Fetal hemoglobin reactivation is a gene therapy strategy for hemoglobinopathies that exploits the developmentally regulated fetal-to-adult globin switch, in which a repressor gene normally silences fetal hemoglobin expression after infancy. Lentiviral-vector-delivered RNA interference (short hairpin RNA) knocks down expression of this repressor in a lineage-restricted (erythroid-specific) manner, reactivating fetal hemoglobin production to compensate for defective adult hemoglobin. This belongs to gene therapy and molecular hematology, applying tissue-specific gene silencing to reverse a developmentally programmed switch rather than correcting the underlying mutated gene directly.
Fetal hemoglobin reactivation is a gene therapy strategy for hemoglobinopathies that exploits the developmentally regulated fetal-to-adult globin switch, in which a repressor gene normally silences f…