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Mostrati risultati da 21 a 40 di 307
Titolo Tipologia Data di pubblicazione Autore(i) File
Current Status on Clinical Development of Adeno-Associated Virus-Mediated Liver-Directed Gene Therapy for Inborn Errors of Metabolism 1.1 Articolo in rivista 2019 Ginocchio, V. M.; Ferla, R.; Auricchio, A.; Brunetti-Pierri, N.
ASSOCIATION BETWEEN GENOTYPE AND DISEASE PROGRESSION IN ITALIAN STARGARDT PATIENTS: A Retrospective Natural History Study 1.1 Articolo in rivista 2019 Di Iorio, Valentina; Orrico, Ada; Esposito, Gabriella; Melillo, Paolo; Rossi, Settimio; Sbordone, Sandro; Auricchio, Alberto; Testa, Francesco; Simonelli, Francesca
Inhibition of Lipofuscin Aggregation by Molecular Tweezers. 6.1 Brevetto 2018 Fraldi, A; Auricchio, A; Monaco, A
Seeing the Light after 25 Years of Retinal Gene Therapy 1.1 Articolo in rivista 2018 Trapani, Ivana; Auricchio, Alberto
High-Throughput Screening Identifies Kinase Inhibitors That Increase Dual Adeno-Associated Viral Vector Transduction in Vitro and in Mouse Retina 1.1 Articolo in rivista 2018 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
AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease 1.1 Articolo in rivista 2017 Gatto, Francesca; Rossi, Barbara; Tarallo, Antonietta; Polishchuk, Elena; Polishchuk, Roman; Carrella, Alessandra; Nusco, Edoardo; Alvino, Filomena Grazia; Iacobellis, Francesca; De Leonibus, Elvira; Auricchio, Alberto; Diez roux, Graciana; Ballabio, Andrea; Parenti, Giancarlo
The future looks brighter after 25 years of retinal gene therapy 1.1 Articolo in rivista 2017 Ali, Robin R; Auricchio, Alberto; Smith, Alexander J.
CLINICAL PRESENTATION AND DISEASE COURSE OF USHER SYNDROME BECAUSE OF MUTATIONS IN MYO7A OR USH2A 1.1 Articolo in rivista 2017 Testa, Francesco; Melillo, Paolo; Bonnet, Crystel; Marcelli, Vincenzo; de Benedictis, Antonella; Colucci, Raffaella; Gallo, Beatrice; Kurtenbach, Anne; Rossi, Settimio; Marciano, Elio; Auricchio, Alberto; Petit, Christine; Zrenner, Eberhart; Simonelli, Francesca; Marciano, Elio
Non-clinical Safety and Efficacy of an AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VI 1.1 Articolo in rivista 2017 Ferla, Rita; Alliegro, Marialuisa; Marteau, Jean brice; Dell'Anno, Margherita; Nusco, Edoardo; Pouillot, Severine; Galimberti, Stefania; Valsecchi, Maria Grazia; Zuliani, Vincent; Auricchio, Alberto
Allele-Specific Silencing of Mutant mRNA Rescues Ultrastructural and Arrhythmic Phenotype in Mice Carriers of the R4496C Mutation in the Ryanodine Receptor Gene (RYR2) 1.1 Articolo in rivista 2017 Bongianino, Rossana; Denegri, Marco; Mazzanti, Andrea; Lodola, Francesco; Vollero, Alessandra; Boncompagni, Simona; Fasciano, Silvia; Rizzo, Giulia; Mangione, Damiano; Barbaro, Serena; Di Fonso, Alessia; Napolitano, Carlo; Auricchio, Alberto; Protasi, Feliciano; Priori, Silvia G.
