Gene therapy of inherited retinal diseases

Project Information

EYEGET

Grant agreement ID: 694323

DOI 10.3030/694323

Project closed

EC signature date 31 August 2016

Start date 1 January 2017

End date 31 December 2022

Funded under

• EXCELLENT SCIENCE - European Research Council (ERC)

Total cost € 2 499 564,00

EU contribution € 2 499 564,00

Coordinated by

FONDAZIONE TELETHON ETS
Italy

Publications

Peer reviewed articles (7)

Intein-mediated protein trans-splicing expands adeno-associated virus transfer capacity in the retina

Author(s): Tornabene, Patrizia; Trapani, Ivana; Minopoli, Renato; Centrulo, Miriam; Lupo, Mariangela; de Simone, Sonia; Tiberi, Paola; Dell'Aquila, Fabio; Marrocco, Elena; Iodice, Carolina; Iuliano, Antonella; Gesualdo, Carlo; Rossi, Settimio; Giaquinto, Laura; Albert, Silvia; Hoyng, Carel B.; Polishchuk, Elena; Cremers, Frans P.M.; Surace, Enrico M.; Simonelli, Francesca; De Matteis, Maria A.; P
Published in: Science Translational Medicine, Issue 11 (492), 2019, ISSN 1946-6234
Publisher: American Association for the Advancement of Science
DOI: 10.1126/scitranslmed.aav4523

Therapeutic homology-independent targeted integration in retina and liver

Author(s): Patrizia Tornabene, Rita Ferla, Manel Llado-Santaeularia, Miriam Centrulo, Margherita Dell’Anno, Federica Esposito, Elena Marrocco, Emanuela Pone, Renato Minopoli, Carolina Iodice, Edoardo Nusco, Settimio Rossi, Hristiana Lyubenova, Anna Manfredi, Lucio Di Filippo, Antonella Iuliano, Annalaura Torella, Giulio Piluso, Francesco Musacchia, Enrico Maria Surace, Davide Cacchiarelli, Vincenzo Nigro &
Published in: Nature Communications, Issue 13, 2023, Page(s) 1963, ISSN 2041-1723
Publisher: Nature Publishing Group
DOI: 10.1038/s41467-022-29550-8

Liver gene therapy with intein-mediated F8 trans-splicing corrects mouse haemophilia A

Author(s): 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
Published in: EMBO Mol Med, Issue 1, 2022, ISSN 1757-4684
Publisher: EMBO Press
DOI: 10.15252/emmm.202115199

Seeing the Light after 25 Years of Retinal Gene Therapy

Author(s): Ivana Trapani, Alberto Auricchio
Published in: Trends in Molecular Medicine, Issue 24/8, 2018, Page(s) 669-681, ISSN 1471-4914
Publisher: Elsevier BV
DOI: 10.1016/j.molmed.2018.06.006

High-Throughput Screening Identifies Kinase Inhibitors That Increase Dual Adeno-Associated Viral Vector Transduction In Vitro and in Mouse Retina

Author(s): Andrea Maddalena, Fabio Dell'Aquila, Pia Giovannelli, Paola Tiberi, Luca Giorgio Wanderlingh, Sandro Montefusco, Patrizia Tornabene, Carolina Iodice, Feliciano Visconte, Annamaria Carissimo, Diego Luis Medina, Gabriella Castoria, Alberto Auricchio
Published in: Human Gene Therapy, Issue 29/8, 2018, Page(s) 886-901, ISSN 1043-0342
Publisher: Mary Ann Liebert Inc.
DOI: 10.1089/hum.2017.220

Triple Vectors Expand AAV Transfer Capacity in the Retina

Author(s): Andrea Maddalena, Patrizia Tornabene, Paola Tiberi, Renato Minopoli, Anna Manfredi, Margherita Mutarelli, Settimio Rossi, Francesca Simonelli, Jurgen K. Naggert, Davide Cacchiarelli, Alberto Auricchio
Published in: Molecular Therapy, Issue 26/2, 2018, Page(s) 524-541, ISSN 1525-0016
Publisher: Nature Publishing Group
DOI: 10.1016/j.ymthe.2017.11.019

Has retinal gene therapy come of age? From bench to bedside and back to bench

Author(s): Ivana Trapani, Alberto Auricchio
Published in: Human Molecular Genetics, 2019, ISSN 0964-6906
Publisher: Oxford University Press
DOI: 10.1093/hmg/ddz130