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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
Adeno-associated virus 8-mediated gene therapy for choroideremia: preclinical studies in in vitro and in vivo models.
- Published in:
- Journal of Gene Medicine, 2014, v. 16, n. 5/6, p. 122, doi. 10.1002/jgm.2768
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- Publication type:
- Article
Novel AAV serotypes for improved ocular gene transfer.
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- Journal of Gene Medicine, 2008, v. 10, n. 4, p. 375, doi. 10.1002/jgm.1126
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- Publication type:
- Article
An Autogenously Regulated Expression System for Gene Therapeutic Ocular Applications.
- Published in:
- Scientific Reports, 2015, p. 17105, doi. 10.1038/srep17105
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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.
- Published in:
- Eye & Brain, 2022, v. 14, p. 137, doi. 10.2147/EB.S377275
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- Publication type:
- Article
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
Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting.
- Published in:
- 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.
- Published in:
- Frontiers in Neuroscience, 2017, p. 1, doi. 10.3389/fnins.2017.00215
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- Publication type:
- Article
Author Correction: An Autogenously Regulated Expression System for Gene Therapeutic Ocular Applications.
- Published in:
- Scientific Reports, 2018, v. 8, n. 1, p. 1, doi. 10.1038/s41598-018-35960-w
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- Publication type:
- Article
Nxnl2 splicing results in dual functions in neuronal cell survival and maintenance of cell integrity.
- Published in:
- Human Molecular Genetics, 2012, v. 21, n. 10, p. 2298, doi. 10.1093/hmg/dds050
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- Publication type:
- Article
Mutations in ABCA4 result in accumulation of lipofuscin before slowing of the retinoid cycle: a reappraisal of the human disease sequence.
- Published in:
- Human Molecular Genetics, 2004, v. 13, n. 5, p. 525, doi. 10.1093/hmg/ddh048
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- Publication type:
- Article
Depicting brighter possibilities for treating blindness.
- Published in:
- Science Translational Medicine, 2019, v. 11, n. 494, p. 1, doi. 10.1126/scitranslmed.aax2324
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- Publication type:
- Article
Nonhuman primate models for diabetic ocular neovascularization using AAV2-mediated overexpression of vascular endothelial growth factor.
- Published in:
- 2005
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- Publication type:
- journal article
Gene therapy and retinitis pigmentosa: advances and future challenges.
- Published in:
- BioEssays, 2001, v. 23, n. 7, p. 662, doi. 10.1002/bies.1092
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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.
- Published in:
- EMBO Molecular Medicine, 2014, v. 6, n. 9, p. 1175, doi. 10.15252/emmm.201404077
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- Publication type:
- Article
Pupillometric analysis for assessment of gene therapy in Leber Congenital Amaurosis patients.
- Published in:
- BioMedical Engineering OnLine, 2012, v. 11, n. 1, p. 40, doi. 10.1186/1475-925X-11-40
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- Publication type:
- Article
Switching on the Lights: The Use of Optogenetics to Advance Retinal Gene Therapy.
- Published in:
- 2011
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- Publication type:
- Opinion
Suppression and Replacement Gene Therapy for Autosomal Dominant Disease in a Murine Model of Dominant Retinitis Pigmentosa.
- Published in:
- Molecular Therapy, 2011, v. 19, n. 4, p. 642, doi. 10.1038/mt.2010.293
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- Publication type:
- Article
Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer.
- Published in:
- Molecular Therapy, 2008, v. 16, n. 3, p. 458, doi. 10.1038/sj.mt.6300389
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- Publication type:
- Article
Gene Transfer to Ocular Stem Cells by Early Gestational Intraamniotic Injection of Lentiviral Vector.
- Published in:
- Molecular Therapy, 2007, v. 15, n. 3, p. 579, doi. 10.1038/sj.mt.6300092
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- Publication type:
- Article
In Vivo Delivery of Recombinant Viruses to the Fetal Murine Cochlea: Transduction Characteristics and Long-Term Effects on Auditory Function.
- Published in:
- Molecular Therapy, 2006, v. 14, n. 3, p. 328, doi. 10.1016/j.ymthe.2006.04.003
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- Publication type:
- Article
881. Safety, Efficacy and Biodistribution of Recombinant AAV2-RPE65 Vector Delivered by Ocular Subretinal Injection.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S339, doi. 10.1016/j.ymthe.2006.08.970
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- Publication type:
- Article
886. AAV-Mediated Somatic Gene Transfer as an Approach To Delineate Pathogenic Mechanisms in an Autosomal Dominant Blindness Disorder Resembling Age-Related Macular Degeneration (AMD).
- Published in:
- Molecular Therapy, 2006, v. 13, p. S341, doi. 10.1016/j.ymthe.2006.08.975
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- Publication type:
- Article
92. Gene Transfer to Ocular Stem Cells by Early Gestational Intra-Amniotic Injection of Lentiviral Vector.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S38, doi. 10.1016/j.ymthe.2006.08.111
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- Publication type:
- Article
488. Adeno-Associated Viral Serotype 2/1 Is a Highly Efficient Vector for In Utero Gene Transfer to Hair Cell Precursors.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S189, doi. 10.1016/j.ymthe.2006.08.558
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- Publication type:
- Article
496. AAV-Mediated, Somatic Gene Transfer to RPE Cells in Order To Study the Pathogenesis of Macular Degeneration: Proteomic and Genomic Analysis.
