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BCL11A-deficient human erythropoiesis is impaired in vitro and after xenotransplantation into mice

28 Feb 2025

Abstract excerpt

ABSTRACT: Genetic depletion of the transcriptional repressor BCL11A in red blood cell precursors alleviates β-hemoglobinopathies by inducing the fetal γ-globin genes. However, additional erythroid genes are regulated by BCL11A and the effects of its deficiency on erythropoiesis are insufficiently described. We discovered that Cas9 disruption of the BCL11A intron 2 erythroid enhancer in CD34+ hematopoietic stem...

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PMID: 40020162
DOI: 10.1182/bloodadvances.2024015574

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