Figure 5.

Engraftment of the Edited Cells in Secondary Recipients

(A) Human cell chimerism in the peripheral blood of secondary recipient NSG mice 10 weeks after transplantation. (B) Human CD45, CD34+CD38lo, and GlyA+ cell chimerism in the bone marrow of secondary recipients 10 weeks after transplantation. (C) Multilineage in vivo differentiation of human unedited and edited cells. (D) Human erythroid (BFU-E) and non-erythroid (CFU-GM) colonies from engrafted cells 10 weeks after transplantation. (E) Indel percentage in bone marrow-derived cells 10 weeks after transplantation. (F) Cell growth of the erythroid cells derived from the secondary recipients’ bone marrow. Proliferative expansion here refers to all donor cells irrespective of editing levels. (G) HbF+ cell frequency in the erythroid cultures. For the erythroid differentiation assays, bone marrow cells of all recipients per group were pooled and plated under erythroid conditions. The error bars represent the SEM of 4 mice.