Fig. 6

Robust β-globin production at the protein level with the forward-oriented β-globin vector in erythroid cells differentiated from plerixafor-mobilized CD34⁺ cells in SCD. a SCD CD34⁺ cells were transduced with the optimized forward-oriented β-globin (or βT87Q-globin including an anti-sickling mutation T87Q) vectors at MOI 50 and the transduced cells were differentiated into erythroid cells for 2 weeks. β-globin production was evaluated by hemoglobin electrophoresis (HbA bands) and/or RP-HPLC (%), and VCNs were evaluated by quantitative PCR. b Globin protein peaks were detected by RP-HPLC. c Globin-expression levels per vector were evaluated by regression lines between VCNs and βT87Q-globin amounts at the protein level. The βT87Q-globin amounts were calculated by the area under the curve in RP-HPLC, compared with α-globin. **p < 0.01 and *p < 0.05 evaluated by Tukey’s HSD test among regression lines. All globin protein analysis experiments were performed in a single run