浅析基于CRISPR-Cas9技术的基因治疗研究进展.PDFVIP

生 物 工 程 学 报 詹长生 等/基于CRISPR-Cas9 技术的基因治疗研究进展 861 Chinese Journal of Biotechnology /cjbcn July 25, 2016, 32(7): 861−869 DOI: 10.13345/j.cjb.150542 ©2016 Chin J Biotech, All rights reserved 综 述 基于CRISPR-Cas9 技术的基因治疗研究进展 詹长生，夏小雨 上海交通大学 医学院，上海 200025 詹长生, 夏小雨. 基于CRISPR-Cas9 技术的基因治疗研究进展. 生物工程学报, 2016, 32(7): 861–869. Zhan CS, Xia XY. Research progress of CRISRP-Cas9 system for gene therapy. Chin J Biotech, 2016, 32(7): 861–869. 摘 要: CRISPR-Cas9 系统是细菌在与噬菌体抗争的进化过程中产生的一种抵御外源DNA 入侵的机制，能有 效识别并剪切外源 DNA 。基于其识别切除外源 DNA 的原理，CRISPR-Cas9 系统被开发成为新一代基因编辑 工具。与ES 打靶、ZFN 、TALEN 等技术途径相比，CRISPR-Cas9 系统操作简便、效率高、成本低，有着极其 广阔的应用前景。本文整理了近年内有关 CRISPR-Cas9 系统的最新文献报道，对该系统工作原理以及针对基 因治疗的研究进展进行综述。 关键词: CRISPR-Cas9 技术，基因编辑，疾病模型，基因治疗，个性化医疗 Research progress of CRISPR-Cas9 system for gene therapy Changsheng Zhan, and Xiaoyu Xia School of Medicine, Shanghai Jiao Tong University, Shanghai 200025, China Abstract: The clustered regulatory interspaced short palindromic repeat-Cas9 (CRISPR-Cas9) system is the part of the prokaryotic immune system, which could recognize and delete the exogenous sequences originated from virus or plasmid. Based on its mechanism, CRISPR-Cas9 system was developed into the new generation of gene editing tool. Compared to the existed technologies such as ES targeting, ZFN or TALEN, CRISPR-Cas9 system is a more efficient, economical and promising approach to manipulate the genome. In this review, we summarize the research progress about CRISPR-Cas9 technology, especially the latest applications in gene therapy studies of human diseases. Keywords: CRISPR-Cas9 technology, gene edit