Molecular BiologyCell Reports MedicinePeer-reviewed
Multiplex base editing of BCL11A regulatory elements to treat sickle cell disease
Letizia Fontana, Pierre Martinucci, Simone Amistadi…·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.
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