FETAL GENE REACTIVATION

Reactivation of silent fetal or embryonic genes could be used for the treatment of genetic diseases caused by mutations of genes normally ex pressed during the adult stage of development. A paradigm of this appr oach is the activation of fetal hemoglobin synthesis in adult individu als and its use in the treatment of beta chain hemoglobinopathies. The current understanding of the molecular control of the beta globin loc us is reviewed, as are the cellular and molecular basis of induction o f fetal hemoglobin in the adult and the approaches used for stimulatio n of fetal hemoglobin synthesis in patients with beta chain hemoglobin opathies.