Abstract
Reactivation of silent fetal or embryonic genes could be used for the treatment of genetic diseases caused by mutations of genes normally expressed during the adult stage of development. A paradigm of this approach is the activation of fetal hemoglobin synthesis in adult individuals and its use in the treatment of β chain hemoglobinopathies. The current understanding of the molecular control of the β globin locus is reviewed, as are the cellular and molecular basis of induction of fetal hemoglobin in the adult and the approaches used for stimulation of fetal hemoglobin synthesis in patients with β chain hemoglobinopathies.
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