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References from Pre-existing retinal inflammation exacerbates gene therapy-associated uveitis in more rapidly progressive retinal degeneration. Local targets link to admitted publications; unresolved targets remain external evidence.
Real-World Safety and Effectiveness of Voretigene Neparvovec: Results up to 2 Years from the Prospective, Registry-Based PERCEIVE Study
10.3390/biom14010122 · 2024 · 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
Gene therapy for Leber’s congenital amaurosis is safe and effective through 1.5 years after vector administration
10.1038/mt.2009.277 · 2010 · External reference
Safety and efficacy of gene transfer for Leber’s congenital amaurosis
10.1056/nejmoa0802315 · 2008 · External reference
Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia
10.1038/s41591-018-0185-5 · 2018 · External reference
Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR
10.1038/s41591-020-0763-1 · 2020 · External reference
Gene therapy in children with AIPL1-associated severe retinal dystrophy: an open-label, first-in-human interventional study
10.1016/s0140-6736(24)02812-5 · 2025 · External reference
Gene Therapies in Clinical Development to Treat Retinal Disorders
10.1007/s40291-024-00722-0 · 2024 · External reference
Gene Therapy-Associated Uveitis (GTAU): Understanding and mitigating the adverse immune response in retinal gene therapy
10.1016/j.preteyeres.2025.101354 · 2025 · External reference
Long-Term Effect of Gene Therapy on Leber’s Congenital Amaurosis
10.1056/nejmoa1414221 · 2015 · External reference
AAV8 Can Induce Innate and Adaptive Immune Response in the Primate Eye
10.1016/j.ymthe.2017.08.018 · 2017 · External reference
Development of retinal atrophy after subretinal gene therapy with voretigene neparvovec
10.1136/bjophthalmol-2021-321023 · 2023 · External reference
Dose-Dependent Progression of Chorioretinal Atrophy at the Injection Site After Subretinal Injection of rAAV2/8 in Nonhuman Primates
10.1016/j.xops.2024.100516 · 2024 · External reference
Retinal Inflammation, Cell Death and Inherited Retinal Dystrophies
10.3390/ijms22042096 · 2021 · External reference
Neuroinflammation in Retinitis Pigmentosa, Diabetic Retinopathy, and Age-Related Macular Degeneration: A Minireview
10.1007/978-3-319-75402-4_23 · 2018 · External reference
The double-edged sword of inflammation in inherited retinal degenerations: Clinical and preclinical evidence for mechanistically and prognostically impactful but treatable complications
10.3389/fcell.2023.1177711 · 2023 · External reference
Importance of Autoimmune Responses in Progression of Retinal Degeneration Initiated by Gene Mutations
10.3389/fmed.2021.672444 · 2021 · External reference
Changes of serum inflammatory molecules and their relationships with visual function in retinitis pigmentosa
2020 · External reference
Intraocular cytokine profile and autoimmune reactions in retinitis pigmentosa, age-related macular degeneration, glaucoma and cataract
10.1111/aos.13899 · 2019 · External reference
Clinical evidence of sustained chronic inflammatory reaction in retinitis pigmentosa
10.1016/j.ophtha.2012.07.006 · 2013 · External reference
Strong upregulation of inflammatory genes accompanies photoreceptor demise in canine models of retinal degeneration
10.1371/journal.pone.0177224 · 2017 · External reference
Immune regulation in the aging retina
10.1016/j.preteyeres.2018.10.003 · 2019 · External reference
Ocular autoimmunity: The price of privilege?
10.1111/j.1600-065x.2006.00439.x · 2006 · External reference
Update on Viral Gene Therapy Clinical Trials for Retinal Diseases
10.1089/hum.2022.159 · 2022 · External reference
Phase 1 Study of Intravitreal Gene Therapy with ADVM-022 for neovascular AMD (OPTIC Trial)
2021 · External reference
Diabetic Macular Edema: Current Understanding, Molecular Mechanisms and Therapeutic Implications
10.3390/cells11213362 · 2022 · External reference
Meta-analysis of Gene Therapy Associated Uveitis (GTAU)
2024 · External reference
Characterizing the cellular immune response to subretinal AAV gene therapy in the murine retina
10.1016/j.omtm.2021.05.011 · 2021 · External reference
A retinitis pigmentosa GTPase regulator (RPGR)- deficient mouse model for X-linked retinitis pigmentosa (RP3)
10.1073/pnas.97.7.3649 · 2000 · External reference
Impaired glutamylation of RPGRORF15 underlies the cone-dominated phenotype associated with truncating distal ORF15 variants
10.1073/pnas.2208707119 · 2022 · External reference
The first reported case of a deletion of the entire RPGR gene in a family with X-linked retinitis pigmentosa
10.1080/13816810.2022.2083181 · 2022 · External reference
Spectrum of Disease Severity in Patients With X-Linked Retinitis Pigmentosa Due to RPGR Mutations
10.1167/iovs.61.14.36 · 2020 · External reference
Microglial and macroglial dynamics in a model of retinitis pigmentosa
10.1016/j.visres.2023.108268 · 2023 · External reference
Cellular signaling and factors involved in Müller cell gliosis: neuroprotective and detrimental effects
10.1016/j.preteyeres.2009.07.001 · 2009 · External reference
Genetic Rescue of X-Linked Retinoschisis Mouse (Rs1−/y) Retina Induces Quiescence of the Retinal Microglial Inflammatory State Following AAV8-RS1 Gene Transfer and Identifies Gene Networks Underlying Retinal Recovery
10.1089/hum.2020.213 · 2021 · External reference
Assessment of Visual Function with Cotoretigene Toliparvovec in X-Linked Retinitis Pigmentosa in the Randomized XIRIUS Phase 2/3 Study
10.1016/j.ophtha.2024.02.023 · 2024 · External reference
Codon-Optimized RPGR Improves Stability and Efficacy of AAV8 Gene Therapy in Two Mouse Models of X-Linked Retinitis Pigmentosa
10.1016/j.ymthe.2017.05.005 · 2017 · External reference
Microglia activation in retinal degeneration
10.1189/jlb.0207114 · 2007 · External reference
Retinal microglia – A key player in healthy and diseased retina
10.1016/j.pneurobio.2018.05.006 · 2019 · External reference
Overview of General and Discriminating Markers of Differential Microglia Phenotypes
10.3389/fncel.2020.00198 · 2020 · External reference
Fate mapping reveals that microglia and recruited monocyte-derived macrophages are definitively distinguishable by phenotype in the retina
10.1038/srep20636 · 2016 · External reference
Microglial Function Is Distinct in Different Anatomical Locations during Retinal Homeostasis and Degeneration
10.1016/j.immuni.2019.02.007 · 2019 · External reference
Müller Glia Activation in Response to Inherited Retinal Degeneration Is Highly Varied and Disease-Specific
10.1371/journal.pone.0120415 · 2015 · External reference
Müller Glia Reactivity and Development of Gliosis in Response to Pathological Conditions
10.1007/978-3-319-75402-4_37 · 2018 · External reference
Damage-Associated Molecular Patterns (DAMPs) in Retinal Disorders
10.3390/ijms23052591 · 2022 · External reference
Microglia Activation and Inflammation during the Death of Mammalian Photoreceptors
10.1146/annurev-vision-121219-081730 · 2020 · External reference
The speed of change: towards a discontinuity theory of immunity?
10.1038/nri3521 · 2013 · External reference
Characterization of the ocular inflammatory response to AAV reveals divergence by sex and age
10.1016/j.ymthe.2025.01.028 · 2025 · External reference
