AUTHOR=Daruich Alejandra , Robert Matthieu P. , Bremond-Gignac Dominique TITLE=Gene therapies in pediatric ophthalmology JOURNAL=Frontiers in Ophthalmology VOLUME=3 YEAR=2023 URL=https://www.frontiersin.org/journals/ophthalmology/articles/10.3389/fopht.2023.1188522 DOI=10.3389/fopht.2023.1188522 ISSN=2674-0826 ABSTRACT=

Genetic pediatric eye disease frequently leads to severe vision impairment or blindness. Voretigene neparvovec is the first approved gene therapy for an inherited retinal dystrophy (IRD). Voretigene neparvovec has been shown to be well tolerated and safe, with encouraging results in terms of efficacy, mainly when administered early in childhood. While we assisted at the first gene therapy available in clinical practice for an IRD, some questions remain unanswered, especially when gene therapy is delivered in young children. We review here the most recent reports and promising ongoing studies concerning various approaches on gene therapy in pediatric ophthalmology.