Fig 2: Preservation of engraftment competency of HSPCs post TRIAMF treatment. 7 × 105 viable HSPCs (from the same donor as used in Fig. 2) 1-day post treatment were transplanted into each sub-lethally irradiated recipient mouse via tail vein injection. (a) Human CD45+ chimerism of untreated (n = 4 mice), mock treated (TRIAMF Mock, n = 5 mice), and g1-RNP edited (TRIAMF + RNP, n = 6 mice) in the peripheral blood 8, 12, 16, and 20 weeks post-transplantation. (b) Human CD45+ chimerism in mouse bone marrow 20 weeks post-transplantation. (c) Lineage distribution of human CD45+ cells isolated from mouse bone marrow 20 weeks post-transplantation. (d) Two aliquots of HSPCs from the same donor were processed by TRIAMF. One aliquot of cells was harvested 48 hours after treatment (Pre-Transplant, n = 1) and the other aliquot of cells were engrafted into NGS mice from which hCD45+ cells were harvested from mouse bone marrow 20 weeks after transplantation (Post-Transplant, n = 6 mice). The total mutation rate and top 5 most frequent indel alleles as a percentage of total NGS reads at HBG1 and HBG2 sites were shown. For the Post-Transplant group, indel patterns are the mean of all the mice. (e) Comparison of HbF+ immunostaining of CD235a+/CD71-/low cells after erythroid differentiation of HSPCs 48 hours post treatment (n = 2, technical duplicate) and hCD45+/CD34+ HSPCs harvested from mouse bone marrow 20 weeks post transplantation (n = 4–6 mice).