High frequency CCR5 editing in human hematopoietic stem progenitor cells protects xenograft mice from HIV infection

Daniel T. Claiborne, Zachary Detwiler, Steffen S. Docken, Todd D. Borland, Deborah Cromer, Amanda Simkhovich, Youdiil Ophinni, Ken Okawa, Timothy Bateson, Tao Chen, Wesley Hudson, Radiana Trifonova, Miles P. Davenport, Tony W. Ho, Christian L. Boutwell () and Todd M. Allen
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Daniel T. Claiborne: The Wistar Institute
Zachary Detwiler: CRISPR Therapeutics
Steffen S. Docken: University of New South Wales
Todd D. Borland: CRISPR Therapeutics
Deborah Cromer: University of New South Wales
Amanda Simkhovich: MIT and Harvard
Youdiil Ophinni: MIT and Harvard
Ken Okawa: MIT and Harvard
Timothy Bateson: MIT and Harvard
Tao Chen: MIT and Harvard
Wesley Hudson: MIT and Harvard
Radiana Trifonova: MIT and Harvard
Miles P. Davenport: University of New South Wales
Tony W. Ho: CRISPR Therapeutics
Christian L. Boutwell: MIT and Harvard
Todd M. Allen: MIT and Harvard

Nature Communications, 2025, vol. 16, issue 1, 1-14

Abstract: Abstract The only cure of HIV has been achieved in a small number of people who received a hematopoietic stem cell transplant (HSCT) comprising allogeneic cells carrying a rare, naturally occurring, homozygous deletion in the CCR5 gene. The rarity of the mutation and the significant morbidity and mortality of such allogeneic transplants precludes widespread adoption of this HIV cure. Here, we show the application of CRISPR/Cas9 to achieve >90% CCR5 editing in human, mobilized hematopoietic stem progenitor cells (HSPC), resulting in a transplant that undergoes normal hematopoiesis, produces CCR5 null T cells, and renders xenograft mice refractory to HIV infection. Titration studies transplanting decreasing frequencies of CCR5 edited HSPCs demonstrate that

Date: 2025
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DOI: 10.1038/s41467-025-55873-3

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