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

Project Information

UshTher

Grant agreement ID: 754848

DOI 10.3030/754848

Project closed

EC signature date 27 November 2017

Start date 1 January 2018

End date 30 June 2023

Funded under

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

Total cost € 5 998 515,75

EU contribution € 5 998 515,75

Coordinated by

FONDAZIONE TELETHON ETS
Italy

Deliverables

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

Publications

Peer reviewed articles (3)

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

Author(s): Ivana Trapani, Alberto Auricchio
Published in: Human Molecular Genetics, Issue 28/R1, 2019, Page(s) R108-R118, ISSN 0964-6906
Publisher: 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

Author(s): 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
Published in: Acta Ophthalmologica, 2021, ISSN 1755-375X
Publisher: Wiley-Blackwell
DOI: 10.1111/aos.14795

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

Author(s): M. Stemerdink, B. García-Bohórquez, R. Schellens, G. Garcia-Garcia, E. Van Wijk & J. M. Millan
Published in: Human Genetics, 2021, ISSN 1434-5161
Publisher: Springer Verlag
DOI: 10.1007/s00439-021-02324-w