Figure 5.

Spleen phenotype correction in ALS20- and CV-I-treated primary Hbbt^h3/+ chimeras

(A) Flow cytometry assessment of the proportion of Ter119⁺ erythroid cells (hematopoietic progenitors - > RBC) and their distribution by CD44⁺ and forward scatter (FCS) in the spleens of Hbb^th3/+ and Hbb^+/+ control mice. (B) Flow cytometry assessment of the spleens of Hbb^th3/+ mice that were treated with either ALS20 or CV-I. (C) Ratio of spleen over body weight (left). (D) Proportion of R1 (pro-erythroblasts), R2 (basophilic), R3 (polychromatic), R4 (orthochromatic and reticulocytes), and R5 (mature erythrocytes) in the spleen of Hbb^+/+ (n = 3), Hbb^th3/+ (n = 21), and in Hbb^th3/+ mice treated with ALS20-T87Q (VCN = 1 ± 0.1) or CV-I (VCN = 1 ± 0.2) (n = 7 and 6 for ALS20 and CV-I treated groups, respectively). Groups were tested by non-parametric ANOVA and multiple comparison analyses. Q1 through 4 gates represent live cells gated for CD71 and Ter119 markers. Asterisks indicate p values referred to ALS20, while pound signs indicate p values referred to CV-I. #p ≤ 0.05; ## or ∗∗p ≤ 0.005; ∗∗∗p ≤ 0.0005, and ∗∗∗∗p < 0.0001.