Enhanced Multilineage Repopulating Activity of IK6-Transduced Human CD34+ CB Cells
(A) Experimental design.
(B) Ratio of ABL1/B2M normalized IKZF1 transcript levels in transduced CD34+ human cell subsets generated in NSG mice (mean ± SEM, four mice).
(C) Representative flow cytometric profiles of total IKAROS protein in human cells generated in five NSG mice (dotted line shows unstained control).
(D) Paired comparisons of total IK6- and control-derived human CD45+ cells in individual mice transplanted 8–10 weeks previously (24 mice from five cohorts).
(E and F) Ten-week posttransplant levels of human CD19+ and surface immunoglobulin M+ (B), CD3+ (T), and CD33+ (GM) cells in the same mice shown in (D).
(G) Platelet levels in the blood of NSG mice transplanted 4–10 weeks previously (mean ± SEM; seven mice from two cohorts).
(H) Colonies derived from equal numbers of IK6- and control-transduced myeloerythroid progenitors sorted from the BM of NSG mice transplanted 10 weeks previously (mean ± SEM; three mice).
(I) Levels of different human CD34+ subsets in the BM of primary NSG mice transplanted 10 weeks previously (mean ± SEM, 14 mice).
Progenitor-enriched fractions: multipotent, CD34+CD38−; lymphoid, CD34+CD38+CD10+; myeloerythroid, CD34+CD38+CD45RA−FLT3+; granulopoietic, CD34+CD38+CD45RA+FLT3+. BFU-E, burst-forming unit-erythroid; CFC-GM, colony-forming cell-GM. ∗p < 0.05; ∗∗p < 0.01; ∗∗∗p < 0.001. See also Figure S2.