Figure 4.
Enforced expression of RPS19 confers a competitive advantage over untransduced Rps19-deficient cells. (A) Experimental strategy to validate the therapeutic potential of RPS19 gene correction. The percentage of transduced (B) total donor-derived white blood cells (CD45.2+GFP+) or (C) myeloid cells (CD45.2+Gr1+CD11b+GFP+) in the peripheral blood before doxycycline administration, 2 weeks and 4 months after doxycycline administration (n = 9–24 per group). (D) BM cellularity of the recipients 17–23 weeks after the doxycycline administration (n = 11–24 per group). (E) The percentage of transduced cells in the hematopoietic stem and progenitor compartments (n = 11–24 per group). Data in (B) are presented as box whisker plots with minimum and maximum values. Error bars in (C) represent standard deviation.