Figure 3.

β-AS3 HS4 and β-AS3 Transduction Efficiencies in Long-Term Repopulating SCD HSCs

(A) Engraftment of human cells in NSG mice transplanted with mock- and LV-transduced SCD BM CD34⁺ cells (MOI = 360, n = 5 mice for each group) and HD mPB (n = 3) and HD CB (n = 2) HSPCs 15 weeks after transplantation. Engraftment is represented as percentage of human CD45⁺ cells in the total murine and human CD45⁺ cell population, in BM, spleen, and thymus. Values shown are mean ± SEM; ns, not significant (one-way ANOVA test). (B) Human hematopoietic progenitor content in BM mononuclear cells (MNC) derived from mice transplanted with either mock- or β-AS3 HS4- and β-AS3-transduced SCD BM HSPCs. We plotted the percentage of BM MNC giving rise to BFU-E and CFU-GM. Values shown are mean ± SEM of four samples per condition. (C) VCN/cell in pools of BFU-E- and CFU-GM-derived from total BM MNC, obtained from mice injected with SCD BM HSPCs transduced with β-AS3 HS4 (n = 2 and n = 3 for BFU-E and CFU-GM, respectively) and β-AS3 (n = 3 and n = 4 for BFU-E and CFU-GM, respectively) LVs at an MOI of 360. Values shown are mean ± SEM. *p ≤ 0.05; **p < 0.01; ****p < 0.0001 (unpaired t test).