Desperate hope: gene therapy tested in one patient with rare blindness
NCT ID NCT03672968
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This expanded access program gave a single patient with Leber Hereditary Optic Neuropathy (a genetic cause of vision loss) an experimental gene therapy called GS010. The treatment was injected into both eyes to test safety. Only one person was involved, so the results are very limited.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
GS010 (gene therapy)
What this could lead to
If successful, this could provide a treatment option for patients with this rare genetic eye disease.
What could go wrong
This is a single-patient expanded access program, not a formal trial. Results may not apply to others, and risks include injection-related complications.
Disclaimer
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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.
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
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.