One-Time gene shot aims to halt rare blindness
NCT ID NCT02652767
First seen Mar 02, 2026 · Last updated Apr 26, 2026 · Updated 10 times
Summary
This study tested a single injection of GS010 gene therapy in 39 people with Leber Hereditary Optic Neuropathy (LHON) who had vision loss for 6 months or less. The goal was to see if the treatment could improve eyesight compared to a sham injection. While the therapy targets the root genetic cause, it does not eliminate the need for ongoing management, so it is classified as disease control.
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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
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