Experimental gene injection aims to save sight in rare eye disease
NCT ID NCT06066008
First seen Apr 29, 2026 · Last updated Jun 22, 2026 · Updated 10 times
Summary
This early-stage trial tested a gene therapy called ZM-01 in just 2 boys aged 3 to 18 with X-linked retinoschisis, a rare genetic eye condition that causes vision loss. The treatment was injected into one eye to check safety and see if it improves vision. The main goal was to monitor side effects, not to prove it works.
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the original study
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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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Wuhan University Renmin Hospital affiliated with Hanchuan Hospital
Xiaogan, Hubei, China
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
ZM-01 (rAAV-hRS1) gene therapy injection
What this could lead to
If successful, this could point toward a treatment that slows vision loss in X-linked retinoschisis.
What could go wrong
This is a very early, tiny trial (only 2 participants) focused on safety, not proof of effectiveness. Gene therapy risks include inflammation or injection-related side effects.
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.