Figure 6.

BCL11A-Erythroid Enhancer KO in Adult Thalassemic Cells from Thalassemia Patients

(A) Erythroid (BFU-E) and non-erythroid (CFU-GM) colonies from untransfected (UNTR) and enhancer-edited thalassemic cells. The error bars represent the SEM of triplicate samples. (B) Morphology of erythroid cells at the endpoint of the erythroid culture (day 18). (C) Indel percentage before and after erythroid differentiation of wild-type (WT) and thalassemic cells. The error bars represent the SEM of 6 thalassemic samples. (D) Percentage of enucleation on day 18 of erythroid differentiation. (E) ROS in normal and thalassemic enucleated and nucleated cells (day 20 of erythroid differentiation). (F) HPLC traces of unedited (green) and enhancer-edited (red) erythroid wild-type and thalassemic cells on day 20 of erythroid differentiation (except for the last bottom graph on day 11, showing a higher presence of β-globin compared with day 20 in the same sample). Genotype, top row (left to right): WT, IVSI-1/IVSI-1, CD39/CD39, CD39/ IVSI-110; bottom row (left to right): CD39/ IVSI-110, IVSI-110/ IVSI-110, IVSII-745/IVSII-745. The data in (A), (D), and (E) are from the *β⁺/β⁺ sample. The data in (C) and (F) represent all analyzed samples.