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References from Sustained improvement in dark-adapted sensitivity but not BCVA after voretigene neparvovec treatment in a mainland Chinese child with biallelic RPE65-associated LCA2: a case report. Local targets link to admitted publications; unresolved targets remain external evidence.
Gene therapy for retinal degeneration
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10.1167/tvst.9.11.9 · 2020 · External reference
A dominant mutation in RPE65 identified by whole-exome sequencing causes retinitis pigmentosa with choroidal involvement
10.1038/ejhg.2011.86 · 2011 · External reference
Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics
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10.1016/j.xops.2026.101090 · 2026 · External reference
Perifoveal chorioretinal atrophy after subretinal voretigene neparvovec-rzyl for RPE65-Mediated leber congenital amaurosis
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Frequency and phenotypic characteristics of RPE65 mutations in the Chinese population
10.1186/s13023-021-01807-3 · 2021 · External reference
Phenotyping and genotyping inherited retinal diseases: molecular genetics, clinical and imaging features, and therapeutics of macular dystrophies, cone and cone-rod dystrophies, rod-cone dystrophies, leber congenital amaurosis, and cone dysfunction syndromes
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Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years
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ISCEV and IPS guideline for the full-field stimulus test (FST)
10.1007/s10633-023-09962-7 · 2024 · External reference
Voretigene neparvovec: a review in RPE65 mutation-associated inherited retinal dystrophy
10.1007/s40291-020-00475-6 · 2020 · External reference
Recombinant adeno-associated virus gene therapy in light of Luxturna (and Zolgensma and Glybera): where are We, and how did we get here?
10.1146/annurev-virology-092818-015530 · 2019 · External reference
Leber congenital amaurosis/early-onset severe retinal dystrophy: clinical features, molecular genetics and therapeutic interventions
10.1136/bjophthalmol-2016-309975 · 2017 · External reference
Critical-period plasticity in the visual cortex
10.1146/annurev-neuro-061010-113813 · 2012 · External reference
Intraocular route of AAV2 vector administration defines humoral immune response and therapeutic potential
2008 · External reference
Gene therapy following subretinal AAV5 vector delivery is not affected by a previous intravitreal AAV5 vector administration in the partner eye
2009 · External reference
Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis
10.1167/iovs.10-6138 · 2011 · External reference
Unlocking therapeutic potential: dual gene therapy for ameliorating the disease phenotypes in a mouse model of RPE65 Leber congenital amaurosis
10.3389/fmed.2023.1291795 · 2023 · External reference
Unresolved reference
2017 · External reference
Unresolved reference
2026 · External reference
Short-term outcomes of pediatric patients with mild autosomal recessive RPE65-Associated retinal dystrophy treated with voretigene neparvovec
10.1167/tvst.14.8.8 · 2025 · External reference
Unresolved reference
2018 · External reference
Cortical visual function in the rd12 mouse model of leber congenital amarousis (LCA) after gene replacement therapy to restore retinal function
10.1016/j.visres.2006.05.010 · 2006 · External reference
Gene therapy restores vision-dependent behavior as well as retinal structure and function in a mouse model of RPE65 Leber congenital amaurosis
10.1016/j.ymthe.2005.09.001 · 2006 · External reference
Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency
10.1038/gt.2010.29 · 2010 · External reference
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10.1080/08164622.2024.2349565 · 2024 · External reference
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10.1073/pnas.91.4.1206 · 1994 · External reference
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10.1073/pnas.0401200101 · 2004 · External reference
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10.1016/j.brs.2020.12.015 · 2021 · External reference
Unresolved reference
2026 · External reference
Full-field stimulus testing: role in the clinic and as an outcome measure in clinical trials of severe childhood retinal disease
10.1016/j.preteyeres.2021.101000 · 2022 · External reference
Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial
10.1016/s0140-6736(17)31868-8 · 2017 · External reference
Relationship between residual visual field and full-field stimulus testing in patients with late-stage retinal degenerative diseases
10.1038/s41598-024-53474-6 · 2024 · External reference
Chorioretinal atrophy after voretigene Neparvovec therapy in RPE65 mutation-associated inherited retinal disease: longitudinal characterization in multimodal imaging
10.1159/000549548 · 2026 · External reference
Electrical devices for visual restoration
10.1016/j.survophthal.2021.08.008 · 2022 · External reference
Full-field stimulus threshold testing: a scoping review of current practice
10.1038/s41433-023-02636-3 · 2024 · External reference
Clinical and pharmacovigilance safety evaluation of LUXTURNA(®) (voretigene neparvovec-rzyl)
10.1080/15569527.2025.2573463 · 2025 · External reference
Full-field scotopic threshold improvement after voretigene Neparvovec-rzyl treatment correlates with chorioretinal atrophy
10.1016/j.ophtha.2023.02.015 · 2023 · External reference
Chorioretinal atrophy growth after voretigene neparvovec retinotopically is connected to retinal functional rescue
10.1167/tvst.13.2.13 · 2024 · External reference
Genetics and phenotypes of RPE65 mutations in inherited retinal degeneration
2000 · External reference
Advancing therapeutic strategies for inherited retinal degeneration: recommendations from the monaciano symposium
10.1167/iovs.14-16049 · 2015 · External reference
Diseases caused by defects in the visual cycle: retinoids as potential therapeutic agents
10.1146/annurev.pharmtox.47.120505.105225 · 2007 · External reference
Mild RPE65-Associated inherited retinal dystrophies: a multimodal clinical and genetic evaluation
10.1167/tvst.14.11.24 · 2025 · External reference
Gene therapy in retinal dystrophies
10.3390/ijms20225722 · 2019 · External reference
