Gene Therapy of Inherited Retinopathies: A Long and Successful Road from Viral Vectors to Patients

Por Colella, Pasqualina, Auricchio, Alberto
Publicado em 2012

Non-erythropoietic erythropoietin derivatives protect from light-induced and genetic photoreceptor degeneration

Por Colella, Pasqualina, Iodice, Carolina, Di Vicino, Umberto, Annunziata, Ida, Surace, Enrico M., Auricchio, Alberto
Publicado em 2011

Transposon-mediated Generation of Cellular and Mouse Models of Splicing Mutations to Assess the Efficacy of snRNA-based Therapeutics

Por Barbon, Elena, Ferrarese, Mattia, van Wittenberghe, Laetitia, Sanatine, Peggy, Ronzitti, Giuseppe, Collaud, Fanny, Colella, Pasqualina, Pinotti, Mirko, Mingozzi, Federico
Publicado no Mol Ther Nucleic Acids (2016)

Myosin7a Deficiency Results in Reduced Retinal Activity Which Is Improved by Gene Therapy

Por Colella, Pasqualina, Sommella, Andrea, Marrocco, Elena, Di Vicino, Umberto, Polishchuk, Elena, Garrido, Marina Garcia, Seeliger, Mathias W., Polishchuk, Roman, Auricchio, Alberto
Publicado em 2013

Effective delivery of large genes to the retina by dual AAV vectors

Por Trapani, Ivana, Colella, Pasqualina, Sommella, Andrea, Iodice, Carolina, Cesi, Giulia, de Simone, Sonia, Marrocco, Elena, Rossi, Settimio, Giunti, Massimo, Palfi, Arpad, Farrar, Gwyneth J, Polishchuk, Roman, Auricchio, Alberto
Publicado em 2014

TFEB Links Autophagy to Lysosomal Biogenesis

Por Settembre, Carmine, Di Malta, Chiara, Polito, Vinicia Assunta, Arencibia, Moises Garcia, Vetrini, Francesco, Erdin, Serkan, Erdin, Serpil Uckac, Huynh, Tuong, Medina, Diego, Colella, Pasqualina, Sardiello, Marco, Rubinsztein, David C., Ballabio, Andrea
Publicado em 2011

Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease

Por Trapani, Ivana, Toriello, Elisabetta, de Simone, Sonia, Colella, Pasqualina, Iodice, Carolina, Polishchuk, Elena V., Sommella, Andrea, Colecchi, Linda, Rossi, Settimio, Simonelli, Francesca, Giunti, Massimo, Bacci, Maria L., Polishchuk, Roman S., Auricchio, Alberto
Publicado no Hum Mol Genet (2015)

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice

Por Allocca, Mariacarmela, Doria, Monica, Petrillo, Marco, Colella, Pasqualina, Garcia-Hoyos, Maria, Gibbs, Daniel, Kim, So Ra, Maguire, Albert, Rex, Tonia S., Di Vicino, Umberto, Cutillo, Luisa, Sparrow, Janet R., Williams, David S., Bennett, Jean, Auricchio, Alberto
Publicado em 2008

Long-term exposure to Myozyme results in a decrease of anti-drug antibodies in late-onset Pompe disease patients

Por Masat, Elisa, Laforêt, Pascal, De Antonio, Marie, Corre, Guillaume, Perniconi, Barbara, Taouagh, Nadjib, Mariampillai, Kuberaka, Amelin, Damien, Mauhin, Wladimir, Hogrel, Jean-Yves, Caillaud, Catherine, Ronzitti, Giuseppe, Puzzo, Francesco, Kuranda, Klaudia, Colella, Pasqualina, Mallone, Roberto, Benveniste, Olivier, Mingozzi, Federico
Publicado no Sci Rep (2016)