CRISPR/Cas9运输系统的研究进展及其在基因相关疾病方面的应用 <sup>*</sup>

doi:10.13523/j.cb.20180311

中国生物工程杂志

2018, Vol. 38

Issue (3): 81-88 DOI: 10.13523/j.cb.20180311

综述

CRISPR/Cas9运输系统的研究进展及其在基因相关疾病方面的应用 ^*

徐然^1,²,陈松^1*(

)

1 淮安市消化道肿瘤重点实验室淮安市医药生物技术研究所江苏护理职业学院淮安 223001
2 南通大学医学院南通 226000

Research Progress of CRISPR/Cas9 Delivery System and Its Application in Gene-related Diseases

Ran XU^1,²,Song CHEN^1*(

)

1 Huai’an Key Laboratory of Gastrointestinal Cancer, Huai’an Institute of Medical Biotechnology, Jiangsu College of Nursing, Huai’an 223001, China;
2 Medical School of Nantong University,Nantong 226000,China

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摘要：

CRISPR/Cas9系统是细菌体在长期进化过程中抵御病毒或噬菌体DNA的一种适应性免疫系统。尽管近年来的一些基因编辑技术,如锌指核酸酶、转录激活因子效应物核酸酶等已经给基因编辑带来了许多便利,但CRISPR/Cas9系统以其独特的优势,给基因编辑领域带来了革命性的变化。CRISPR/Cas9系统在生物研究、基因相关疾病的治疗、疾病的基因水平研究等多方面都有广泛的应用。综述近年来CRISPR/Cas9运输系统的研究进展及其在基因缺陷疾病方面取得的应用成果,分析慢病毒和腺相关病毒运载基因编辑元件的利弊,并探讨CRISPR/Cas9系统在基因编辑效率方面的影响因素及仍然存在的问题。

关键词： CRISPR/Cas9运输系统; 基因相关疾病; 基因编辑

Abstract:

The clustered regularly interspaced short palindromic repeats (CRISPR)/associated protein 9 (CRISPR/Cas9) is an adaptive immune system. CRISPR/Cas9 technology can be used to edit single or multiple genes in a wide variety of cell types and organisms in vitro and in vivo. In recent years, some of the gene editing techniques, such as Zinc finger nuclease and transcriptional activator-like effector nuclease, have brought a lot of convenience to gene editing. But CRISPR/Cas9 system with its unique features has brought a revolutionary change to the field of gene editing. CRISPR/Cas9 technology has been widely used in the fields of biological research, the treatment of gene-related diseases, and the research of disease in gene level. The research progress of CRISPR/Cas9 delivery system and its application in genetic diseases are reviewed, and the pros and cons of lentivirus and adeno-associated virus vectors in the delivery of gene editing elements are discussed,and the problems that still exist in the CRISPR/Cas9 system are explored.

Key words: CRISPR/Cas9 delivery system Gene-related diseases Gene editing

收稿日期: 2017-07-28 出版日期: 2018-04-04

ZTFLH:

Q812

基金资助: 国家自然科学基金(81572877);江苏省自然科学基金(SBK2015021930);淮安市应用研究与科技攻关(社会发展)计划(HAS2014015)

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引用本文:

徐然,陈松. CRISPR/Cas9运输系统的研究进展及其在基因相关疾病方面的应用 ^*[J]. 中国生物工程杂志, 2018, 38(3): 81-88.

Ran XU,Song CHEN. Research Progress of CRISPR/Cas9 Delivery System and Its Application in Gene-related Diseases. China Biotechnology, 2018, 38(3): 81-88.

链接本文:

https://manu60.magtech.com.cn/biotech/CN/10.13523/j.cb.20180311 或 https://manu60.magtech.com.cn/biotech/CN/Y2018/V38/I3/81

图1 CRISPR/Cas系统基本结构

图2 CRISPR/Cas9系统作用机制[12]

图3 dCas9和Nickase作用机制[15]

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