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

Informations projet

UshTher

N° de convention de subvention: 754848

DOI 10.3030/754848

Projet clôturé

Date de signature de la CE 27 Novembre 2017

Date de début 1 Janvier 2018

Date de fin 30 Juin 2023

Financé au titre de

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

Coût total € 5 998 515,75

Contribution de l’UE € 5 998 515,75

Coordonné par

FONDAZIONE TELETHON ETS
Italie

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Les liens vers les livrables et les publications des projets du 7e PC, ainsi que les liens vers certains types de résultats spécifiques tels que les jeux de données et les logiciels, sont récupérés dynamiquement sur OpenAIRE .

Livrables

Documents, rapports (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

Sites Web, dépôts de brevet, vidéos, 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

Publications

Articles approuvés par les pairs (3)

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, Numéro 28/R1, 2019, Page(s) R108-R118, ISSN 0964-6906
Éditeur: 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

Auteurs: 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
Publié dans: Acta Ophthalmologica, 2021, ISSN 1755-375X
Éditeur: Wiley-Blackwell
DOI: 10.1111/aos.14795

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

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