Gene therapies in pediatric ophthalmology

Alejandra Daruich; Matthieu P Robert; Dominique Bremond-Gignac

doi:10.3389/fopht.2023.1188522

Gene therapies in pediatric ophthalmology

Front Ophthalmol (Lausanne). 2023 Jun 5:3:1188522. doi: 10.3389/fopht.2023.1188522. eCollection 2023.

Authors

Alejandra Daruich^{1

2}, Matthieu P Robert^{1

3}, Dominique Bremond-Gignac^{1

2}

Affiliations

¹ Ophthalmology Department, Necker-Enfants Malades University Hospital, Assistance Publique-Hôpitaux de Paris (AP-HP), Paris Cité University, Paris, France.
² INSERM, UMRS1138, Team 17, From physiopathology of ocular diseases to clinical development, Sorbonne Paris Citeí University, Centre de Recherche des Cordeliers, Paris, France.
³ Borelli Centre, UMR 9010 CNRS-SSA-ENS Paris Saclay-Paris Cité University, Paris, France.

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.

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

Publication types

Review