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Promotion of endoplasmic reticulum retrotranslocation by overexpression of E3 ubiquitin‐protein ligase synoviolin 1 reduces endoplasmic reticulum stress and preserves cone photoreceptors in cyclic nucleotide‐gated channel deficiency.
- Published in:
- FASEB Journal, 2024, v. 38, n. 17, p. 1, doi. 10.1096/fj.202400198R
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- Article
Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa.
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- Frontiers in Neuroscience, 2017, p. 1, doi. 10.3389/fnins.2017.00342
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- Article
Targeting iodothyronine deiodinases locally in the retina is a therapeutic strategy for retinal degeneration.
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- FASEB Journal, 2016, v. 30, n. 12, p. 4313, doi. 10.1096/fj.201600715R
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- Article
Targeted Gene Delivery to the Enteric Nervous System Using AAV: A Comparison Across Serotypes and Capsid Mutants.
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- Molecular Therapy, 2015, v. 23, n. 3, p. 488, doi. 10.1038/mt.2015.7
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- Article
NADH-dehydrogenase Type-2 Suppresses Irreversible Visual Loss and Neurodegeneration in the EAE Animal Model of MS.
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- Molecular Therapy, 2013, v. 21, n. 10, p. 1876, doi. 10.1038/mt.2013.104
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- Article
Transient Photoreceptor Deconstruction by CNTF Enhances rAAV-Mediated Cone Functional Rescue in Late Stage CNGB3-Achromatopsia.
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- Molecular Therapy, 2013, v. 21, n. 6, p. 1131, doi. 10.1038/mt.2013.50
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- Article
A Comprehensive Review of Retinal Gene Therapy.
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- Molecular Therapy, 2013, v. 21, n. 3, p. 509, doi. 10.1038/mt.2012.280
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- Article
Viral-mediated vision rescue of a novel AIPL1 cone-rod dystrophy model.
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- Human Molecular Genetics, 2015, v. 24, n. 3, p. 670, doi. 10.1093/hmg/ddu487
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- Article
RD3 gene delivery restores guanylate cyclase localization and rescues photoreceptors in the Rd3 mouse model of Leber congenital amaurosis 12.
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- Human Molecular Genetics, 2013, v. 22, n. 19, p. 3894, doi. 10.1093/hmg/ddt244
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- Article
Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosis.
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- Human Molecular Genetics, 2011, v. 20, n. 23, p. 4569, doi. 10.1093/hmg/ddr391
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- Article
ELOVL4-Mediated Production of Very Long-Chain Ceramides Stabilizes Tight Junctions and Prevents Diabetes-Induced Retinal Vascular Permeability.
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- Diabetes, 2018, v. 67, n. 4, p. 769, doi. 10.2337/db17-1034
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- Article
SARM1 depletion rescues NMNAT1-dependent photoreceptor cell death and retinal degeneration.
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- eLife, 2020, p. 1, doi. 10.7554/eLife.62027
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- Article
Novel Methodology for Creating Macaque Retinas with Sortable Photoreceptors and Ganglion Cells.
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- Frontiers in Neuroscience, 2016, v. 10, p. 1, doi. 10.3389/fnins.2016.00551
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- Article
Functional study of two biochemically unusual mutations in GUCY2D Leber congenital amaurosis expressed via adenoassociated virus vector in mouse retinas.
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- Molecular Vision, 2016, v. 22, p. 1
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- Article
Virally delivered Channelrhodopsin-2 Safely and Effectively Restores Visual Function in Multiple Mouse Models of Blindness.
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- Molecular Therapy, 2011, v. 19, n. 7, p. 1220, doi. 10.1038/mt.2011.69
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- Article
Long-term Retinal Function and Structure Rescue Using Capsid Mutant AAV8 Vector in the rd10 Mouse, a Model of Recessive Retinitis Pigmentosa.
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- Molecular Therapy, 2011, v. 19, n. 2, p. 234, doi. 10.1038/mt.2010.273
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- Article
881. Safety, Efficacy and Biodistribution of Recombinant AAV2-RPE65 Vector Delivered by Ocular Subretinal Injection.
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- Molecular Therapy, 2006, v. 13, p. S339, doi. 10.1016/j.ymthe.2006.08.970
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- Article
Safety of Recombinant Adeno-Associated Virus Type 2–RPE65 Vector Delivered by Ocular Subretinal Injection.
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- Molecular Therapy, 2006, v. 13, n. 6, p. 1074, doi. 10.1016/j.ymthe.2006.03.005
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- Article
Improving retinal vascular endothelial cell tropism through rational rAAV capsid design.
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- PLoS ONE, 2023, v. 18, n. 5, p. 1, doi. 10.1371/journal.pone.0285370
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- Article
Cone Phosphodiesterase-6α' Restores Rod Function and Confers Distinct Physiological Properties in the Rod Phosphodiesterase-6β-Deficient rd1O Mouse.
