U7.623 lentiviral vector–induced β-globin pre-mRNA repair in erythroid progenitors from patient with IVS2-745/IVS2-1 thalassemia.

(A)RT-PCR. On days 1 and 2 of culture, the cells were transduced for 5 hours with either no virus (lane 1), mock, GFP lentiviral vector (lane 2), or U7.623 lentiviral vector (lane 3). Lane 4 is RNA from normal blood. (B) Immunodetection of hemolysates separated by electrophoresis on cellulose acetate with antihuman hemoglobin antibody (“Materials and methods”). Lane 1, hemoglobin standards; lane 2, (N) normal blood; lane 3, mock-transduced cells; lane 4, cells transduced with GFP lentiviral vector; lane 5, cells transduced with U7.623 lentiviral vector.