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.