P. Sova

An Integrated Gene Therapy Strategy for γ-Globin Addition and HbF Reactivation in β-Thalassemia

Background: β-Thalassemia and other β-hemoglobinopathies arise from defective β-globin production, and reactivating fetal hemoglobin (HbF) is a well-established strategy to ameliorate disease severity. Two principal gene-therapy approaches—erythroid-specific γ-globin gene addition and shRNA/shmiRNA-mediated knockdown o...