Fig. 3: CD33^Δ2-edited CD34⁺ engraft, recapitulate a complete hematopoietic system and display resistance to GO CD33-targeted therapy in vivo.

a Schematic of the experiment was created in BioRender. Du, X. (2025) https://BioRender.com/y35d204. b, c Measure of engraftment by percentage of human CD45⁺ cells and of hematopoietic repopulation by frequency of progenitors myeloid (CD123) and lymphoid (CD10), as well as mature myeloid (CD14) and lymphoid (CD19), and T cells (CD3) within the human CD45 population in peripheral blood at 8 weeks post-transplantation and in the BM at 16 weeks post-transplantation. (Unedited: n = 5, Edited: n = 9, 2 independent experiments, 2 donors, unpaired two-tailed t-test). d On-target editing (HTS analysis) in edited cells kept in vitro (pre-Transplant) or harvested in the BM 16 weeks post-Transplantation. (2 independent experiments, 2 donors, one-way ANOVA, Tukey’s multiple comparisons test) e Left, frequency of CD14⁺ myeloid cells in BM of 12 weeks post-transplanted mice. Right, frequency of CD14⁺CD33⁻ cells in the BM of 12 weeks post-transplanted mice before and one week after Gemtuzumab Ozogamicin (GO) treatment (0.5 ug per mouse). UE, unedited cells. (n = 6, 2 independent experiments, 2 donors, one-way ANOVA, Tukey’s multiple comparisons test). All error bars in this figure show ±SEM. Source data are provided as a Source Data file.