A Short-Term Trial of Butyrate to Stimulate Fetal-Globin-Gene Expression in the β-Globin Disorders

Sickle cell anemia and the β-thalassemia syndromes are prevalent disorders caused by mutations affecting the adult-globin (beta-globin) chain of hemoglobin A (the chains designated as α 2 β 2 ) 1 – 5 . Sickle cell anemia was the first disease to be characterized at the molecular level 3 – 5 . Definitive treatment for the underlying condition has not followed, however, except for bone marrow transplantation in the few patients for whom there are appropriate donors. Increased production or prolonged expression of fetal globin (γ-globin) in sufficient quantities can ameliorate both disorders 6 – 13 . Chemotherapeutic agents, including azacitidine, cytarabine, and hydroxyurea, have been . . .