Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease

door Alliegro, Marialuisa, Ferla, Rita, Nusco, Edoardo, De Leonibus, Chiara, Settembre, Carmine, Auricchio, Alberto
Gepubliceerd in Mol Ther (2016)

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

door Ferla, Rita, Alliegro, Marialuisa, Marteau, Jean-Brice, Dell’Anno, Margherita, Nusco, Edoardo, Pouillot, Severine, Galimberti, Stefania, Valsecchi, Maria Grazia, Zuliani, Vincent, Auricchio, Alberto
Gepubliceerd in Mol Ther Methods Clin Dev (2017)

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

door 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
Gepubliceerd in Mol Ther Methods Clin Dev (2020)