Figure 5.
Studies with Human CD34+ Cells
(A) Schematic of the experiment. Human CD34+ cells were transduced ex vivo, and 24 hr later, transplanted into sublethally irradiated NOG mice. Mice that received the HDAd-γ-globin/mgmt vector were treated with O6-BG/BCNU as indicated. 10 weeks after transplantation, mice were sacrificed, and bone-marrow-derived human CD34+ cells were subjected to erythroid in vitro differentiation. (B) Percentage of human cells expressing γ-globin at day 14 and 18 of erythroid differentiation. (C) Adult (β-globin) and γ-globin mRNA in cells at day 14 of in vitro differentiation. (D) HPLC for globin chains. γ/α-globin and beta/α-globin percentages at day 18 of erythroid differentiation. (E) Percentage of human-lineage-marker-positive cells in the bone marrow at week 9 after transplantation.