Gene therapy shows promise for rare eye disease
NCT ID NCT02652767
First seen Mar 02, 2026 · Last updated May 01, 2026 · Updated 11 times
Summary
This study tested a one-time gene therapy injection (GS010) in 39 people with Leber Hereditary Optic Neuropathy (LHON), a genetic condition that causes rapid vision loss. Participants had vision loss for 6 months or less. The goal was to see if the treatment could improve eyesight compared to a sham injection. The trial is complete, and results focus on changes in visual acuity over 48 weeks.
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Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Contacts and locations
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Locations
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Centre National Hospitalier d'Ophtalmologie des Quinze-VingtCentre National Hospitalier d'Ophtalmologie des Quinze-Vingt
Paris, Paris, France
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Department of Neurology, University of Munich, Friedrich-Baur-Institute
Munich, 80336, Germany
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Department of Ophthalmology, Emory University School of Medicine
Atlanta, Georgia, 30322, United States
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Doheny Eye Center, University of California, Los Angeles
Los Angeles, California, 91105, United States
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IRCCS Istituto delle Scienze Neurologiche di Bologna, UOC Clinica Neurologica, Ospedale Bellaria
Bologna, Bologna, Italy
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Moorfields Eye Hospital NHS Foundation Trust
London, EC1V 2PD, United Kingdom
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Neuro Ophthalmologic Associates, Wills Eye Hospital, Thomas Jefferson University
Philadelphia, Pennsylvania, 19107, United States
Conditions
Explore the condition pages connected to this study.