CRISPR/Cas9基因编辑技术的应用研究进展

近年来,CRISPR/Cas9基因编辑技术经过不断完善和改造,正逐步取代锌指核酸酶（ZFN）技术和转录激活因子样效应物核酸酶（TALEN）技术,成为具有广阔应用前景的第三代基因编辑技术。CRISPR/Cas9技术目前已广泛应用于细胞的基因编辑和基因调节、基因敲除动物模型的构建、人类疾病动物模型的治疗研究等领域。此外,已有相关实验证实该技术能够在人类胚胎中发挥基因编辑的作用,并且国内外已有2项CRISPR/Cas9临床试验正在开展,未来应用于临床靶向治疗前景广阔。现就CRISPR/Cas9系统的基本结构、作用原理及其应用的最新研究进展进行综述。...

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Bibliographic Details
Published in国际生殖健康/计划生育杂志 Vol. 36; no. 6; pp. 482 - 487
Main Author 陈怡李 姚书忠
Format Journal Article
LanguageChinese
Published 中山大学附属第一医院妇产科, 广州,510080 2017
Subjects
CRISPR/Cas9
临床试验
基因编辑
基因表达调控
模型,动物
胚胎研究
模型,动物
基因表达调控
基因编辑
Models,animal
胚胎研究
CRISPR/Cas9
Gene editing
Embryo research
Clinical trial
Gene expression regulation
临床试验
Online AccessGet full text
ISSN1674-1889
DOI10.3969/j.issn.1674-1889.2017.06.010

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Summary:近年来,CRISPR/Cas9基因编辑技术经过不断完善和改造,正逐步取代锌指核酸酶（ZFN）技术和转录激活因子样效应物核酸酶（TALEN）技术,成为具有广阔应用前景的第三代基因编辑技术。CRISPR/Cas9技术目前已广泛应用于细胞的基因编辑和基因调节、基因敲除动物模型的构建、人类疾病动物模型的治疗研究等领域。此外,已有相关实验证实该技术能够在人类胚胎中发挥基因编辑的作用,并且国内外已有2项CRISPR/Cas9临床试验正在开展,未来应用于临床靶向治疗前景广阔。现就CRISPR/Cas9系统的基本结构、作用原理及其应用的最新研究进展进行综述。
Bibliography:While the CRISPR/Cas9 gene editing technology has been continuously improved and reformed in recent years, it has been gradually replaced zinc-finger nuclease（ZFN） and transcription activator-like effector nuclease（TALEN） technology as the third generation gene editing technology with wide application prospect.CRISPR/Cas9 technology has been widely used in the cellular gene editing and gene regulation, gene knockout animal models construction and the human disease treatment research based on animal models. In addition, related experiments have already confirmed that this technology can play a role in gene editing of human embryos. There are two CRISPR/Cas9 clinical trials at present. It is worth looking forward to be applied in clinical targeted therapy. In this paper, the basic structure, the principle of action and potential application of CRISPR/Cas9 gene editing technology are briefly reviewed.
12-1400/R
ISSN:1674-1889
DOI:10.3969/j.issn.1674-1889.2017.06.010