Sodium Butyrate Enhances Fetal Globin Gene Expression in Erythroid Progenitors of Patients With Hb SS and ß Thalassemia

• Published in: Blood Vol. 74; no. 1; pp. 454 - 459
• Main Authors: Perrine, Susan P., Miller, Barbara A., Faller, Douglas V., Cohen, Ruth A., Vichinsky, Elliott P., Hurst, Deborah, Lubin, Bertram H., Papayannopoulou, Thalia
• Format: Journal Article
• Language: English
• Published: Elsevier Inc 01.07.1989
• ISSN: 0006-4971; 1528-0020
• DOI: 10.1182/blood.V74.1.454.454

Increasing the expression of the γ globin genes is considered a useful therapeutic approach to the β globin diseases. Because butyrate and α-amino-n-butyric acid (ABA) augment γ globin expression in normal neonatal and adult erythroid progenitors, we investigated the effects of sodium butyrate and ABA on erythroid progenitors of patients with β thalassemia and sickle cell anemia who might benefit from such an effect. Both substances increased fetal hemoglobin (Hb F) expression in Bfu-e from 7% to 30% above levels found in control cultures from the same subjects with sickle cell anemia. The fraction of cultured erythroblasts producing Hb F increased more than 20% with sodium butyrate treatment in 70% of cultures. In most cultures, this produced >20% total Hb F and >70% F cells, levels which have been considered beneficial in ameliorating clinical symptoms. Alpha: non-alpha (α-non-α) imbalance was decreased by 36% in erythroid progenitors of patients with β thalassemia cultured in the presence of butyrate compared with control cultures from the same subjects. These data suggest that sodium butyrate may have therapeutic potential for increasing γ globin expression in the β globin diseases.