A rare genetic variant of BPIFB4 predisposes to high blood pressure via impairment of nitric oxide signaling 1.1 Articolo in rivista 2017 Vecchione, Carmine; Villa, Francesco; Carrizzo, Albino; Spinelli, Chiara Carmela; Damato, Antonio; Ambrosio, Mariateresa; Ferrario, Anna; Madonna, Michele; Uccellatore, Annachiara; Lupini, Silvia; Maciag, Anna; Ryskalin, Larisa; Milanesi, Luciano; Frati, Giacomo; Sciarretta, Sebastiano; Bellazzi, Riccardo; Genovese, Stefano; Ceriello, Antonio; Auricchio, Alberto; Malovini, Alberto; Puca, Annibale Alessandro
Triple Vectors Expand AAV Transfer Capacity in the Retina 1.1 Articolo in rivista 2017 Maddalena, Andrea; Tornabene, Patrizia; Tiberi, Paola; Minopoli, Renato; Manfredi, Anna; Mutarelli, Margherita; Rossi, Settimio; Simonelli, Francesca; Naggert, Jurgen K.; Cacchiarelli, Davide; Auricchio, Alberto
Combined Ttherapy fot lysosomal storage disease 6.1 Brevetto 2016 Auricchio, A
Treatment of bone growth disorders 6.1 Brevetto 2016 Settembre, Carmine; Cinque, Laura; Bartolomeo, Rosa; Auricchio, Alberto; Trapani, Ivana; Toriello, Elisabetta
En Face Spectral-Domain Optical Coherence Tomography for the Monitoring of Lesion Area Progression in Stargardt Disease 1.1 Articolo in rivista 2016 Melillo, Paolo; Testa, Francesco; Rossi, Settimio; Di Iorio, Valentina; Orrico, Ada; Auricchio, Alberto; Simonelli, Francesca
Evaluation of ocular gene therapy in an italian patient affected by congenital leber amaurosis type 2 treated in both eyes 2.1 Contributo in volume (Capitolo o Saggio) 2016 Testa, Francesco; Maguire, Albert M; Rossi, Settimio; Marshall, Kathleen; Auricchio, Alberto; Melillo, Paolo; Bennett, Jean; Simonelli, Francesca
An innovative strategy for the molecular diagnosis of Usher syndrome identifies causal biallelic mutations in 93% of European patients 1.1 Articolo in rivista 2016 Bonnet, Crystel; Riahi, Zied; Chantot Bastaraud, Sandra; Smagghe, Luce; Letexier, Mélanie; Marcaillou, Charles; Lefèvre, Gaëlle M; Hardelin, Jean Pierre; El Amraoui, Aziz; Singh Estivalet, Amrit; Mohand Saïd, Saddek; Kohl, Susanne; Kurtenbach, Anne; Sliesoraityte, Ieva; Zobor, Ditta; Gherbi, Souad; Testa, Francesco; Simonelli, Francesca; Banfi, Sandro; Fakin, Ana; Glavač, Damjan; Jarc Vidmar, Martina; Zupan, Andrej; Battelino, Saba; Martorell Sampol, Loreto; Claveria, Maria Antonia; Catala Mora, Jaume; Dad, Shzeena; Møller, Lisbeth B; Rodriguez Jorge, Jesus; Hawlina, Marko; Auricchio, Alberto; Sahel, José Alain; Marlin, Sandrine; Zrenner, Eberhart; Audo, Isabelle; Petit, Christine
Low-dose Gene Therapy Reduces the Frequency of Enzyme Replacement Therapy in a Mouse Model of Lysosomal Storage Disease 1.1 Articolo in rivista 2016 Alliegro, Marialuisa; Ferla, Rita; Nusco, Edoardo; DE LEONIBUS, Chiara; Settembre, Carmine; Auricchio, Alberto
A comprehensive map of CNS transduction by eight recombinant adeno-associated virus serotypes upon cerebrospinal fluid administration in pigs 1.1 Articolo in rivista 2016 Sorrentino, Nicolina Cristina; Maffia, Veronica; Strollo, Sandra; Cacace, Vincenzo; Romagnoli, Noemi; Manfredi, Anna; Ventrella, Domenico; Dondi, Francesco; Barone, Francesca; Giunti, Massimo; Graham, Anne Renee; Huang, Yan; Kalled, Susan L; Auricchio, Alberto; Bacci, Maria Laura; Surace, Enrico Maria; Fraldi, Alessandro
Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial 1.1 Articolo in rivista 2016 Bennett, Jean; Wellman, Jennifer; Marshall, Kathleen A; Mccague, Sarah; Ashtari, Manzar; DiStefano Pappas, Julie; Elci, Okan U; Chung, Daniel C; Sun, Junwei; Wright, J. Fraser; Cross, Dominique R; Aravand, Puya; Cyckowski, Laura L; Bennicelli, Jeannette L; Mingozzi, Federico; Auricchio, Alberto; Pierce, Eric A; Ruggiero, Jason; Leroy, Bart P; Simonelli, Francesca; High, Katherine A; Maguire, Albert M.
Mostrati risultati da 21 a 40 di 307
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