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

14 Sept 2022

Abstract excerpt

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 persistence of different recombinant adeno-associated virus (rAAV) vector genomes and the preclinical and clinical evidence...

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PMID: 36103843
DOI: 10.1159/000526317

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