High-Throughput Screening Identifies Kinase Inhibitors That Increase Dual Adeno-Associated Viral Vector Transduction In Vitro and in Mouse Retina

por Maddalena, Andrea, Dell'Aquila, Fabio, Giovannelli, Pia, Tiberi, Paola, Wanderlingh, Luca Giorgio, Montefusco, Sandro, Tornabene, Patrizia, Iodice, Carolina, Visconte, Feliciano, Carissimo, Annamaria, Medina, Diego Luis, Castoria, Gabriella, Auricchio, Alberto
Publicado 2018

Liver‐directed gene therapy for ornithine aminotransferase deficiency

por Boffa, Iolanda, Polishchuk, Elena, De Stefano, Lucia, Dell'Aquila, Fabio, Nusco, Edoardo, Marrocco, Elena, Audano, Matteo, Pedretti, Silvia, Caterino, Marianna, Bellezza, Ilaria, Ruoppolo, Margherita, Mitro, Nico, Cellini, Barbara, Auricchio, Alberto, Brunetti‐Pierri, Nicola
Publicado 2023

Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B

por Ferla, Rita, Dell’Aquila, Fabio, Doria, Monica, Ferraiuolo, Maria, Noto, Alessia, Grazioli, Fabiana, Ammendola, Virginia, Testa, Francesco, Melillo, Paolo, Iodice, Carolina, Risca, Giulia, Tedesco, Novella, le Brun, Pierre Romain, Surace, Enrico Maria, Simonelli, Francesca, Galimberti, Stefania, Valsecchi, Maria Grazia, Marteau, Jean-Brice, Veron, Philippe, Colloca, Stefano, Auricchio, Alberto
Publicado 2023