CRISPR基因魔剪助力HIV/AIDS治愈  

Magic scissors CRISPR and HIV/AIDS cure pursue

作　　者：李云[1,2] 周佳义 张锦鹏[1,2] 姜东伯[1] 杨琨[1] 

机构地区：[1]第四军医大学基础部免疫学教研室,陕西西安710032 [2]第四军医大学学员旅,陕西西安710032

出　　处：《转化医学电子杂志》2017年第2期60-64,共5页E-Journal of Translational Medicine

基　　金：国家自然科学基金面上项目(81171977)

摘　　要：抗逆转录病毒疗法(ART)通过高效抑制艾滋(AIDS)患者体内Ⅰ型免疫缺陷病毒(HIV-1)的复制,获得了显著的临床效果,但由于潜伏在患者体内病毒库的存在,艾滋病仍然是不治之症.近年来,基因编辑技术发展迅速,以锌指核酸酶(ZFNs)技术、转录激活因子样效应核酸酶(TALENs)技术和sgRNA(single guide RNA)引导的CRISPR/Cas9技术最为引人注目.利用这些基因手术刀,可以从多个途径预防病毒对体内正常细胞的感染,抑制或阻止其在体内的复制,例如对能被病毒感染的CD4+T细胞的两种辅受体CCR5和CXCR4基因进行突变,或者将整合到被感染的人体细胞中的前病毒DNA切除.其中CRISPR/Cas9技术以其强大的基因编辑和调控转录激活关闭的潜能,加之操作的易行和通用等特点,成为当前治疗艾滋病的最具前景的基因编辑技术.anti-retrovirus therapy has achieved obvious clinical efficiency by suppressing the replication of HIV-1 in the patients with acquired immunodeficiency syndrome, AIDS. However,AIDS still remains incurable because of latent virus reservoirs in patients infected. In recent years,with the rapid development of genome editing technologies,the ZFNs（ zinc-finger nucleases）and TALENs（ transcription activator-like effector nucleases） and CRISPR / Cas9 guided by single guide RNA have been utilized to edit the gene of HIV-1 co-receptors,such as CCR5 and CXCR4 in CD4＋T cells,or to excise the provirus genes which integrate into the genome of somatic cells infected,aiming to prevent the normal cells from HIV-1 or to restrict the replication of the virus in human body. At present,CRISPR / Cas9（ Clustered regularly interspaced short palindromic repeats / CRISPR-associated nuclease 9） has been shedding new light on HIV-1 cure pursuing,due to its powerful potential in genomic editing and transcription regulation in common with feasible manipulation.

关 键 词：ART 基因编辑技术 CRISPR/Cas9 人类免疫缺陷病毒 

分 类 号：R512.91[医药卫生—内科学]