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.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for LEBER HEREDITARY OPTIC NEUROPATHY (OPTIC, ATROPHY, HEREDITARY, LEBER) are added.
By submitting, you agree to our Terms of use
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.