- Gene therapy was evaluated for pediatric patients with AIPL1-associated severe retinal dystrophy.
- Substantial improvements in visual acuity and functional vision were observed.
- The study led by Dr. Michel Michaelides showed promising results after administering the rAAV8 vector carrying the AIPL1 coding sequence.
- Safety and efficacy were assessed using various tests including visual acuity tests, functional vision observations, and visual evoked potentials.
- Limitations of the study included the low number of treated patients, absence of a control group, and challenges in measuring visual function in young children with severe sight impairment.
Vision Improvements With Gene Therapy for Pediatric Retinal Dystrophy
Conexiant
March 13, 2025
