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

Deliverables

Official logo of UshTher

Official logo of UshTher

Communication of participation to meetings and conferences RP1

Communication of participation to meetings and conferences

Flyer publication

Flyer publication

Contact database of relevant representatives of stakeholder groups

Contact database of relevant representatives of stakeholder groups

Report Planned communication activity

Report Planned communication activity

Periodic report on project dissemination and exploitation RP2

Periodic report on project dissemination and exploitation RP2

Periodic report on project dissemination and exploitation

Periodic report on project dissemination and exploitation

Detailed dissemination plan

Detailed dissemination plan

Establishment of the Data Safety Monitoring Board (DSMB)

Establishment of the Data Safety Monitoring Board (DSMB)

Connection with patient organization

Connection with patient organization

Communication of peer reviewed high impact publications 1

Communication of peer reviewed high impact publications 1

Publications

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, 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