AMAUROSIS OF LEBER

Clinical trials for AMAUROSIS OF LEBER explained in plain language.

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Completed 1

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

⭐️ CURE ⭐️ Completed

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 …

Matched conditions: AMAUROSIS OF LEBER

Phase: PHASE1 • Sponsor: University of Pennsylvania • Aim: ⭐️ CURE ⭐️

Last updated Jul 19, 2026 00:00 UTC

AMAUROSIS OF LEBER

Get alerted when new AMAUROSIS OF LEBER trials appear

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