Fig. 3: UBA1^mut hematochimeric mice preserve myeloid and NK cell outputs but have poor lymphopoietic potential.

a, Schematic representation of the in vivo transplantation experiment. b, Percentage of human cells in PB, spleen and BM of hematochimeric mice transplanted with UBA1^mut or UBA1^wt HSPCs (n = 12 and 11). The median is shown with the IQR. c–e, Percentage of human B cells (c), myeloid cells (d) and NK cells (e) within total cells in PB and hematopoietic organs of mice from b (n = 12 and 11). The median is shown with the IQR. f, Percentage of human HSPCs in the BM of mice from b (n = 12 and 11). The median is shown with the IQR. g, Percentage of cells within human CD34⁺ HSPCs expressing the CD19 or CD13 markers, or none of them, in mice from b (n = 12 and 11). Values given are mean ± s.e.m. h,i, Percentage of UBA1^mut cells across hematopoietic populations in spleen (h) and BM (i) of mice from b (n = 12, 11). The median is shown with the IQR. j, Percentage of cells bearing insertions and deletions in the infused HSPC edited product (n = 1) and in the spleen and BM of hematochimeric mice 14 weeks after transplantation (n = 4 per group). The median is shown with the IQR. i, The Kruskal–Wallis test was used and, for all other panels, the Mann–Whitney U-test. *P < 0.05; **P < 0.01; ***P < 0.001; ****P < 0.0001. Ctrl, control. Panel a created with BioRender.com.