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

Author:

Testa Francesco1,Carreño Ester2,van den Born L. Ingeborgh3,Melillo Paolo1,Perea-Romero Irene45,Di Iorio Valentina1,Risca Giulia6,Iodice Clemente Maria1,Pennings Ronald J. E.7,Karali Marianthi18,Banfi Sandro89,Auricchio Alberto91011,Galimberti Stefania6,Ayuso Carmen45,Simonelli Francesca1

Affiliation:

1. Eye Clinic, Multidisciplinary Department of Medical, Surgical and Dental Sciences, University of Campania “Luigi Vanvitelli,” Naples, Italy

2. Department of Ophthalmology, Health Research Institute-Fundación Jiménez Díaz University Hospital, Universidad Autónoma de Madrid (IIS-FJD, UAM), Madrid, Spain

3. The Rotterdam Eye Hospital, Rotterdam, the Netherlands

4. Department of Genetics, Health Research Institute-Fundación Jiménez Díaz University Hospital, Universidad Autónoma de Madrid (IIS-FJD, UAM), Madrid, Spain

5. Center for Biomedical Network Research on Rare Diseases (CIBERER), Instituto de Salud Carlos III, Madrid, Spain

6. Bicocca Bioinformatics, Biostatistics and Bioimaging Centre, Department of Medicine and Surgery, University of Milano-Bicocca, Milan, Italy

7. Department of Otorhinolaryngology, Hearing & Genes, Radboud University Medical Center, Nijmegen, the Netherlands

8. Medical Genetics, Department of Precision Medicine, University of Campania “Luigi Vanvitelli,” Naples, Italy

9. Telethon Institute of Genetics and Medicine (TIGEM), Pozzuoli, Italy

10. Medical Genetics, Department of Advanced Biomedical Sciences, Federico II University, Naples, Italy

11. AAVantgarde Bio, Milan, Italy

Publisher

Association for Research in Vision and Ophthalmology (ARVO)

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