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

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

Ezrin defines TSC complex activation at endosomal compartments through EGFR–AKT signaling by Giuliana Giamundo, Daniela Intartaglia, Eugenio Del Prete, Elena Polishchuk, Fabrizio Andreone, Marzia Ognibene, Sara Buonocore, Bruno Hay Mele, Francesco Giuseppe Salierno, Jlenia Monfregola, Dario Antonini, Paolo Grumati, Alessandra Eva, Rossella De Cegli, Ivan Conte

Get full text

Correction of oxidative stress enhances enzyme replacement therapy in Pompe disease by Antonietta Tarallo, Carla Damiano, Sandra Strollo, Nadia Minopoli, Alessia Indrieri, Elena Polishchuk, Francesca Zappa, Edoardo Nusco, Simona Fecarotta, Caterina Porto, Marcella Coletta, Roberta Iacono, Marco Moracci, Roman Polishchuk, Diego Luis Medina, Paola Imbimbo, Daria Maria Monti, Maria Antonietta De Matteis, Giancarlo Parenti

Get full text

Beclin‐1‐mediated activation of autophagy improves proximal and distal urea cycle disorders by Leandro R Soria, Sonam Gurung, Giulia De Sabbata, Dany P Perocheau, Angela De Angelis, Gemma Bruno, Elena Polishchuk, Debora Paris, Paola Cuomo, Andrea Motta, Michael Orford, Youssef Khalil, Simon Eaton, Philippa B Mills, Simon N Waddington, Carmine Settembre, Andrés F Muro, Julien Baruteau, Nicola Brunetti‐Pierri

Get full text

Impact of liver fibrosis on AAV-mediated gene transfer to mouse hepatocytes by Rosa Ferriero, Gemma Bruno, Agnese Padula, Simone Pisano, Iolanda Boffa, Marco Gargaro, Teresa Imperatore, Maria Battipaglia, Silvia Vivenzio, Claudia Perna, Edoardo Nusco, Luigi Ferrante, Adrian Westhaus, Maddison Knight, Giorgia Manni, Severo Campione, Evaristo Di Napoli, Elena Polishchuk, Roman Polishchuk, Orlando Paciello, Nicola Brunetti-Pierri, Leszek Lisowski, Francesca Fallarino, Pasquale Piccolo

Get full text

TPC2 rescues lysosomal storage in mucolipidosis type IV, Niemann–Pick type C1, and Batten disease by Anna Scotto Rosato, Einar K Krogsaeter, Dawid Jaślan, Carla Abrahamian, Sandro Montefusco, Chiara Soldati, Barbara Spix, Maria Teresa Pizzo, Giuseppina Grieco, Julia Böck, Amanda Wyatt, Daniela Wünkhaus, Marcel Passon, Marc Stieglitz, Marco Keller, Guido Hermey, Sandra Markmann, Doris Gruber‐Schoffnegger, Susan Cotman, Ludger Johannes, Dennis Crusius, Ulrich Boehm, Christian Wahl‐Schott, Martin Biel, Franz Bracher, Elvira De Leonibus, Elena Polishchuk, Diego L Medina, Dominik Paquet, Christian Grimm

Get full text