FCCC LOGO Faculty Publications
Hou P , Chen S , Wang S , Yu X , Chen Y , Jiang M , Zhuang K , Ho W , Hou W , Huang J , Guo D
Genome editing of CXCR4 by CRISPR/cas9 confers cells resistant to HIV-1 infection
Sci Rep. 2015 Oct 20;5 :15577
PMID: 26481100    PMCID: PMC4612538    URL: http://www.ncbi.nlm.nih.gov/pubmed/26481100
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Genome editing via CRISPR/Cas9 has become an efficient and reliable way to make precise, targeted changes to the genome of living cells. CXCR4 is a co-receptor for the human immunodeficiency virus type 1 (HIV-1) infection and has been considered as an important therapeutic target for AIDS. CXCR4 mediates viral entry into human CD4(+) cells by binding to envelope protein, gp120. Here, we show that human CXCR4 gene is efficiently disrupted by CRISPR/Cas9-mediated genome editing, leading to HIV-1 resistance of human primary CD4(+) T cells. We also show that the Cas9-mediated ablation of CXCR4 demonstrated high specificity and negligible off-target effects without affecting cell division and propagation. The precise and efficient genome editing of CXCR4 will provide a new strategy for therapeutic application against HIV-1 infection.
Hou, Panpan Chen, Shuliang Wang, Shilei Yu, Xiao Chen, Yu Jiang, Meng Zhuang, Ke Ho, Wenzhe Hou, Wei Huang, Jian Guo, Deyin England Sci Rep. 2015 Oct 20;5:15577. doi: 10.1038/srep15577.