High-level β-globin expression and preferred intragenic integration after lentiviral transduction of human cord blood stem cells
J. Clin. Invest. Suzan Imren, et al. 114:953 doi:10.1172/JCI21838 [Go to this article.]

Figure 2
Assessment of 16 NOD/SCID mice and 3 NOD/SCID-nu/nu mice transplanted with β^A-T87Q-globin lentivirus-infected cord blood cells. (A) Time course studies of the total number of human hematopoietic (CD45/71⁺) cells generated in two representative experiments. Values are the mean ± SEM for 8 mice in experiment 1 (filled squares) and 11 mice in experiment 2 (filled circles). Of all cells present in the marrow in experiment 1, 20 weeks after transplant, 4% ± 2% were B-lymphoid (CD19/20⁺) cells and 0.7% ± 0.4% were mature granulopoietic (CD15⁺) cells; of all cells present in the marrow in experiment 2, 16 weeks after transplant, 63% ± 4% were B-lymphoid cells and 7% ± 1% were mature granulopoietic cells. (B) PCR analysis to detect provirus-positive hematopoietic colonies produced by human CFCs isolated from the marrow of individual mice (n = 18) at different time points after transplant in a total of five experiments. Values determined at 3 weeks after transplant are not connected to the values determined at later time points because the cells present at these two different times are thought to be derived from different types of repopulating cells (43). The overall proportion of colonies with the β^A-T87Q-globin transgene in all 17 recipients analyzed 11_24 weeks after transplant was 45% ± 6% (range 12%_91%).