Gene Therapy Reverses a Rare Cause of Vision Loss [Video]

Key Takeaways

A rare inherited illness called LCA1 typically causes people to start losing their sight in early childhoodResearchers say a form of gene therapy injected below the retina can bring back vision to patientsTwo patients experienced a 10,000-fold boost in vision

FRIDAY, Sept. 6, 2024 (HealthDay News) — Gene therapy may restore vision to children and adults robbed of their sight by a rare inherited condition called Leber congenital amaurosis, researchers report.

The illness is caused by mutations in the GUCY2D gene, which is critical to producing proteins that enable vision. People with this form of Leber congenital amaurosis (LCA1) typically lose their vision in early childhood. Just under 100,000 people are thought to be affected worldwide.

However, a team of researchers at the University of Pennsylvania say that a new gene therapy injected under the retinas of children and adults with LCA1 improved vision by 100 to 10,000 times.

“Even …