In Vitro Induction of the Gamma Globin Gene in Erythroid Cells Derived from CD133+ by Thalidomide and Sodium Butyrate

Induction of fetal hemoglobin (HbF) is one of the most promising therapies of β-thalassemia and sickle cell disease, as HbF permanent induction can substantially ameliorate the clinical symptoms of these genetic disorders. Several classes of therapeutics have been implemented to induce γ-globin expression in beta-thalassemia patients and subsequently to raise total hemoglobin levels. Sodium butyrate and Talidomide are two of the known agents. This study was conducted on erythroid cells differentiated from cord blood CD133 + stem cells. Four groups were designed and gamma globin was induced by thalidomide and sodium butyrate treatment (100 μM concentrations). Gamma gene expression was evaluated using Quantitative Real Time PCR techniques. Among the treated groups with sodium butyrate and thalidomide, it was shown that although these two agents both affected the induction of gamma and beta genes, thalidomide had a greater potency on beta cluster genes than sodium butyrate. Our results showed that the pharmaceutical regime of thalidomide and sodium butyrate could be useful for the treatment of β-Thalassemia and sickle cell patients because of the induction of the beta gene cluster.