Gene Therapy for Inherited Retinal Disease: Long-Term Durability of Effect

• Published in: Ophthalmic research Vol. 66; no. 1; pp. 179 - 196
• Main Authors: Leroy, Bart P., Fischer, M. Dominik, Flannery, John G., MacLaren, Robert E., Dalkara, Deniz, Scholl, Hendrik P.N., Chung, Daniel C., Spera, Claudio, Viriato, Daniel, Banhazi, Judit
• Format: Journal Article
• Language: English
• Published: Basel, Switzerland S. Karger AG 01.01.2023; Karger
• Subjects: Analysis; Anopheles; Genes; Genetic aspects; Genetic vectors; Genomes; Genomics; Health aspects; Life Sciences; Review Article; Durability; Viral vector; Gene therapy; RPE65; Inherited retinal disease

Abstract The recent approval of voretigene neparvovec (Luxturna®) for patients with biallelic RPE65 mutation-associated inherited retinal dystrophy with viable retinal cells represents an important step in the development of ocular gene therapies. Herein, we review studies investigating the episomal...