Gene therapy demonstrates visual improvement in retinal dystrophy for at least 5 years: Study
Voretigene neparvovec-rzyl gene therapy demonstrates visual improvement in patients with retinal dystrophy and the beneficial effect lasts for at least five years, finds clinical trial.
The results of study suggest that visual improvements were maintained for at least five years in the original intervention group (bilateral subretinal Voretigene neparvovec-rzyl gene therapy), and four years in the delayed intervention group (Voretigene neparvovec-rzyl gene therapy after 1 year). The findings of the study were presented at the American Academy of Ophthalmology 2020, virtually held annual scientific meeting from November 13 to 15, 2020.
Voretigene neparvovec-rzyl is a gene therapy intended for use in individuals with confirmed biallelic RPE65 mutation-associated retinal dystrophy and viable retinal cells. Mutations in the RPE65 gene are associated with several clinical manifestations including nyctalopia, decreased visual field and decreased visual acuity. Voretigene neparvovec-rzyl uses a non-pathogenic recombinant adeno-associated virus vector serotype 2 (AAV2) to deliver cDNA encoding RPE65 protein to target cells in the retina.
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