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RNA Splicing Factor Mutations That Cause Retinitis Pigmentosa Result in Circadian Dysregulation.
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- Journal of Biological Rhythms, 2020, v. 35, n. 1, p. 72, doi. 10.1177/0748730419887876
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- Publication type:
- Article
Refining surgical techniques for efficient posterior semicircular canal gene delivery in the adult mammalian inner ear with minimal hearing loss.
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- Scientific Reports, 2021, v. 11, n. 1, p. 1, doi. 10.1038/s41598-021-98412-y
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- Publication type:
- Article
Neuroplasticity of the Lateral Geniculate Nucleus in Response to Retinal Gene Therapy in a Group of Patients with RPE65 Mutations.
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- Eye & Brain, 2022, v. 14, p. 137, doi. 10.2147/EB.S377275
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- Publication type:
- Article
The Signalling Role of the αvβ5-Integrin Can Impact the Efficacy of AAV in Retinal Gene Therapy.
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- Pharmaceuticals (14248247), 2012, v. 5, n. 5, p. 447, doi. 10.3390/ph5050447
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- Publication type:
- Article
Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter.
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- EMBO Molecular Medicine, 2014, v. 6, n. 9, p. 1175, doi. 10.15252/emmm.201404077
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- Publication type:
- Article
Central visual pathways affected by degenerative retinal disease before and after gene therapy.
- Published in:
- Brain: A Journal of Neurology, 2024, v. 147, n. 9, p. 3234, doi. 10.1093/brain/awae096
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- Publication type:
- Article
Disruption of CEP290 microtubule/membrane-binding domains causes retinal degeneration.
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- Journal of Clinical Investigation, 2013, v. 123, n. 10, p. 4525, doi. 10.1172/JCI69448
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- Article
Disruption of intraflagellar protein transport in photoreceptor cilia causes Leber congenital amaurosis in humans and mice.
- Published in:
- 2011
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- Publication type:
- journal article
The human visual cortex responds to gene therapy-mediated recovery of retinal function.
- Published in:
- 2011
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- Publication type:
- journal article
Lighting a candle in the dark: advances in genetics and gene therapy of recessive retinal dystrophies.
- Published in:
- Journal of Clinical Investigation, 2010, v. 120, n. 9, p. 3042, doi. 10.1172/JCI42258
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- Publication type:
- Article
Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.
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- 2008
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- Publication type:
- journal article
Retinal ganglion cell loss induced by acute optic neuritis in a relapsing model of multiple sclerosis.
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- Multiple Sclerosis (13524585), 2006, v. 12, n. 5, p. 526, doi. 10.1177/1352458506070629
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- Article
Comparative AAV-eGFP Transgene Expression Using Vector Serotypes 1–9, 7m8, and 8b in Human Pluripotent Stem Cells, RPEs, and Human and Rat Cortical Neurons.
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- Stem Cells International, 2019, p. 1, doi. 10.1155/2019/7281912
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- Publication type:
- Article
Improving the Quantification of the Lateral Geniculate Nucleus in Magnetic Resonance Imaging Using a Novel 3D-Edge Enhancement Technique.
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- Frontiers in Computational Neuroscience, 2021, v. 15, p. 1, doi. 10.3389/fncom.2021.708866
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- Publication type:
- Article
Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting.
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- Frontiers in Neuroscience, 2017, p. 1, doi. 10.3389/fnins.2017.00503
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- Publication type:
- Article
Protocols for Visually Guided Navigation Assessment of Efficacy of Retina-Directed Cell or Gene Therapy in Canines.
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- Frontiers in Neuroscience, 2017, p. 1, doi. 10.3389/fnins.2017.00215
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- Publication type:
- Article
Gene therapy restores vision in a canine model of childhood blindness.
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- Nature Genetics, 2001, v. 28, n. 1, p. 92, doi. 10.1038/88327
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- Publication type:
- Article
Mutation of a nuclear receptor gene, NR2E3, causes enhanced S cone syndrome, a disorder of retinal cell fate.
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- Nature Genetics, 2000, v. 24, n. 2, p. 127, doi. 10.1038/72777
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- Article
Rococo and Other Worlds: Selected Poems.
- Published in:
- 2014
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- Publication type:
- Book Review
Temporal and spatial regulation of gene expression mediated by the promoter for the human tissue inhibitor of metalloproteinases-3 (TIMP-3)-encoding gene.
- Published in:
- Developmental Dynamics, 1998, v. 211, n. 3, p. 228, doi. 10.1002/(SICI)1097-0177(199803)211:3<228::AID-AJA4>3.0.CO;2-J
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- Publication type:
- Article
Optical absorption, depolarization, and scatter of epitaxial single-crystal chemical-vapor-deposited diamond at 1.064 μm.
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- Optical Engineering, 2007, v. 46, n. 6, p. 64002, doi. 10.1117/1.2748044
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- Publication type:
- Article
Instrument for on-line monitoring of surface roughness of machined surfaces.
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- Optical Engineering, 2000, v. 39, n. 12, p. 3247, doi. 10.1117/1.1318786
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- Publication type:
- Article
An Autogenously Regulated Expression System for Gene Therapeutic Ocular Applications.
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- Scientific Reports, 2015, p. 17105, doi. 10.1038/srep17105
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- Publication type:
- Article
Correction: AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models.
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- PLoS ONE, 2015, v. 10, n. 6, p. 1, doi. 10.1371/journal.pone.0129982
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- Publication type:
- Article
fMRI of Retina-Originated Phosphenes Experienced by Patients with Leber Congenital Amaurosis.
