SOX6 Downregulation Induces γ-Globin in Human β-Thalassemia Major Erythroid Cells.

ABSTRACT:

Background

Fetal hemoglobin (HbF; α₂γ₂) is a potent genetic modifier of the severity of β-thalassemia and sickle cell anemia. Differences in the levels of HbF that persist into adulthood affect the severity of sickle cell disease and the β-thalassemia syndromes. Sry type HMG box (SOX6) is a potent silencer of HbF. Here, we reactivated γ-globin expression by downregulating SOX6 to alleviate anemia in the β-thalassemia patients.

Methods

SOX6 was downregulated by lentiviral RNAi (RNA interference) in K562 cell line and an in vitro culture model of human erythropoiesis in which erythroblasts are derived from the normal donor mononuclear cells (MNC) or β-thalassemia major MNC. The expression of γ

SUBMITTER: Li J 

PROVIDER: S-EPMC5733236 | biostudies-literature | 2017

REPOSITORIES: biostudies-literature