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

作　　者：韩英伦[1,2] 李庆伟[1,2] 

机构地区：[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

出　　处：《遗传》2016年第1期9-16,共8页Hereditas（Beijing)

基　　金：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);大连市科技计划(编号:2013E11SF056)资助~~

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。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 latestresearch 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.

关 键 词：CRISPR/Cas9 人类免疫缺陷病毒 基因组编辑 

分 类 号：R512.91[医药卫生—内科学] R450[医药卫生—临床医学]