Human hematopoietic stem / progenitor cells modified by zinc-finger nucleases targeted to CCR 5 control HIV-1 in vivo

  title={Human hematopoietic stem / progenitor cells modified by zinc-finger nucleases targeted to CCR 5 control HIV-1 in vivo},
  author={Nathalia G Holt and Jianbin Wang and Kenneth Eunhan Kim and Geoffrey Friedman and Xingchao Wang and Vanessa Taupin and Gay M. Crooks and Donald B Kohn and Philip D. Gregory and Michael C. Holmes and Paula M. Cannon},
CCR5 is the major HIV-1 co-receptor, and individuals homozygous for a 32-bp deletion in CCR5 are resistant to infection by CCR5-tropic HIV-1. Using engineered zinc-finger nucleases (ZFNs), we disrupted CCR5 in human CD34+ hematopoietic stem/ progenitor cells (HSPCs) at a mean frequency of 17% of the total alleles in a population. This procedure produces both monoand bi-allelically disrupted cells. ZFN-treated HSPCs retained the ability to engraft NOD/SCID/IL2rγnull mice and gave rise to… CONTINUE READING
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