Gene therapy of inherited retinal diseases

Publications

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

Auteurs: 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
Publié dans: Science Translational Medicine, Numéro 11 (492), 2019, ISSN 1946-6234
Éditeur: American Association for the Advancement of Science
DOI: 10.1126/scitranslmed.aav4523

Therapeutic homology-independent targeted integration in retina and liver

Auteurs: 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 &
Publié dans: Nature Communications, Numéro 13, 2023, Page(s) 1963, ISSN 2041-1723
Éditeur: Nature Publishing Group
DOI: 10.1038/s41467-022-29550-8

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

Auteurs: 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
Publié dans: EMBO Mol Med, Numéro 1, 2022, ISSN 1757-4684
Éditeur: EMBO Press
DOI: 10.15252/emmm.202115199

Seeing the Light after 25 Years of Retinal Gene Therapy

Auteurs: Ivana Trapani, Alberto Auricchio
Publié dans: Trends in Molecular Medicine, Numéro 24/8, 2018, Page(s) 669-681, ISSN 1471-4914
Éditeur: 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

Auteurs: 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
Publié dans: Human Gene Therapy, Numéro 29/8, 2018, Page(s) 886-901, ISSN 1043-0342
Éditeur: Mary Ann Liebert Inc.
DOI: 10.1089/hum.2017.220

Triple Vectors Expand AAV Transfer Capacity in the Retina

Auteurs: Andrea Maddalena, Patrizia Tornabene, Paola Tiberi, Renato Minopoli, Anna Manfredi, Margherita Mutarelli, Settimio Rossi, Francesca Simonelli, Jurgen K. Naggert, Davide Cacchiarelli, Alberto Auricchio
Publié dans: Molecular Therapy, Numéro 26/2, 2018, Page(s) 524-541, ISSN 1525-0016
Éditeur: 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

Auteurs: Ivana Trapani, Alberto Auricchio
Publié dans: Human Molecular Genetics, 2019, ISSN 0964-6906
Éditeur: Oxford University Press
DOI: 10.1093/hmg/ddz130

Droits de propriété intellectuelle

GENOME EDITING METHODS AND CONSTRUCTS

Numéro de demande/publication: 20 19078019
Date: 2019-10-15
Demandeur(s): FONDAZIONE TELETHON ETS

INTEIN PROTEINS AND USES THEREOF

Numéro de demande/publication: 20 19078020
Date: 2019-10-15
Demandeur(s): FONDAZIONE TELETHON ETS

VECTOR SYSTEM

Numéro de demande/publication: 20 22062989
Date: 2022-05-12
Demandeur(s): FONDAZIONE TELETHON ETS

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Droits de propriété intellectuelle

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