Fig. 5: Robust CCR5 editing in HSPCs is stable in vivo and gives rise to differentiated myeloid and lymphoid cell lineages lacking CCR5.

A Representative FACS plots demonstrating ablation of surface CCR5 protein in CD33+ myeloid cells (left panel) and CD3+/CD4+ T cells (right panel) in the peripheral blood of xenograft mice 20 weeks post-transplant. B The percentage of CCR5-expressing myeloid cells as measured by flow cytometry in the peripheral blood of mice transplanted with 1 × 106 untouched (n = 15, gray bars), mock edited (GFP gRNA) (n = 19, green bars), or dual gRNA CCR5 edited (n = 19) HSPCs at monthly intervals. **denotes p < 0.01 and statistics generated from the Student’s t-test and p values are two-tailed. Bars represent mean and error bars denote ± SD. C The percentage of CCR5-expressing CD3+ T cells at 20 and 24 weeks post-transplant in xenograft mice with appreciable human T cell numbers from different HSPC transplant groups. **denotes p < 0.01 and statistics generated from the Student’s t-test and p values are two-tailed. Bars represent mean and error bars denote ± SD.