Adenine base editor–mediated correction of the common and severe IVS1-110 (G>A) β-thalassemia mutation

por Hardouin, Giulia, Antoniou, Panagiotis, Martinucci, Pierre, Felix, Tristan, Manceau, Sandra, Joseph, Laure, Masson, Cécile, Scaramuzza, Samantha, Ferrari, Giuliana, Cavazzana, Marina, Miccio, Annarita
Publicado 2023

Editing a γ-globin repressor binding site restores fetal hemoglobin synthesis and corrects the sickle cell disease phenotype

por Weber, Leslie, Frati, Giacomo, Felix, Tristan, Hardouin, Giulia, Casini, Antonio, Wollenschlaeger, Clara, Meneghini, Vasco, Masson, Cecile, De Cian, Anne, Chalumeau, Anne, Mavilio, Fulvio, Amendola, Mario, Andre-Schmutz, Isabelle, Cereseto, Anna, El Nemer, Wassim, Concordet, Jean-Paul, Giovannangeli, Carine, Cavazzana, Marina, Miccio, Annarita
Publicado 2020

Base-editing-mediated dissection of a γ-globin cis-regulatory element for the therapeutic reactivation of fetal hemoglobin expression

por Antoniou, Panagiotis, Hardouin, Giulia, Martinucci, Pierre, Frati, Giacomo, Felix, Tristan, Chalumeau, Anne, Fontana, Letizia, Martin, Jeanne, Masson, Cecile, Brusson, Megane, Maule, Giulia, Rosello, Marion, Giovannangeli, Carine, Abramowski, Vincent, de Villartay, Jean-Pierre, Concordet, Jean-Paul, Del Bene, Filippo, El Nemer, Wassim, Amendola, Mario, Cavazzana, Marina, Cereseto, Anna, Romano, Oriana, Miccio, Annarita
Publicado 2022