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Enhanced γ-globin reactivation and sickle cell correction through a repressor-to-activator motif switch in the <i>HBG1/2</i> promoters

2026-04-09

Abstract excerpt

<h4>ABSTRACT</h4> Sickle cell disease (SCD) is caused by the production of an abnormal adult hemoglobin that generates sickle-shaped red blood cells (RBCs). Transplantation of autologous genetically corrected hematopoietic stem/progenitor cells (HSPCs) represents a promising therapy. Persistent fetal hemoglobin expression improves SCD. Here, we engineered the fetal HBG1/2 promoters by replacing the BCL11A repres...

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Literature Corpus work
f550ab87-6117-5b9d-87d4-492717546ad7
DOI
10.64898/2026.04.07.716887
Open publication

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Enhanced γ-globin reactivation and sickle cell correction through a repressor-to-activator motif switch in the <i>HBG1/2</i> promotersDOI 10.64898/2026.04.07.716887
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