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Effective gene therapy for beta-thalassemia major (beta-TM) requires consistent, high expression of human beta-globin (hbeta-globin) in red blood cells (RBCs). Several groups have now shown that(More)
Effective gene therapy for β-thalassemia major (β-TM) requires consistent, high expression of human β-globin (hβ-globin) in red blood cells (RBCs). Several groups have now shown that lentiviral (LV)(More)