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- PLoS ONE, 2014, v. 9, n. 1, p. 1, doi. 10.1371/journal.pone.0086068
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- Publication type:
- Article
AAV-Mediated Gene Therapy for Choroideremia: Preclinical Studies in Personalized Models
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- PLoS ONE, 2013, v. 8, n. 5, p. 1, doi. 10.1371/journal.pone.0061396
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- Publication type:
- Article
AAV9 Targets Cone Photoreceptors in the Nonhuman Primate Retina.
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- PLoS ONE, 2013, v. 8, n. 1, p. 1, doi. 10.1371/journal.pone.0053463
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- Article
Nicotinic acid as adjuvant therapy in newly admitted schizophrenic patients.
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- Canadian Medical Association Journal, 1970, v. 102, n. 9, p. 939
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- Publication type:
- Article
AAV2.7m8 is a powerful viral vector for inner ear gene therapy.
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- Nature Communications, 2019, v. 10, n. 1, p. 1, doi. 10.1038/s41467-018-08243-1
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- Publication type:
- Article
Compensatory Cross-Modal Plasticity Persists After Sight Restoration.
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- Frontiers in Neuroscience, 2020, v. 14, p. 1, doi. 10.3389/fnins.2020.00291
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- Publication type:
- Article
Pupillometric analysis for assessment of gene therapy in Leber Congenital Amaurosis patients.
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- BioMedical Engineering OnLine, 2012, v. 11, n. 1, p. 40, doi. 10.1186/1475-925X-11-40
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- Publication type:
- Article
Short-term Assessment of Subfoveal Injection of Adeno-Associated Virus-Mediated hCHM Gene Augmentation in Choroideremia Using Adaptive Optics Ophthalmoscopy.
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- 2022
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- Publication type:
- journal article
Utility of In Vitro Mutagenesis of RPE65 Protein for Verification of Mutational Pathogenicity Before Gene Therapy.
- Published in:
- 2019
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- Publication type:
- journal article
Institutional Conflict of Interest.
- Published in:
- 2016
- By:
- Publication type:
- Letter
Basic fibroblast growth factor induces a transformed phenotype in normal human melanocytes.
- Published in:
- Oncogene, 1999, v. 18, n. 47, p. 6469, doi. 10.1038/sj.onc.1203066
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- Publication type:
- Article
Gene therapy and retinitis pigmentosa: advances and future challenges.
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- BioEssays, 2001, v. 23, n. 7, p. 662, doi. 10.1002/bies.1092
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- Publication type:
- Article
Resource reviews.
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- 1995
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- Publication type:
- Book Review
A CRISPR view of the 2020 Nobel Prize in Chemistry.
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- 2021
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- Publication type:
- journal article
Blinded by the light: a nonhuman primate model of achromatopsia.
- Published in:
- 2019
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- Publication type:
- journal article
Exploiting metabolic and antioxidant pathways to maintain vision in blinding disease.
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- Journal of Clinical Investigation, 2015, v. 125, n. 4, p. 1390, doi. 10.1172/JCI80821
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- Publication type:
- Article
Depicting brighter possibilities for treating blindness.
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- Science Translational Medicine, 2019, v. 11, n. 494, p. 1, doi. 10.1126/scitranslmed.aax2324
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- Publication type:
- Article
Plasticity of the human visual system after retinal gene therapy in patients with Leber’s congenital amaurosis.
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- Science Translational Medicine, 2015, v. 7, n. 296, p. 1, doi. 10.1126/scitranslmed.aaa8791
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- Publication type:
- Article
Basal exon skipping and genetic pleiotropy: A predictive model of disease pathogenesis.
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- Science Translational Medicine, 2015, v. 7, n. 291, p. 1, doi. 10.1126/scitranslmed.aaa5370
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- Publication type:
- Article
Novel mobility test to assess functional vision in patients with inherited retinal dystrophies.
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- Clinical & Experimental Ophthalmology, 2018, v. 46, n. 3, p. 247, doi. 10.1111/ceo.13022
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- Publication type:
- Article
In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery.
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- Human Molecular Genetics, 2016, v. 25, n. 12, p. 2552, doi. 10.1093/hmg/ddw118
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- Publication type:
- Article
Nxnl2 splicing results in dual functions in neuronal cell survival and maintenance of cell integrity.
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- Human Molecular Genetics, 2012, v. 21, n. 10, p. 2298, doi. 10.1093/hmg/dds050
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- Publication type:
- Article
Seeing the Light.
- Published in:
- Science Translational Medicine, 2013, v. 5, n. 175, p. 1, doi. 10.1126/scitranslmed.3005798
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- Publication type:
- Article
AAV2 Gene Therapy Readministration in Three Adults with Congenital Blindness.
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- Science Translational Medicine, 2012, v. 4, n. 120, p. 1, doi. 10.1126/scitranslmed.3002865
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- Publication type:
- Article
Dosage Thresholds for AAV2 and AAV8 Photoreceptor Gene Therapy in Monkey.
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- Science Translational Medicine, 2011, v. 3, n. 88, p. 1, doi. 10.1126/scitranslmed.3002103
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- Publication type:
- Article
Safety and Efficacy of Subretinal Readministration of a Viral Vector in Large Animals to Treat Congenital Blindness.
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- Science Translational Medicine, 2010, v. 2, n. 21, p. 1, doi. 10.1126/scitranslmed.3000659
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- Publication type:
- Article