- Published in:
- Molecular Therapy, 2006, v. 13, p. S192, doi. 10.1016/j.ymthe.2006.08.566
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- Publication type:
- Article
Safety of Recombinant Adeno-Associated Virus Type 2–RPE65 Vector Delivered by Ocular Subretinal Injection.
- Published in:
- Molecular Therapy, 2006, v. 13, n. 6, p. 1074, doi. 10.1016/j.ymthe.2006.03.005
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- Publication type:
- Article
Long-Term Restoration of Rod and Cone Vision by Single Dose rAAV-Mediated Gene Transfer to the Retina in a Canine Model of Childhood Blindness
- Published in:
- Molecular Therapy, 2005, v. 12, n. 6, p. 1072, doi. 10.1016/j.ymthe.2005.08.008
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- Publication type:
- Article
135. AAV-Mediated Gene Delivery To Evaluate the Biology of an Inherited Macular Degeneration
- Published in:
- 2005
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- Publication type:
- Abstract
Systemic but not intraocular Epo Gene Transfer Protects the Retina from Light-and Genetic-Induced Degeneration
- Published in:
- Molecular Therapy, 2004, v. 10, n. 5, p. 855, doi. 10.1016/j.ymthe.2004.07.027
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- Publication type:
- Article
In Utero Gene Therapy Rescues Vision in a Murine Model of Congenital Blindness
- Published in:
- Molecular Therapy, 2004, v. 9, n. 2, p. 182, doi. 10.1016/j.ymthe.2003.11.013
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- Publication type:
- Article
Inhibition of Retinal Neovascularization by Intraocular Viral-Mediated Delivery of Anti-angiogenic Agents
- Published in:
- Molecular Therapy, 2002, v. 6, n. 4, p. 490, doi. 10.1006/mthe.2002.0702
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- Publication type:
- Article
Pharmacological Regulation of Protein Expression from Adeno-Associated Viral Vectors in the Eye
- Published in:
- Molecular Therapy, 2002, v. 6, n. 2, p. 238, doi. 10.1006/mthe.2002.0660
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- Publication type:
- Article
A Deviant Immune Response to Viral Proteins and Transgene Product Is Generated on Subretinal Administration of Adenovirus and Adeno-associated Virus
- Published in:
- Molecular Therapy, 2002, v. 5, n. 2, p. 125, doi. 10.1006/mthe.2002.0525
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- Publication type:
- Article
AAV2.7m8 is a powerful viral vector for inner ear gene therapy.
- Published in:
- Nature Communications, 2019, v. 10, n. 1, p. 1, doi. 10.1038/s41467-018-08243-1
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- Publication type:
- Article
A Virtual Reality Orientation and Mobility Test for Inherited Retinal Degenerations: Testing a Proof-of-Concept After Gene Therapy.
- Published in:
- Clinical Ophthalmology, 2021, v. 15, p. 939, doi. 10.2147/OPTH.S292527
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- Publication type:
- 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.
- Published in:
- Stem Cells International, 2019, p. 1, doi. 10.1155/2019/7281912
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- Publication type:
- Article
Resource reviews.
- Published in:
- 1995
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- Publication type:
- Book Review
fMRI of Retina-Originated Phosphenes Experienced by Patients with Leber Congenital Amaurosis.
- Published in:
- 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
- Published in:
- 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.
- Published in:
- PLoS ONE, 2013, v. 8, n. 1, p. 1, doi. 10.1371/journal.pone.0053463
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- Publication type:
- Article
Federal Response to the Opioid Crisis.
- Published in:
- Current HIV/AIDS Reports, 2018, v. 15, n. 4, p. 293, doi. 10.1007/s11904-018-0398-8
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- Publication type:
- Article
Gene therapy restores vision in a canine model of childhood blindness.
- Published in:
- 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.
- Published in:
- Nature Genetics, 2000, v. 24, n. 2, p. 127, doi. 10.1038/72777
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- Publication type:
- Article
Optical absorption, depolarization, and scatter of epitaxial single-crystal chemical-vapor-deposited diamond at 1.064 μm.
- Published in:
- 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.
- Published in:
- Optical Engineering, 2000, v. 39, n. 12, p. 3247, doi. 10.1117/1.1318786
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- Publication type:
- Article
Plasticity of the human visual system after retinal gene therapy in patients with Leber’s congenital amaurosis.
- Published in:
- 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.
- Published in:
- Science Translational Medicine, 2015, v. 7, n. 291, p. 1, doi. 10.1126/scitranslmed.aaa5370
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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.
- Published in:
- Science Translational Medicine, 2012, v. 4, n. 120, p. 1, doi. 10.1126/scitranslmed.3002865
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- Publication type:
- Article