A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease
Anne Chalumeau, Maria Bou Dames, Letizia Fontana, Simone Amistadi, Panagiotis Antoniou, Priyanka Loganathan, Margaux Mombled, Guillaume Corre, Martin Peterka, Mario Amendola, Carine Giovannangeli, Marcello Maresca, Annarita Miccio, Mégane Brusson
This paper describes a prime editing strategy to modify γ-globin promoters in HSPCs to increase HbF expression for sickle cell disease treatment.
Blood · Aug 26, 2025Peer-reviewedMolecular Biology