2016年3月23日/生物谷BIOON/--人免疫缺陷病毒（HIV）导致获得性免疫缺陷综合征（AIDS），即艾滋病。在一项新的研究中，来自美国天普大学路易斯-卡茨医学院（Lewis Katz School of Medicine at Temple University）的研究人员开发出一种特定的基因编辑系统CRISPR/Cas9，从而为最终治愈HIV感染铺平道路。他们证实利用这种基因编辑系统能够有效地和安全地将这种病毒从在体外培养的人T细胞的DNA中清理掉。相关研究结果于2016年3月4日在线发表在自然出版集团旗下的Scientific Reports期刊上，论文标题为“Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR/Cas9 Gene Editing”。
Elimination of HIV-1 Genomes from Human T-lymphoid Cells by CRISPR/Cas9 Gene Editing
Rafal Kaminski, Yilan Chen, Tracy Fischer, Ellen Tedaldi, Alessandro Napoli, Yonggang Zhang, Jonathan Karn, Wenhui Hu & Kamel Khalili
We employed an RNA-guided CRISPR/Cas9 DNA editing system to precisely remove the entire HIV-1 genome spanning between 5′ and 3′ LTRs of integrated HIV-1 proviral DNA copies from latently infected human CD4+ T-cells. Comprehensive assessment of whole-genome sequencing of HIV-1 eradicated cells ruled out any off-target effects by our CRISPR/Cas9 technology that might compromise the integrity of the host genome and further showed no effect on several cell health indices including viability, cell cycle and apoptosis. Persistent co-expression of Cas9 and the specific targeting guide RNAs in HIV-1-eradicated T-cells protected them against new infection by HIV-1. Lentivirus-delivered CRISPR/Cas9 significantly diminished HIV-1 replication in infected primary CD4+ T-cell cultures and drastically reduced viral load in ex vivo culture of CD4+ T-cells obtained from HIV-1 infected patients. Thus, gene editing using CRISPR/Cas9 may provide a new therapeutic path for eliminating HIV-1 DNA from CD4+ T-cells and potentially serve as a novel and effective platform toward curing AIDS.