Cure My Disease

Don't miss your cure!

 Log in / Sign up

One-Time gene injection aims to restore sight in rare childhood blindness

NCT ID NCT00481546

Completed ⭐️ CURE ⭐️ Sponsor: University of Pennsylvania Source: ClinicalTrials.gov ↗

First seen Jul 17, 2026 · Last updated Jul 17, 2026

Summary

This trial tests a gene therapy for people with a rare inherited eye disease called Leber congenital amaurosis, caused by mutations in the RPE65 gene. A harmless virus is used to deliver a working copy of the gene into cells under the retina. The goal is to restore vision with a single injection. The study includes both adults and children and is primarily checking for safety.

What this could mean

Our plain-language read of the trial. This is informational only — not medical advice or a prediction.

Active substance
a gene therapy vector carrying a working copy of the RPE65 gene, injected under the retina
What this could lead to
If successful, this could lead to a one-time gene therapy that restores vision in people with a rare, inherited form of blindness.
What could go wrong
This is an early-phase trial focused on safety, so it is too soon to know if the treatment will work. There are risks from the injection itself and the immune system may react to the virus used to deliver the gene.

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 AMAUROSIS OF LEBER are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Leber congenital amaurosis retinal disorder

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

AMAUROSIS OF LEBER RETINAL DISEASES

Contacts and locations

Locations

  • Scheie Eye Institute, University of Pennsylvania

    Philadelphia, Pennsylvania, 19104, United States

  • Shands Children's Hospital, University of Florida

    Gainesville, Florida, 32610, United States

More trials for these conditions

Other studies related to the condition(s) this trial covers.