BloodMolecular BiologyPeer-reviewed journal article
A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease
Anne ChalumeauiD, Maria Bou DamesiD, Letizia FontanaiD, Simone AmistadiiD, Panagiotis AntoniouiD, Priyanka Loganathan, Margaux MomblediD, Guillaume CorreiD, Martin PeterkaiD, Mario AmendolaiD, Carine GiovannangeliiD, Marcello MarescaiD, Annarita MiccioiD, Mégane BrussoniD
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.
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- 10
- FWCI
- 6.43
- Field percentile
- top 3%
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