CRISPR/Cas9-Mediated CCR5 Ablation in Human Hematopoietic Stem/Progenitor Cells Confers HIV-1 Resistance In Vivo

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; Hu, Chen; Deng, Hongkui

doi:10.1016/j.ymthe.2017.04.027

Public

CRISPR/Cas9-Mediated CCR5 Ablation in Human Hematopoietic Stem/Progenitor Cells Confers HIV-1 Resistance In Vivo

Published in Molecular Therapy • May 17, 2017

Authors:

Lei Xu

Huan Yang

Yang Gao

Abstract

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 s...

Subject

CRISPR

Haematopoiesis

In vivo

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