Fig. 4: Phenotypic rescue of SCD characteristics in ex vivo-differentiated PE3max-treated human erythroid progeny from HSPCs transplanted into immunodeficient mice.
a, Percentage of β-like globins measured by ion exchange HPLC in CD235a+ cells from human SCD patient cells at 17 weeks post-transplantation. Bars and error bars represent cumulative averages + s.d. of each protein across 2–5 mice per donor. b, Representative phase-contrast images of reticulocytes derived from 2xPE3max-treated or untreated SCD HSPCs incubated for 24 h with 2% oxygen. Scale bars, 100 µM. c, Quantification of sickled reticulocytes from images as in b from over 400 randomly selected cells per image. Cells were counted by a blinded observer for all conditions. Lines with error bars represent mean ± s.d., with each dot representing the percentage of sickled cells in one image from the specified donor. Significance was determined with one-sided multiple-paired t-tests correcting for multiple comparisons using the Holm-Šídák correction method. All P values are indicated.
