Gene Therapy Shows Promise in Restoring Vision for Patients with Rare Genetic Disorder

Patients with Leber congenital amaurosis type 1 experienced significant vision improvements in recent clinical trials.

The therapy, ATSN-101, was tested on 15 patients, including three children, with mutations in the GUCY2D gene.
Participants who received the highest doses reported up to a 10,000-fold improvement in vision.
Improvements were noticeable within a month and lasted for at least a year.
The study confirmed the safety of the treatment, with only minor side effects reported.
Researchers aim to refine the therapy and expand its application to other forms of congenital retinal blindness.