Sustained Rescue of Rod Function and Probable Non-Cell-Autonomous Rescue of Cones after Gene Therapy
- PMID: 31443790
- PMCID: PMC9121307
- DOI: 10.1016/j.ophtha.2019.03.007
Sustained Rescue of Rod Function and Probable Non-Cell-Autonomous Rescue of Cones after Gene Therapy
Comment on
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Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.Ophthalmology. 2019 Sep;126(9):1273-1285. doi: 10.1016/j.ophtha.2019.06.017. Epub 2019 Jun 22. Ophthalmology. 2019. PMID: 31443789 Clinical Trial.
References
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- Maguire AM, Russell S, Wellman JA, et al. Efficacy, safety, and durability of voretigene neparvovec-rzyl in RPE65 mutation-associated inherited retinal dystrophy: results of phase 1 and 3 trials. Ophthalmology. 2019;126:1273–1285. - PubMed
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- Kunchithapautham K, Coughlin B, Crouch RK, Rohrer B. Cone outer segment morphology and cone function in the Rpe65−/− Nrl−/− mouse retina are amenable to retinoid replacement. Invest Ophthalmol Vis Sci. 2009;50(10): 4858–4864. - PubMed
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