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
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Locations
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Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts, INSERM-DGOS CIC1423
Paris, 75012, France
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Harvard Univ., Massachusetts Eye and Ear Infirmary
Boston, Massachusetts, 02114, United States
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Helsinki University Hospital
Helsinki, 00280, Finland
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INRET Clínica e Centro de Pesquisa
Belo Horizonte, Minas Gerais, 30150-270, Brazil
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Johns Hopkins University, Wilmer Eye Institute
Baltimore, Maryland, 21287, United States
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Moorfields Eye Hospital
London, UK EC1V 2PD, United Kingdom
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University of California San Francisco
San Francisco, California, 94158, United States
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University of Pennsylvania
Philadelphia, Pennsylvania, 19104, United States
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University of Toronto, Hospital for Sick Children
Toronto, Ontario, M5G0A4, Canada
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University of Tuebingen, Centre for Ophthalmology
Tübingen, 72076, Germany
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Vista Vision Eye Clinic
Brescia, 25123, Italy
Conditions
Explore the condition pages connected to this study.