Figure 6.

Maintained beta-thalassemia phenotype correction in secondary ALS20 Hbb^th3/+ chimeras

(A and B) Flow cytometry assessment of Ter119⁺ erythroid cells (hematopoietic progenitors - > RBC) and their distribution by CD44⁺ and forward scatter (FCS) in the spleens and bone marrow, respectively, of a primary ALS20 Hbbt^h3/+ chimera (left column) and three secondary chimeras (right, other three columns) that were generated with the bone marrow harvested from a primary chimera. (C) Values of VCN, chimeric hemoglobin % (resulting from the association of two human beta-globin chains and two mouse alpha-globin chains), and hematopoietic values from complete blood cell counts of the secondary chimeras (n = 3 for each group) from two independent transplants normalized by values of primary chimeras (indicated by the red line at 1, for individual primary chimeras), with a 1:3 ratio between primary and secondary chimeras analyzed in two independent transplants (transplant A and B). (D) Images of spleens and relative spleen/body weight ratios in untreated Hbb^th3/+ chimera, primary ALS20 treated Hbb^th3/+ chimera, and secondary ALS20 treated Hbb^th3/+ chimeras. (E) Chromatographic separation of chimeric hemoglobin and mouse hemoglobin (endogenous mouse α₂β₂) of mice represented in (A) and (B).