TALEs activate fetal-globin expression in human CD34 differentiated erythroid cells. (A) New vector design for fetal-globin gene action. The 1.7-kb shortened LCR enhancer (called dLCR) and β-globin promoter were used to driveTALE7b and TALE11 expression. 400v5m3 is the lenti-γ globin vector previously developed in this laboratory for the gene therapy of SCD. (B). Human CD34 cells subjected to 3-phase erythroid differentiation at day 19, and harvested cells for RNA. Relative fetal-globin mRNA level was determined by qPCR, using HBG as probe, normalized by HBA amount. The vector copy number (VCN) was determined and depicted in the graph. The statistical significance was calculated by 1-way ANOVA followed by Dunnett’s multiple comparisons test. *P = .01; ****P < .0001. Values are mean ± SEM. (C). Bar graphs show proportions of the fetal-globin protein relative to total globin amount level determined by HPLC.