Scientists map rare eye disease to pave way for gene therapy

NCT ID NCT05312736

Summary

This study aims to understand how a rare genetic eye disease called gyrate atrophy progresses over four years in people who are following standard dietary treatment. Researchers will track changes in vision, eye structure, and blood levels in 46 participants to gather essential information. The data will help design future clinical trials for a potential one-time gene therapy treatment.

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Contacts and locations

Locations

  • Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, INSERM-DGOS CIC1423

    Paris, 75012, France

  • Harvard Univ., Massachusetts Eye and Ear Infirmary

    Boston, Massachusetts, 02114, United States

  • Helsinki University Hospital

    Helsinki, 00280, Finland

  • INRET Clínica e Centro de Pesquisa

    Belo Horizonte, Minas Gerais, 30150-270, Brazil

  • Johns Hopkins University, Wilmer Eye Institute

    Baltimore, Maryland, 21287, United States

  • Moorfields Eye Hospital

    London, UK EC1V 2PD, United Kingdom

  • University of California San Francisco

    San Francisco, California, 94158, United States

  • University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • University of Toronto, Hospital for Sick Children

    Toronto, Ontario, M5G0A4, Canada

  • University of Tuebingen, Centre for Ophthalmology

    Tübingen, 72076, Germany

  • Vista Vision Eye Clinic

    Brescia, 25123, Italy

Conditions

Explore the condition pages connected to this study.