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

by Albertini, P., Annoni, A., Ayuso, E., Biffi, M., Brombin, C., Canepari, C., Cantore, A., Curto, R., Drager, D., Follenzi, A., Liu, T., Milani, M., Mueller, C., Naldini, L., Patarroyo-White, S., Plati, T., Russo, F., Visigalli, I.
Published 2022