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

Deliverables

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, 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

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