Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases
Esposito, Federica, Dell’Aquila, Fabio, Rhiel, Manuel, Auricchio, Stefano, Chmielewski, Kay Ole, Andrieux, Geoffroy, Ferla, Rita, Horrach, Paula Sureda, Padmanabhan, Arjun, Di Cunto, Roberto, Notaro, Simone, Santeularia, Manel Llado, Boerries, Melanie, Dell’Anno, Margherita, Nusco, Edoardo, Padula, Agnese, Nutarelli, Sofia, Cornu, Tatjana I., Sorrentino, Nicolina Cristina, Piccolo, Pasquale, Trapani, Ivana, Cathomen, Toni, Auricchio, Alberto
Published in Cell reports. Medicine (16.07.2024)
Published in Cell reports. Medicine (16.07.2024)
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Liver-Directed Adeno-Associated Virus-Mediated Gene Therapy for Mucopolysaccharidosis Type VI
Brunetti-Pierri, Nicola, Ferla, Rita, Ginocchio, Virginia Maria, Rossi, Alessandro, Fecarotta, Simona, Romano, Roberta, Parenti, Giancarlo, Yildiz, Yilmaz, Zancan, Stefano, Pecorella, Valentina, Dell'Anno, Margherita, Graziano, Mafalda, Alliegro, Marialuisa, Andria, Generoso, Santamaria, Francesca, Brunetti-Pierri, Raffaella, Simonelli, Francesca, Nigro, Vincenzo, Vargas, Maria, Servillo, Giuseppe, Borgia, Francesco, Soscia, Ernesto, Gargaro, Marco, Funghini, Silvia, Tedesco, Novella, Le Brun, Pierre Romain, Rupar, Charles A, Prasad, Chitra, O'Callaghan, Mar, Mitchell, John J, Danos, Olivier, Marteau, Jean-Brice, Galimberti, Stefania, Valsecchi, Maria Grazia, Veron, Philippe, Mingozzi, Federico, Fallarino, Francesca, la Marca, Giancarlo, Sivri, H Serap, Auricchio, Alberto
Published in NEJM evidence (01.07.2022)
Published in NEJM evidence (01.07.2022)
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Therapeutic homology-independent targeted integration in retina and liver
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
Published in Nature communications (12.04.2022)
Published in Nature communications (12.04.2022)
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Low incidence of hepatocellular carcinoma in mice and cats treated with systemic adeno-associated viral vectors
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
Published in Molecular therapy. Methods & clinical development (12.03.2021)
Published in Molecular therapy. Methods & clinical development (12.03.2021)
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Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI
Ferla, Rita, Alliegro, Marialuisa, Marteau, Jean-Brice, Dell'Anno, Margherita, Nusco, Edoardo, Pouillot, Severine, Galimberti, Stefania, Valsecchi, Maria Grazia, Zuliani, Vincent, Auricchio, Alberto
Published in Molecular therapy. Methods & clinical development (15.09.2017)
Published in Molecular therapy. Methods & clinical development (15.09.2017)
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