Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI

por Ferla, Rita, Alliegro, Marialuisa, Marteau, Jean-Brice, Dell’Anno, Margherita, Nusco, Edoardo, Pouillot, Severine, Galimberti, Stefania, Valsecchi, Maria Grazia, Zuliani, Vincent, Auricchio, Alberto
Publicado 2017

Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors

por Ferla, Rita, Alliegro, Marialuisa, Dell’Anno, Margherita, Nusco, Edoardo, Cullen, John M., Smith, Stephanie N., Wolfsberg, Tyra G., O’Donnell, Patricia, Wang, Ping, Nguyen, Anh-Dao, Chandler, Randy J., Chen, Zelin, Burgess, Shawn M., Vite, Charles H., Haskins, Mark E., Venditti, Charles P., Auricchio, Alberto
Publicado 2020

Therapeutic homology-independent targeted integration in retina and liver

por Tornabene, Patrizia, Ferla, Rita, Llado-Santaeularia, Manel, Centrulo, Miriam, Dell’Anno, Margherita, Esposito, Federica, Marrocco, Elena, Pone, Emanuela, Minopoli, Renato, Iodice, Carolina, Nusco, Edoardo, Rossi, Settimio, Lyubenova, Hristiana, Manfredi, Anna, Di Filippo, Lucio, Iuliano, Antonella, Torella, Annalaura, Piluso, Giulio, Musacchia, Francesco, Surace, Enrico Maria, Cacchiarelli, Davide, Nigro, Vincenzo, Auricchio, Alberto
Publicado 2022