Gene therapies in pediatric ophthalmology

Alejandra Daruich (1, 2) , Matthieu Robert (1, 3) , Dominique Bremond-Gignac (1, 2)

1 Service d'ophtalmologie [CHU Necker]
2 CRC (UMR_S_1138 / U1138) - Centre de Recherche des Cordeliers
3 CB - CB - Centre Borelli - UMR 9010

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.

Keywords: gene therapy; children; voretigene neparvovec; pediatric ophthalmology; genetic ocular diseases; aniridia; optogenetic therapy; ataluren

Domains: Sensory Organs; Biotechnology; Pediatrics

Alejandra Daruich, Matthieu Robert, Dominique Bremond-Gignac. Gene therapies in pediatric ophthalmology. Frontiers in Ophthalmology, 2023, 3, pp.1188522. ⟨10.3389/fopht.2023.1188522⟩. ⟨hal-04544311⟩