Figure 3.
Normal engraftment and multilineage differentiation of ADA2-transduced HDs’ CD34+ cells in NSGW41 mice. (A) The average VCN per genome was evaluated in ADA2- and GFP-transduced HSPCs expanded in vitro (LC), in total peripheral blood leukocytes at 7, 14, and 20 weeks posttransplant, and at 20 weeks in the BM and spleen (SPL) of NSGW41 mice receiving LV-ADA2– or LV-GFP–transduced lineage-negative cells (n = 6 mice per group). Longitudinal analysis of human cell chimerism (CD45+ cells; B) and B, T, myeloid, and NK cells (C) in peripheral blood at 7, 14, and 20 posttransplantation. (D) Flow cytometry analysis showing the total number of HSCs and various progenitor populations in the BM of NSGW41 mice. Data are mean ± standard error. **P < .01, ***P < .001. iGranulo, immature granulocytes; mGranulo, mature granulocytes.

Normal engraftment and multilineage differentiation of ADA2-transduced HDs’ CD34+ cells in NSGW41 mice. (A) The average VCN per genome was evaluated in ADA2- and GFP-transduced HSPCs expanded in vitro (LC), in total peripheral blood leukocytes at 7, 14, and 20 weeks posttransplant, and at 20 weeks in the BM and spleen (SPL) of NSGW41 mice receiving LV-ADA2– or LV-GFP–transduced lineage-negative cells (n = 6 mice per group). Longitudinal analysis of human cell chimerism (CD45+ cells; B) and B, T, myeloid, and NK cells (C) in peripheral blood at 7, 14, and 20 posttransplantation. (D) Flow cytometry analysis showing the total number of HSCs and various progenitor populations in the BM of NSGW41 mice. Data are mean ± standard error. **P < .01, ***P < .001. iGranulo, immature granulocytes; mGranulo, mature granulocytes.

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