Desperate hope: gene therapy shot for one patient with rare blindness
NCT ID NCT03672968
First seen Mar 10, 2026 · Last updated May 14, 2026 · Updated 7 times
Summary
This study provided a single patient with Leber Hereditary Optic Neuropathy (LHON) access to an experimental gene therapy called GS010. The treatment was injected into both eyes to try to protect or restore vision. The goal was to check safety, not to cure the disease.
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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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Conditions
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