Inherited and age-related retinal degeneration is the hallmark of a large group of
heterogeneous diseases and is the main cause of untreatable blindness today. Genetic …

Until recently, there was no approved treatment for a retinal degenerative disease.
Subretinal injection of a recombinant adeno-associated virus (AAV) delivering the normal …

Background Phase 1 studies have shown potential benefit of gene replacement in RPE65-
mediated inherited retinal dystrophy. This phase 3 study assessed the efficacy and safety of …

Purpose To report the durability of voretigene neparvovec-rzyl (VN) adeno-associated viral
vector–based gene therapy for RPE65 mutation–associated inherited retinal dystrophy …

Highlights•Voretigene neparvovec-rzyl has not been shown to cure most patients.•Safety
concerns of voretigene neparvovec-rzyl are unlike most medicines.•Total societal costs of …

Located between vessels of the choriocapillaris and light-sensitive outer segments of the
photoreceptors, the retinal pigment epithelium (RPE) closely interacts with photoreceptors in …

Retinal pigment epithelial cells (RPE) constitute a simple layer of cuboidal cells that are
strategically situated behind the photoreceptor (PR) cells. The inconspicuousness of this …

Early-onset, severe retinal dystrophy caused by mutations in the gene encoding retinal
pigment epithelium–specific 65-kD protein (RPE65) is associated with poor vision at birth …

Background Safety and efficacy have been shown in a phase 1 dose-escalation study
involving a unilateral subretinal injection of a recombinant adeno-associated virus (AAV) …

Leber congenital amaurosis (LCA) is a group of autosomal recessive blinding retinal
diseases that are incurable. One molecular form is caused by mutations in the RPE65 …