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Hereditas(Beijing) >

2019 , Vol. 41 >Issue 1: 18 - 27

DOI: https://doi.org/10.16288/j.yczz.18-142

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

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  • 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 date: 2018-08-16

  Revised date: 2018-10-27

  Online published: 2018-12-06

Supported by

[Supported by the National Natural Science Foundation of China (Nos. 31471115, 81670109, 81672698)]

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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

Cite this article

Yunxiao Ren, Rudan Xiao, Xiaomin Lou, Xiangdong Fang . Research advance and application in the gene therapy of gene editing technologies[J]. Hereditas(Beijing), 2019 , 41(1) : 18 -27 . DOI: 10.16288/j.yczz.18-142

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