Amendola, Mario
Amendola, Mario
Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression
2022-01-01 Antoniou, P.; Hardouin, G.; Martinucci, P.; Frati, G.; Felix, T.; Chalumeau, A.; Fontana, L.; Martin, J.; Masson, C.; Brusson, M.; Maule, G.; Rosello, M.; Giovannangeli, C.; Abramowski, V.; de Villartay, J. -P.; Concordet, J. -P.; Del Bene, F.; El Nemer, W.; Amendola, M.; Cavazzana, M.; Cereseto, A.; Romano, O.; Miccio, A.
Combination of lentiviral and genome editing technologies for the treatment of sickle cell disease
2022-01-01 Ramadier, S.; Chalumeau, A.; Felix, T.; Othman, N.; Aknoun, S.; Casini, A.; Maule, G.; Masson, C.; De Cian, A.; Frati, G.; Brusson, M.; Concordet, J. -P.; Cavazzana, M.; Cereseto, A.; El Nemer, W.; Amendola, M.; Wattellier, B.; Meneghini, V.; Miccio, A.
Correction of β-thalassemia by CRISPR/Cas9 editing of the α-globin locus in human hematopoietic stem cells
2021-01-01 Pavani, Giulia; Laurent, Marine; Amor, Fatima; Cantelli, Erika; Chalumeau, Anne; Maule, Giulia; Tachtsis, Alexandra; Concordet, Jean-Paul; Cereseto, Anna; Mavilio, Fulvio; Ferrari, Giuliana; Amendola, Mario
Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype
2020-01-01 Weber, L.; Frati, G.; Felix, T.; Hardouin, G.; Casini, A.; Wollenschlaeger, C.; Meneghini, V.; Masson, C.; de Cian, A.; Chalumeau, A.; Mavilio, F.; Amendola, M.; Andre-Schmutz, I.; Cereseto, A.; El Nemer, W.; Concordet, J. -P.; Giovannangeli, C.; Cavazzana, M.; Miccio, A.