Gene therapy of inherited retinal diseases

Publikacje

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

Autorzy: 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
Opublikowane w: Science Translational Medicine, Numer 11 (492), 2019, ISSN 1946-6234
Wydawca: American Association for the Advancement of Science
DOI: 10.1126/scitranslmed.aav4523

Therapeutic homology-independent targeted integration in retina and liver

Autorzy: 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 &
Opublikowane w: Nature Communications, Numer 13, 2023, Strona(/y) 1963, ISSN 2041-1723
Wydawca: Nature Publishing Group
DOI: 10.1038/s41467-022-29550-8

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

Autorzy: 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
Opublikowane w: EMBO Mol Med, Numer 1, 2022, ISSN 1757-4684
Wydawca: EMBO Press
DOI: 10.15252/emmm.202115199

Seeing the Light after 25 Years of Retinal Gene Therapy

Autorzy: Ivana Trapani, Alberto Auricchio
Opublikowane w: Trends in Molecular Medicine, Numer 24/8, 2018, Strona(/y) 669-681, ISSN 1471-4914
Wydawca: 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

Autorzy: 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
Opublikowane w: Human Gene Therapy, Numer 29/8, 2018, Strona(/y) 886-901, ISSN 1043-0342
Wydawca: Mary Ann Liebert Inc.
DOI: 10.1089/hum.2017.220

Triple Vectors Expand AAV Transfer Capacity in the Retina

Autorzy: Andrea Maddalena, Patrizia Tornabene, Paola Tiberi, Renato Minopoli, Anna Manfredi, Margherita Mutarelli, Settimio Rossi, Francesca Simonelli, Jurgen K. Naggert, Davide Cacchiarelli, Alberto Auricchio
Opublikowane w: Molecular Therapy, Numer 26/2, 2018, Strona(/y) 524-541, ISSN 1525-0016
Wydawca: 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

Autorzy: Ivana Trapani, Alberto Auricchio
Opublikowane w: Human Molecular Genetics, 2019, ISSN 0964-6906
Wydawca: Oxford University Press
DOI: 10.1093/hmg/ddz130

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