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Analysis of α/β-globin ratios in transduced primary β°/β+-thalassemia erythroid cells
CD34+ cells from a transfusion-dependent individual with β°/β+-thalassemia were transduced (n = 2) with the BB305, LVβ-shSCR, and LVβ-shα2 vectors at low MOI, grown in methylcellulose for 2 weeks, and analyzed by qPCR, qRT-PCR, and HPLC for VCN determination and globin gene expression. (A) Correlation of the βT87Q-globin chain fraction relative to VCN. (B) α/β-mRNA ratios relative to UT cells.
