发育医学电子杂志 Vol. 5 35-44
发育基础
综述
CRISPR/Cas9的原理及其在疾病治疗中的研究进展
王亮 赵同标
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 100101
The principle and recent advances in CRISPR/Cas9 and its applications in therapies
WANG Liang, ZHAO Tong-biao
摘要 规律成簇间隔短回文重复序列（clustered regularly interspaced short palindromic repeats，CRISPR）及其相关Cas9蛋白介导的新一代基因编辑技术具有操作容易、成本低廉的优势，可实现精确高效的基因编辑功能，包括定点插入、定点敲除及定点突变等，已广泛用于多种动植物的基因编辑。近年来，CRISPR技术迅速发展，应用范围已拓展到基因调控、表观修饰、基因成像等领域。其在疾病治疗方面更是显示出广阔的应用前景。本文首先介绍CRISPR的作用机制、工作模型，进而就其研究历史及最新进展进行阐述，并对其在疾病治疗方面的应用进行归纳举例。
关键词:
CRISPR/Cas9
原理
基因编辑
疾病治疗
收稿日期: 2016-12-01
出版日期: 2017-01-30
发布日期: 2018-01-19
期的出版日期: 2017-01-30
基金资助: 科技部973计划（2013CB966901）；国家自然科学青年基金（31400831）；国家自然科学面上基金（31570995）
通讯作者:
赵同标：http://sourcedb.ioz.cas.cn/zw/zjrc/shengzhi/201206/t20120625_3604428.html
E-mail: tbzhao@ioz.ac.cn
引用本文:
王亮 赵同标. CRISPR/Cas9的原理及其在疾病治疗中的研究进展 [J]. 发育医学电子杂志, 2017, 5(1): 35-44.
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