Manipulation of Developmental γ-Globin Expression: an Approach for Healing Hemoglobinopathies
β-Hemoglobinopathies are highly prevalent inherited blood disorders caused by quantitative or structural defects in β-globin chains of adult hemoglobin. Inadequate curative options for these disorders have prompted the development of alternative therapies, among which reactivation of developmentally silenced fetal γ-globin holds great promise. The review by Venkatesan et al. (e00253-20) summarizes the natural mechanisms (hereditary persistence of fetal hemoglobin mutations and single nucleotide polymorphisms) and pharmacological agents for γ-globin derepression. In addition, the review sheds light on various γ-globin-inducing gene therapy approaches adopted and the clinical progress achieved in the field.
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