Figure 3.

βAS3m/miR7m LV-transduced HSPCs engraft and differentiate in NBSGW mice

(A) Human SCD HSPCs were either mock-transduced (mock) or transduced with βAS3 or βAS3m/miR7m LVs at an MOI of 125 for 24 h. After transduction, cells were transplanted into NBSGW mice. After 18 weeks, chimerism, cell lineage reconstitution, and VCN/cell were evaluated. (B) Frequency (%) of human CD45⁺ (hCD45⁺) cells in the BM, spleen, and thymus of the transplant recipients (n = 4–6 mice per group). (C) Frequencies (%) of CD11b⁺, CD14⁺, and CD15⁺ cells in the hCD45⁺ population in the BM and in the spleen of the transplant recipients (n = 4–6 mice per group). (D) Frequencies (%) of CD3⁺ and CD19⁺ cells in the hCD45⁺ population in the BM and in spleen of the transplant recipients (n = 4–6). (E) Frequencies (%) of CD235a⁺ cells in the negative human (h) and mouse (m) CD45 population in the BM and spleen of the transplant recipients (n = 4–6 mice/group). (B–E) Kruskal-Wallis test, ns. (F) VCN/cell measured by ddPCR in the input cells (bulk of BFU-Es and CFU-GMs) and in the BM of transplant recipients (n = 3–6 mice/group). No significant statistical difference in VCN/cell was observed between the two LVs in BM cells (unpaired t test).