Base editing of β0 thalassemia mutations as a therapeutic strategy for β-hemoglobinopathies: efficacy and genotoxicity studies
Giulia Hardouin, Pierre Martinucci, Samantha Scaramuzza, Panagiotis Antoniou, Federico Corradi, Alexandra Tachtsidi, Guillaume Corre, Margaux Mombled, Sandra Manceau, Cécile Rouillon, Cécile Masson, Laure Joseph, Isabelle Thuret, NathalY Network, Eric Allemand, , Annarita Miccio
Preclinical safety and efficacy studies of a new gene therapy approach for patients with severe β-hemoglobinopathies.
bioRxiv · Aug 21, 2025 · PreprintGenetics