CRISPR/Cas9基因组编辑技术在HIV-1感染治疗中的应用进展

doi:10.16288/j.yczz.15-284

遗传 ›› 2016, Vol. 38 ›› Issue (1): 9-16.doi: 10.16288/j.yczz.15-284

CRISPR/Cas9基因组编辑技术在HIV-1感染治疗中的应用进展

韩英伦^{1, 2}, 李庆伟^{1, 2}

1. 辽宁师范大学生命科学学院,大连 116029;
2. 辽宁师范大学七鳃鳗研究中心,大连 116029

收稿日期:2015-06-19 修回日期:2015-09-07 出版日期:2016-01-20 发布日期:2016-01-20
通讯作者: 李庆伟,教授,博士生导师,研究方向：细胞生物学。E-mail: liqw@263.net E-mail:hanyinglun@163.com
作者简介:韩英伦,讲师,研究方向：细胞生物学。
基金资助:
国家重点基础研究发展规划(973计划)(编号：2013CB835304),全国海洋公益项目(编号：201305016),国家自然科学基金项目(编号：31170353, 31202020, 31271323)和大连市科技计划(编号：2013E11SF056)资助

Application progress of CRISPR/Cas9 genome editing technology in the treatment of HIV-1 infection

Yinglun Han^{1, 2}, Qingwei Li^{1, 2}

1. College of Life Science, Liaoning Normal University, Dalian 116029, China;
2. Lamprey Research Center, Liaoning Normal University, Dalian 116029, China

Received:2015-06-19 Revised:2015-09-07 Online:2016-01-20 Published:2016-01-20
Supported by:
[Supported by grants from the National Program on Key Basic Research Project (973 Program) (No; 2013CB835304), the National Marine Public Projects (No; 201305016), the National Natural Science Foundation of China (General Program) (Nos; 31170353, 31202020, 31271323), and Dalian Science and Technology Program (No; 2013E11SF056)]

摘要/Abstract

摘要： 基因治疗是将外源正常基因通过一定方式导入人体靶细胞以纠正或补偿因基因缺陷和异常引起的疾病,从而达到治疗目的。因此,基因治疗的技术方法在研究持续感染HIV-1或潜伏感染HIV-1原病毒患者的治疗中具有重大的现实意义。目前,现有的基因治疗方法存在识别靶向位点有限及脱靶几率大等主要问题。最新研究表明来源于细菌和古菌的规律间隔成簇短回文重复序列及其相关核酸酶9系统[Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated nuclease 9 (Cas9), CRISPR/Cas9]已被成功改造成基因组定点编辑工具。因此,如何利用CRISPR/Cas9系统实现对HIV-1病毒基因组进行高效靶向修饰,从而达到治疗HIV-1感染病患的目的已经成为当前研究的热点。本文参考最新国内外研究成果,重点介绍了 CRISPR/Cas9基因组编辑技术在HIV-1感染疾病治疗中的应用,主要包括CCR5基因编辑、清除HIV-1原病毒以及活化HIV-1原病毒,以期为HIV-1感染疾病的预防与治疗提供重要研究参考。

关键词: CRISPR/Cas9, 人类免疫缺陷病毒, 基因组编辑

Abstract: The goal of gene therapy is to introduce foreign genes into human target cells in a certain way to correct or compensate diseases caused by defective or abnormal genes. Therefore, gene therapy has great practical significance in studying the treatment of persistent or latent HIV-1 infection. At present, the existing methods of gene therapy have some major defects such as limited target site recognition and high frequency of off-targets. The latest research showed that the clustered regularly interspaced short palindromic repeats (CRISPR) /CRISPR-associated nuclease 9 (Cas9) system from bacteria and archaea has been successfully reformed to a targeted genome editing tool. Thus, how to achieve the goal of treating HIV-1 infection by modifying targeted HIV-1 virus genome effectively using the CRISPR/Cas9 system has become a current research focus. Here we review the latest achievements worldwide and briefly introduce applications of the CRISPR/Cas9 genome editing technology in the treatment of HIV-1 infection, including CCR5 gene editing, removal of HIV-1 virus and activation of HIV-1 virus, in order to provide reference for the prevention and treatment of HIV-1 infection.

Key words: CRISPR /Cas9, Human immunodeficiency virus, genome editing

韩英伦, 李庆伟. CRISPR/Cas9基因组编辑技术在HIV-1感染治疗中的应用进展[J]. 遗传, 2016, 38(1): 9-16.

Yinglun Han, Qingwei Li. Application progress of CRISPR/Cas9 genome editing technology in the treatment of HIV-1 infection[J]. HEREDITAS(Beijing), 2016, 38(1): 9-16.

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CRISPR/Cas9基因组编辑技术在HIV-1感染治疗中的应用进展

Application progress of CRISPR/Cas9 genome editing technology in the treatment of HIV-1 infection

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