Enhanced γ-globin reactivation and sickle cell correction through a repressor-to- activator motif switch in the HBG1/2 promoters
Anne Chalumeau, Panagiotis Antoniou, Maria Bou Dames, Pierre Martinucci, Elena Retana, Pragya Gupta, Mike A. Firth, Muralidhar Reddivari, Aathira Sarath Chandran, Jonathan S. Yen, Martin Peterka, Marcello Maresca, Mégane Brusson, Annarita Miccio
This paper demonstrates that replacing the BCL11A repressor binding site with a TAL1:GATA1 activator motif in HBG1/2 promoters enhances γ-globin reactivation and corrects sickle cell disease in hematopoietic stem/progenitor cells.
Molecular Therapy — Nucleic Acids · Jun 6, 2026Peer-reviewedBioengineering