Multiplex base editing of BCL11A regulatory elements to treat sickle cell disease
Letizia Fontana, Pierre Martinucci, Simone Amistadi, Tristan Félix, Margaux Mombled, Alexandra Tachtsidi, Guillaume Corre, Anne Chalumeau, Giulia Hardouin, Jeanne Martin, Oriana Romano, Mario Amendola, Panagiotis Antoniou, Annarita Miccio
This study uses multiplex base editing to target BCL11A enhancers, increasing fetal hemoglobin production and reducing sickle cell disease symptoms without causing significant genomic damage.
Cell Reports Medicine · Sep 27, 2025Peer-reviewedMolecular Biology