Liver-directed lentiviral gene therapy corrects hemophilia A mice and achieves normal-range factor VIII activity in non-human primates

por Milani, Michela, Canepari, Cesare, Liu, Tongyao, Biffi, Mauro, Russo, Fabio, Plati, Tiziana, Curto, Rosalia, Patarroyo-White, Susannah, Drager, Douglas, Visigalli, Ilaria, Brombin, Chiara, Albertini, Paola, Follenzi, Antonia, Ayuso, Eduard, Mueller, Christian, Annoni, Andrea, Naldini, Luigi, Cantore, Alessio
Publicado 2022