Clinical trial of gene therapy with dual AAV vectors for retinitis pigmentosa in patients with Usher syndrome type IB

Informazioni relative al progetto

UshTher

ID dell’accordo di sovvenzione: 754848

DOI

10.3030/754848

Progetto chiuso

Data della firma CE 27 Novembre 2017

Data di avvio 1 Gennaio 2018

Data di completamento 30 Giugno 2023

Finanziato da

• SOCIETAL CHALLENGES - Health, demographic change and well-being

Costo totale

€ 5 998 515,75

Contributo UE

€ 5 998 515,75

Coordinato da

FONDAZIONE TELETHON ETS
Italy

CORDIS fornisce collegamenti ai risultati finali pubblici e alle pubblicazioni dei progetti ORIZZONTE.

I link ai risultati e alle pubblicazioni dei progetti del 7° PQ, così come i link ad alcuni tipi di risultati specifici come dataset e software, sono recuperati dinamicamente da .OpenAIRE .

Risultati finali

Documents, reports (19)

Official logo of UshTher
Flyer publication with clinical trial results
Communication of participation to meetings and conferences RP1

Communication of participation to meetings and conferences

Flyer publication
Contact database of relevant representatives of stakeholder groups
Report Planned communication activity
Final report on project dissemination and exploitation
Communication of participation to meetings and conferences final RP

Communication of participation to meetings and conferences

Periodic report on project dissemination and exploitation RP2
UshTher Clinical study - Report on status of posting results
Communication of peer reviewed high impact publications 2
Communication of participation to meetings and conferences RP2

Communication of participation to meetings and conferences

Periodic report on project dissemination and exploitation
Detailed dissemination plan
Communication of peer reviewed high impact publications 3
Establishment of the Data Safety Monitoring Board (DSMB)
Dissemination of the trial results through the project website
Connection with patient organization
Communication of peer reviewed high impact publications 1

Websites, patent fillings, videos etc. (6)

Social Network strategy plan
Open Day event 1
Website and intranet portal
Open Day event 3
Report of completed communication activities
Open Day event 2

Pubblicazioni

Peer reviewed articles (5)

Multicentric Longitudinal Prospective Study in a European Cohort of <i>MYO7A</i> Patients: Disease Course and Implications for Gene Therapy

Autori: Francesco Testa, Ester Carreño, L. Ingeborgh van den Born, Paolo Melillo, Irene Perea-Romero, Valentina Di Iorio, Giulia Risca, Clemente Maria Iodice, Ronald J. E. Pennings, Marianthi Karali, Sandro Banfi, Alberto Auricchio, Stefania Galimberti, Carmen Ayuso, Francesca Simonelli
Pubblicato in: Investigative Ophthalmology &amp; Visual Science, Numero 65, 2024, Pagina/e 25, ISSN 1552-5783
Editore: Association for Research in Vision and Ophthalmology (ARVO)
DOI: 10.1167/iovs.65.6.25

Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B

Autori: Rita Ferla, Fabio Dell’Aquila, Monica Doria, Maria Ferraiuolo, Alessia Noto, Fabiana Grazioli, Virginia Ammendola, Francesco Testa, Paolo Melillo, Carolina Iodice, Giulia Risca, Novella Tedesco, Pierre Romain le Brun, Enrico Maria Surace, Francesca Simonelli, Stefania Galimberti, Maria Grazia Valsecchi, Jean-Brice Marteau, Philippe Veron, Stefano Colloca, Alberto Auricchio
Pubblicato in: Molecular Therapy - Methods &amp; Clinical Development, Numero 28, 2025, Pagina/e 396-411, ISSN 2329-0501
Editore: Elsevier BV
DOI: 10.1016/j.omtm.2023.02.002

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

Autori: Ivana Trapani, Alberto Auricchio
Pubblicato in: Human Molecular Genetics, Numero 28/R1, 2019, Pagina/e R108-R118, ISSN 0964-6906
Editore: Oxford University Press
DOI: 10.1093/hmg/ddz130

Genotype–phenotype correlation in patients with Usher syndrome and pathogenic variants in MYO7A : implications for future clinical trials

Autori: Lilián Galbis‐Martínez, Fiona Blanco‐Kelly, Gema García‐García, Almudena Ávila‐Fernández, Teresa Jaijo, Carla Fuster‐García, Irene Perea‐Romero, Olga Zurita‐Muñoz, Belén Jimenez‐Rolando, Ester Carreño, Blanca García‐Sandoval, José M. Millán, Carmen Ayuso
Pubblicato in: Acta Ophthalmologica, 2021, ISSN 1755-375X
Editore: Wiley-Blackwell
DOI: 10.1111/aos.14795

Genetics, pathogenesis and therapeutic developments for Usher syndrome type 2.

Autori: M. Stemerdink, B. García-Bohórquez, R. Schellens, G. Garcia-Garcia, E. Van Wijk & J. M. Millan
Pubblicato in: Human Genetics, 2021, ISSN 1434-5161
Editore: Springer Verlag
DOI: 10.1007/s00439-021-02324-w