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

Chalumeau, A.; Bou Dames, M.; Antoniou, P.; Loganathan, P.; Mombled, M.; Corre, G.; Peterka, M.; Amendola, M.; Giovannangeli, C.; Maresca, M.; Miccio, A.; Brusson, M.

doi:10.1101/2025.01.13.632780

bioRxiv · 10.1101/2025.01.13.632780

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

Chalumeau, A.·Bou Dames, M.·Antoniou, P.·Loganathan, P.·Mombled, M.·Corre, G.·Peterka, M.·Amendola, M.·Giovannangeli, C.·Maresca, M.·Miccio, A.·Brusson, M.

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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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Chalumeau, A., Bou Dames, M., Antoniou, P., Loganathan, P., Mombled, M., Corre, G., Peterka, M., Amendola, M., Giovannangeli, C., Maresca, M., Miccio, A., Brusson, M.. 2025-01-14. A prime editing strategy to rewrite the γ-globin promoters and reactivate fetal hemoglobin for sickle cell disease. https://doi.org/10.1101/2025.01.13.632780

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