Selective Advantage of Corrected Cells of the Erythroid Lineage
(A) Representative FACS analysis of BM cells and peripheral RBCs from each group of mice. BM cells were analyzed for Ter-119 expression, and three different groups were differentiated by their SSC and FSC profiles (upper left part): (I) RBCs, (II) intermediate normoblasts, and (III) early normoblasts. BM cells were also analyzed for Gr-1 expression (upper middle part). GFP expression was then analyzed in each population of erythroid (Ter-119) and myeloid (Gr-1) BM cells and in peripheral RBCs (right part).
(B) Ratio of the percentage of GFP expression between the different populations of erythropoietic cells and Gr-1-positive cells. ∗p < 0.01 versus MNDpGFP vector in normal mice; ∗∗p < 0.001 versus EFpGFP vector in normal and CEP mice; ∗∗∗p < 0.001 versus ESpUROS-EFpGFP vector in the different populations of erythropoietic cells of CEP mice.
Values for (B) represent mean ± SD.