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- Journal of Neuroscience, 2013, v. 33, n. 29, p. 11745, doi. 10.1523/JNEUROSCI.1536-13.2013
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- Article
Inhibitor of Apoptosis-Stimulating Protein of p53 (iASPP) Is Required for Neuronal Survival after Axonal Injury.
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- PLoS ONE, 2014, v. 9, n. 4, p. 1, doi. 10.1371/journal.pone.0094175
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- Article
Natural History of Cone Disease in the Murine Model of Leber Congenital Amaurosis Due to <i>CEP290</i> Mutation: Determining the Timing and Expectation of Therapy.
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- PLoS ONE, 2014, v. 9, n. 3, p. 1, doi. 10.1371/journal.pone.0092928
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- Article
Recombinant AAV-Mediated <i>BEST1</i> Transfer to the Retinal Pigment Epithelium: Analysis of Serotype-Dependent Retinal Effects.
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- PLoS ONE, 2013, v. 8, n. 10, p. 1, doi. 10.1371/journal.pone.0075666
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- Article
Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors.
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- PLoS ONE, 2013, v. 8, n. 4, p. 1, doi. 10.1371/journal.pone.0062097
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- Article
Evaluation of Lateral Spread of Transgene Expression following Subretinal AAV–Mediated Gene Delivery in Dogs.
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- PLoS ONE, 2013, v. 8, n. 4, p. 1, doi. 10.1371/journal.pone.0060218
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- Article
AAV-Mediated Cone Rescue in a Naturally Occurring Mouse Model of CNGA3-Achromatopsia.
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- PLoS ONE, 2012, v. 7, n. 4, p. 1, doi. 10.1371/journal.pone.0035250
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- Article
Ab-Externo AAV-Mediated Gene Delivery to the Suprachoroidal Space Using a 250 Micron Flexible Microcatheter.
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- PLoS ONE, 2011, v. 6, n. 2, p. 1, doi. 10.1371/journal.pone.0017140
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- Article
Functional and Behavioral Restoration of Vision by Gene Therapy in the Guanylate Cyclase-1 (GC1) Knockout Mouse.
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- PLoS ONE, 2010, v. 5, n. 6, p. 1, doi. 10.1371/journal.pone.0011306
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- Article
Patients and animal models of CNGβ1-deficient retinitis pigmentosa support gene augmentation approach.
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- 2018
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- journal article
Safety and Effects of the Vector for the Leber Hereditary Optic Neuropathy Gene Therapy Clinical Trial.
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- JAMA Ophthalmology, 2014, v. 132, n. 4, p. 409, doi. 10.1001/jamaophthalmol.2013.7630
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- Article
A Novel Mouse Model of MYO7A USH1B Reveals Auditory and Visual System Haploinsufficiencies.
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- Frontiers in Neuroscience, 2019, v. 13, p. 1, doi. 10.3389/fnins.2019.01255
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- Article
Utilizing minimally purified secreted rAAV for rapid and cost-effective manipulation of gene expression in the CNS.
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- Molecular Neurodegeneration, 2020, v. 15, n. 1, p. 1, doi. 10.1186/s13024-020-00361-z
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- Article
Rapid, widespread transduction of the murine myocardium usingself-complementary Adeno-associated virus.
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- Genetic Vaccines & Therapy, 2007, v. 5, p. 13, doi. 10.1186/1479-0556-5-13
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- Article
Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness.
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- Human Molecular Genetics, 2015, v. 24, n. 21, p. 6229, doi. 10.1093/hmg/ddv341
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- Article
Adeno-Associated Virus (AAV) Capsid Stability and Liposome Remodeling During Endo/Lysosomal pH Trafficking.
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- Viruses (1999-4915), 2020, v. 12, n. 6, p. 668, doi. 10.3390/v12060668
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- Article
Small GTPases Rab8a and Rab11a Are Dispensable for Rhodopsin Transport in Mouse Photoreceptors.
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- PLoS ONE, 2016, v. 11, n. 8, p. 1, doi. 10.1371/journal.pone.0161236
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- Article
AAV-Mediated Clarin-1 Expression in the Mouse Retina: Implications for USH3A Gene Therapy.
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- PLoS ONE, 2016, v. 11, n. 2, p. 1, doi. 10.1371/journal.pone.0148874
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- Article
Capsid Mutated Adeno-Associated Virus Delivered to the Anterior Chamber Results in Efficient Transduction of Trabecular Meshwork in Mouse and Rat.
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- PLoS ONE, 2015, v. 10, n. 6, p. 1, doi. 10.1371/journal.pone.0128759
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- Article