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.
Overview
- 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.