Li, Jing Lai, Yongrong Shi, Lingling BCL11A Down-Regulation Induces γ-Globin in Human β-Thalassemia Major Erythroid Cells <p>Fetal hemoglobin (Hb F, α2γ2) is a potent genetic modifier of the severity of β-thalassemia (β-thal) and sickle cell anemia. Differences in the levels of HbF that persist into adulthood affect the severity of sickle cell disease and the β-thal syndromes. B-cell lymphoma 11 A (BCL11A) is a potent silencer of HbF. Here, we reactivated γ-globin expression by down-regulating BCL11A to alleviate anemia in the β-thal major (β-TM) patients. BCL11A were down-regulated by lentiviral RNAi (RNA interference) in the K562 cell line and an <i>in vitro</i> culture model of human erythropoiesis in which erythroblasts are derived from the normal donor mononuclear cells (MNC) or β-TM MNC. The expression of γ-globin were analyzed by qPCR (quantitative real-time polymerase chain reaction) and Western blot techniques. Our data showed that down-regulation of BCL11A induces γ-globin production in the K562 cell line and human erythrocytes from normal donors and β-TM donors, without altering erythroid maturation. This is the first report on γ-globin induction by down-regulation of BCL11A in human erythroblasts derived from β-TM.</p> B-cell lymphoma 11A (BCL11A);β-thalassemia major (β-TM);γ-globin 2019-01-17
    https://tandf.figshare.com/articles/journal_contribution/BCL11A_Down-Regulation_Induces_-Globin_in_Human_-Thalassemia_Major_Erythroid_Cells/7599200
10.6084/m9.figshare.7599200.v1