基因编辑技术及其在基因治疗中的应用

doi:10.16288/j.yczz.18-142

遗传 ›› 2019, Vol. 41 ›› Issue (1): 18-27.doi: 10.16288/j.yczz.18-142

基因编辑技术及其在基因治疗中的应用

任云晓^1,^2,³,肖茹丹^1,⁴,娄晓敏^1,^2,^3,⁴,方向东^1,^2,^3,⁴

^1. 中国科学院北京基因组研究所,中国科学院基因组科学与信息重点实验室,北京 100101
^2. 中国科学院大学中丹学院,北京 101408
^3. 中国-丹麦科研教育中心,北京 101408
^4. 中国科学院大学生命科学学院,北京 100049

收稿日期:2018-08-16 修回日期:2018-10-27 出版日期:2019-01-20 发布日期:2018-12-06
作者简介:任云晓,硕士研究生,专业方向：疾病组学与转化医学研究。E-mail: renyunxiao16@big.ac.cn
基金资助:
国家自然科学基金项目(编号：31471115,81670109,81672698)资助

Research advance and application in the gene therapy of gene editing technologies

Ren Yunxiao^1,^2,³,Xiao Rudan^1,⁴,Lou Xiaomin^1,^2,^3,⁴,Fang Xiangdong^1,^2,^3,⁴

^1. CAS Key Laboratory of Genome Sciences and Information, Beijing Institute of Genomics, Chinese Academy of Sciences, Beijing 100101, China
^2. Sino-Danish College, University of Chinese Academy of Sciences, Bejing 101408, China
^3. Sino-Danish Center for Education and Research, Beijing 101408, China
^4. Life Sciences College, University of Chinese Academy of Sciences, Beijing 100049, China

Received:2018-08-16 Revised:2018-10-27 Online:2019-01-20 Published:2018-12-06
Supported by:
[Supported by the National Natural Science Foundation of China (Nos. 31471115, 81670109, 81672698)]

摘要/Abstract

摘要：

基因编辑技术是以特异性改变遗传物质靶向序列为目标的技术。近年来,锌指核酸酶(zinc finger nuclease, ZFN)、类转录激活因子效应核酸酶(transcription activator-like effector nuclease, TALEN)、规律成簇的间隔短回文重复(regular clustering of short palindrome repeats, CRISPR)和单碱基编辑(base editing, BE)技术的相继出现,不仅为基因功能研究提供了有力的工具,还为生命医学提供了新的治疗方案。基因编辑技术已经大范围应用于动物细胞模型的构建、药物靶点的筛查和基因功能研究等,在基因治疗领域也展现出广阔的应用前景。本文就基因编辑技术的研究进展及其在基因治疗中的应用进行了概述,并对基因编辑技术的的原理、发展史、优缺点以及在基因治疗中的应用前景和机遇挑战进行了讨论,以期为基因编辑技术的临床转化提供参考。

关键词: 基因编辑技术, CRISPR/Cas9, 单碱基编辑, 基因治疗

Abstract:

Gene editing technologies are used to specifically edit the target sequence. With the development of zinc finger nuclease (ZFN), transcription activator-like effector nuclease (TALEN), regular clustering of short palindrome repeats (CRISPR) and single base editing (BE) techniques, gene editing technologies not only provide powerful tools for gene functional studies, but also offer new therapeutic strategies in biomedical research. Gene editing has demonstrated broad application prospects in the gene therapy field, as well as in the construction of animal and cell models, drug target screening and gene functional research. In this review, we summarize several typical gene editing technologies, their characteristics and applications in gene therapy and discusses their opportunities and challenges in gene therapy, thereby providing critical insights and references on the clinical application of gene editing technologies.

Key words: gene editing technologies, CRISPR/Cas9, BE, gene therapy

任云晓, 肖茹丹, 娄晓敏, 方向东. 基因编辑技术及其在基因治疗中的应用[J]. 遗传, 2019, 41(1): 18-27.

Ren Yunxiao, Xiao Rudan, Lou Xiaomin, Fang Xiangdong. Research advance and application in the gene therapy of gene editing technologies[J]. Hereditas(Beijing), 2019, 41(1): 18-27.

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