅猛的研究工具CRISPR/Cas 系统进行介绍，包括其结构、作用原理、类型及应用等，并重点阐述同源重组或非同源末端连接修复途径介导的基因定向编辑技术及应用。

CRISPR-induced genomic editing via homology-dependent and independent repair pathways: recent insights， applications and perspectives

ZHU Ping-Hui1#， LUO Qun1#， WANG Yao-Feng1，2， FENG Bo1，2*

(1 South China Institute for Stem Cell Biology and Regenerative Medicine， Guangzhou Institute of Biomedicine and Health， Chinese Academy of Sciences， Guangzhou 510530， China; 2 School of Biomedical Sciences， The Chinese University of Hong Kong， Hong Kong SAR， China)

Abstract: Abstract: CRISPR is a high effective and robust system to target a specific genomic region with high accuracy. It has become the most popular genomic editing tool in biomedical and clinical research nowadays. CRISPR complex can induce double-strand breaks at a specific genomic region. Mediated by homology-dependent or independent repair pathways， precious genomic editing can occur at the broken sites， using various donor designs. Here we review the composition， principle， as well as various types of CRISPR systems discovered and applied in current biomedical studies. In addition， a summary of knock-in strategies mediated by homology-dependent or independent repair pathways will be presented. Recent improvements and applications of these technologies will also be discussed. Furthermore， we highlight the current challenges in CRISPR-induced genomic editing in biomedical and clinical research.