Gene therapy of inherited retinal diseases

Publicaciones

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

Autores: 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
Publicado en: Science Translational Medicine, Edición 11 (492), 2019, ISSN 1946-6234
Editor: American Association for the Advancement of Science
DOI: 10.1126/scitranslmed.aav4523

Therapeutic homology-independent targeted integration in retina and liver

Autores: 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 &
Publicado en: Nature Communications, Edición 13, 2023, Página(s) 1963, ISSN 2041-1723
Editor: Nature Publishing Group
DOI: 10.1038/s41467-022-29550-8

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

Autores: 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
Publicado en: EMBO Mol Med, Edición 1, 2022, ISSN 1757-4684
Editor: EMBO Press
DOI: 10.15252/emmm.202115199

Seeing the Light after 25 Years of Retinal Gene Therapy

Autores: Ivana Trapani, Alberto Auricchio
Publicado en: Trends in Molecular Medicine, Edición 24/8, 2018, Página(s) 669-681, ISSN 1471-4914
Editor: 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

Autores: 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
Publicado en: Human Gene Therapy, Edición 29/8, 2018, Página(s) 886-901, ISSN 1043-0342
Editor: Mary Ann Liebert Inc.
DOI: 10.1089/hum.2017.220

Triple Vectors Expand AAV Transfer Capacity in the Retina

Autores: Andrea Maddalena, Patrizia Tornabene, Paola Tiberi, Renato Minopoli, Anna Manfredi, Margherita Mutarelli, Settimio Rossi, Francesca Simonelli, Jurgen K. Naggert, Davide Cacchiarelli, Alberto Auricchio
Publicado en: Molecular Therapy, Edición 26/2, 2018, Página(s) 524-541, ISSN 1525-0016
Editor: 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

Autores: Ivana Trapani, Alberto Auricchio
Publicado en: Human Molecular Genetics, 2019, ISSN 0964-6906
Editor: Oxford University Press
DOI: 10.1093/hmg/ddz130

Buscando datos de OpenAIRE...

Publicaciones

Compartir esta página

Descargar