1. AAV-mediated transcription factor EB (TFEB) gene delivery ameliorates muscle pathology and function in the murine model of Pompe Disease. Issue 1 (December 2017) Authors: Gatto, Francesca; Rossi, Barbara; Tarallo, Antonietta; Polishchuk, Elena; Polishchuk, Roman; Carrella, Alessandra; Nusco, Edoardo; Alvino, Filomena Grazia; Iacobellis, Francesca; Leonibus, Elvira; Auricchio, Alberto; Diez-Roux, Graciana; Ballabio, Andrea; Parenti, Giancarlo Journal: Scientific reports Issue: Volume 7:Issue 1(2017) Page Start: 1 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
2. Allele-Specific Silencing of Mutant mRNA Rescues Ultrastructural and Arrhythmic Phenotype in Mice Carriers of the R4496C Mutation in the Ryanodine Receptor Gene (RYR2). Issue 5 (18th August 2017) Authors: 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. Journal: Circulation research Issue: Volume 121:Issue 5(2017) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
3. ASSOCIATION BETWEEN GENOTYPE AND DISEASE PROGRESSION IN ITALIAN STARGARDT PATIENTS: A Retrospective Natural History Study. Issue 7 (July 2019) Authors: Di Iorio, Valentina; Orrico, Ada; Esposito, Gabriella; Melillo, Paolo; Rossi, Settimio; Sbordone, Sandro; Auricchio, Alberto; Testa, Francesco; Simonelli, Francesca Journal: Retina Issue: Volume 39:Issue 7(2019) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
4. CLINICAL PRESENTATION AND DISEASE COURSE OF USHER SYNDROME BECAUSE OF MUTATIONS IN MYO7A OR USH2A. Issue 8 (August 2017) Authors: 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 Journal: Retina Issue: Volume 37:Issue 8(2017:Aug.) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
5. Effective delivery of large genes to the retina by dual AAV vectors. Issue 2 (16th December 2013) Authors: Trapani, Ivana; Colella, Pasqualina; Sommella, Andrea; Iodice, Carolina; Cesi, Giulia; de, Sonia; Marrocco, Elena; Rossi, Settimio; Giunti, Massimo; Palfi, Arpad; Farrar, Gwyneth J; Polishchuk, Roman; Auricchio, Alberto Journal: EMBO molecular medicine Issue: Volume 6:Issue 2(2014:Feb.) Page Start: 194 Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
6. Genetic Analysis Reveals a Longevity-Associated Protein Modulating Endothelial Function and Angiogenesis. Issue 4 (31st July 2015) Authors: Villa, Francesco; Carrizzo, Albino; Spinelli, Chiara C.; Ferrario, Anna; Malovini, Alberto; Maciąg, Anna; Damato, Antonio; Auricchio, Alberto; Spinetti, Gaia; Sangalli, Elena; Dang, Zexu; Madonna, Michele; Ambrosio, Mariateresa; Sitia, Leopoldo; Bigini, Paolo; Calì, Gaetano; Schreiber, Stefan; Pe... Journal: Circulation research Issue: Volume 117:Issue 4(2015) Page Start: Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
7. Light‐responsive microRNA miR‐211 targets Ezrin to modulate lysosomal biogenesis and retinal cell clearance. (10th March 2020) Authors: Naso, Federica; Intartaglia, Daniela; Falanga, Danila; Soldati, Chiara; Polishchuk, Elena; Giamundo, Giuliana; Tiberi, Paola; Marrocco, Elena; Scudieri, Paolo; Di Malta, Chiara; Trapani, Ivana; Nusco, Edoardo; Salierno, Francesco Giuseppe; Surace, Enrico Maria; Galietta, Luis JV; Banfi, Sandro; A... Journal: EMBO journal Issue: Volume 39:Number 8(2020) Page Start: n/a Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
8. Liver gene therapy with intein‐mediated F8 trans‐splicing corrects mouse haemophilia A. Issue 6 (2nd May 2022) Authors: Esposito, Federica; Lyubenova, Hristiana; Tornabene, Patrizia; Auricchio, Stefano; Iuliano, Antonella; Nusco, Edoardo; Merlin, Simone; Olgasi, Cristina; Manni, Giorgia; Gargaro, Marco; Fallarino, Francesca; Follenzi, Antonia; Auricchio, Alberto Journal: EMBO molecular medicine Issue: Volume 14:Issue 6(2022) Page Start: n/a Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
9. Liver‐directed gene therapy for ornithine aminotransferase deficiency. Issue 4 (17th January 2023) Authors: 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 Journal: EMBO molecular medicine Issue: Volume 15:Issue 4(2023) Page Start: n/a Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗
10. MiR‐181a/b downregulation: a mutation‐independent therapeutic approach for inherited retinal diseases. Issue 11 (4th October 2022) Authors: Carrella, Sabrina; Di Guida, Martina; Brillante, Simona; Piccolo, Davide; Ciampi, Ludovica; Guadagnino, Irene; Garcia Piqueras, Jorge; Pizzo, Mariateresa; Marrocco, Elena; Molinari, Marta; Petrogiannakis, Georgios; Barbato, Sara; Ezhova, Yulia; Auricchio, Alberto; Franco, Brunella; De Leonibus, E... Journal: EMBO molecular medicine Issue: Volume 14:Issue 11(2022) Page Start: n/a Record Type: Journal Article View Content: Available online (eLD content is only available in our Reading Rooms) ↗