Maria Bou Dames on Muck Rack

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A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease

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By Anne Chalumeau, Panagiotis Antoniou, Maria Bou Dames, Priyanka Loganathan
| biorxiv.org
Abstract Fetal hemoglobin (HbF) reactivation is a promising therapy for beta-hemoglobinopathies. We developed a prime editing strategy that introduces multiple mutations in the fetal gamma-globin promoters of patients' hematopoietic stem/progenitor cells (HSPCs), boosting HbF expression and offering alternative therapeutic perspectives. Competing Interest Statement The authors have declared no competing interest.
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