Human ^Aγ-globin expression in normal murine red cells.

(A) FACS analysis for human γ-globin expression (HbF-PE fluorescence) in the red cells of normal mice 17 weeks following transplantation with normal BM cells transduced with the d432β-^Aγ 3′RE lentiviral vector. Shown at top is a histogram for γ-globin expression (solid lines) in the red cells of a β-thalassemic mouse harboring 2 copies of a β-spectrin promoter-driven γ-globin transgene (THAL 2γ). Below are histograms for γ-globin expression in the red cells of mice that underwent transplantation (mouse number indicated at right). The dotted line indicates the staining profile of red cells from a control mouse that did not undergo transplantation. (B) RNase protection analysis for the levels of vector-encoded^Aγ-globin and endogenous murine α-globin mRNAs. RNA (0.25 μg) from reticulocytes of the mice indicated above each lane was hybridized to a ³²P-labeled antisense riboprobe that protects both exon 2 of the γ-globin gene and exon 2 of the murine α-globin gene. The relative γ/α ratio, indicated below each lane, was obtained by dividing the γ-globin signal (hγ) by the α-globin signal (mα). Neg indicates negative control mouse; Tg, sample from the above-described β-thalassemic/γ-globin transgenic mouse.