Gene therapy hope for kids and adults with rare Blindness-Causing mutation

This early-phase study tests a gene therapy called LX107 for people with a rare inherited eye disease caused by mutations in the AIPL1 gene. The treatment is given as an injection under the retina to try to improve vision and slow disease progression. The study will include 13 adults and children aged 4 and older, and will focus on safety and whether the therapy helps with mobility in different lighting conditions.