Impact of age at administration, lysosomal storage, and transgene regulatory elements on AAV2/8-mediated rat liver transduction. by Gabriella Cotugno, Patrizia Annunziata, Maria Vittoria Barone, Marianthi Karali, Sandro Banfi, Alberto Auricchio

Get full text

Effective AAV‐mediated gene therapy in a mouse model of ethylmalonic encephalopathy by Ivano Di Meo, Alberto Auricchio, Costanza Lamperti, Alberto Burlina, Carlo Viscomi, Massimo Zeviani

Get full text

Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy. by Pasqualina Colella, Andrea Sommella, Elena Marrocco, Umberto Di Vicino, Elena Polishchuk, Marina Garcia Garrido, Mathias W Seeliger, Roman Polishchuk, Alberto Auricchio

Get full text

Disease rescue and increased lifespan in a model of cardiomyopathy and muscular dystrophy by combined AAV treatments. by Carmen Vitiello, Stefania Faraso, Nicolina Cristina Sorrentino, Giovanni Di Salvo, Edoardo Nusco, Gerardo Nigro, Luisa Cutillo, Raffaele Calabrò, Alberto Auricchio, Vincenzo Nigro

Get full text

Liver gene therapy with intein‐mediated F8 trans‐splicing corrects mouse haemophilia A by Federica Esposito, Hristiana Lyubenova, Patrizia Tornabene, Stefano Auricchio, Antonella Iuliano, Edoardo Nusco, Simone Merlin, Cristina Olgasi, Giorgia Manni, Marco Gargaro, Francesca Fallarino, Antonia Follenzi, Alberto Auricchio

Get full text

Effective delivery of large genes to the retina by dual AAV vectors by Ivana Trapani, Pasqualina Colella, Andrea Sommella, Carolina Iodice, Giulia Cesi, Sonia de Simone, Elena Marrocco, Settimio Rossi, Massimo Giunti, Arpad Palfi, Gwyneth J Farrar, Roman Polishchuk, Alberto Auricchio

Get full text

Worsening of cardiomyopathy using deflazacort in an animal model rescued by gene therapy. by Ida Luisa Rotundo, Stefania Faraso, Elvira De Leonibus, Gerardo Nigro, Carmen Vitiello, Alessio Lancioni, Daniele Di Napoli, Sigismondo Castaldo, Vincenzo Russo, Fabio Russo, Giulio Piluso, Alberto Auricchio, Vincenzo Nigro

Get full text

Recombinant vectors based on porcine adeno-associated viral serotypes transduce the murine and pig retina. by Agostina Puppo, Alexander Bello, Anna Manfredi, Giulia Cesi, Elena Marrocco, Michele Della Corte, Settimio Rossi, Massimo Giunti, Maria Laura Bacci, Francesca Simonelli, Enrico Maria Surace, Gary P Kobinger, Alberto Auricchio

Get full text

MicroRNA-restricted transgene expression in the retina. by Marianthi Karali, Anna Manfredi, Agostina Puppo, Elena Marrocco, Annagiusi Gargiulo, Mariacarmela Allocca, Michele Della Corte, Settimio Rossi, Massimo Giunti, Maria Laura Bacci, Francesca Simonelli, Enrico Maria Surace, Sandro Banfi, Alberto Auricchio

Get full text

Liver‐directed gene therapy for ornithine aminotransferase deficiency by Iolanda Boffa, Elena Polishchuk, Lucia De Stefano, Fabio Dell'Aquila, Edoardo Nusco, Elena Marrocco, Matteo Audano, Silvia Pedretti, Marianna Caterino, Ilaria Bellezza, Margherita Ruoppolo, Nico Mitro, Barbara Cellini, Alberto Auricchio, Nicola Brunetti‐Pierri

Get full text