Article
CRISPR/Cas9-Mediated CCR5 Ablation in Human Hematopoietic Stem/Progenitor Cells Confers HIV-1 Resistance In Vivo.
Molecular therapy : the journal of the American Society of Gene Therapy - 2 Aug 2017
Xu Lei, Yang Huan, Gao Yang, Chen Zeyu, Xie Liangfu, Liu Yulin, Liu Ying, Wang Xiaobao, Li Hanwei, Lai Weifeng, He Yuan, Yao Anzhi, Ma Liying, Shao Yiming, Zhang Bin, Wang Chengyan, Chen Hu, Deng Hongkui
Abstract excerpt
Transplantation of hematopoietic stem cells (HSCs) with a naturally occurring CCR5 mutation confers a loss of detectable HIV-1 in the patient, making ablation of the CCR5 gene in HSCs an ideal therapy for an HIV-1 cure. Although CCR5 disruption has been attempted in CD4+ T cells and hematopoietic stem/progenitor cells (HSPCs), efficient gene editing with high specificity and long-term therapeutic potential...
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