Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis. - Wikidata - wikimedia - TriplyDB

Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis.

Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis.

http://www.wikidata.org/entity/Q40292682

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Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial AAV2 delivery of Flt23k intraceptors inhibits murine choroidal neovascularization Comparative analysis of DNA nanoparticles and AAVs for ocular gene delivery Structural Insights into Adeno-Associated Virus Serotype 5 Human cone photoreceptor dependence on RPE65 isomerase The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation.RPE65: role in the visual cycle, human retinal disease, and gene therapy.AAV2-mediated subretinal gene transfer of hIFN-α attenuates experimental autoimmune uveoretinitis in mice.Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy My career path for developing gene therapy for blinding diseases: the importance of mentors, collaborators, and opportunities.A 350 bp region of the proximal promoter of Rds drives cell-type specific gene expression.Transduction of the inner mouse retina using AAVrh8 and AAVrh10 via intravitreal injection Differential proteomics and functional research following gene therapy in a mouse model of Leber congenital amaurosis Altered expression of retinal molecular markers in the canine RPE65 model of Leber congenital amaurosis AAV-mediated photoreceptor transduction of the pig cone-enriched retina.Therapeutic challenges to retinitis pigmentosa: from neuroprotection to gene therapy.Gene therapy for ocular diseases Gene therapy for choroideremia using an adeno-associated viral (AAV) vector.Key enzymes of the retinoid (visual) cycle in vertebrate retina Republished review: Gene therapy for ocular diseases.Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse.Subretinal AAV2.COMP-Ang1 suppresses choroidal neovascularization and vascular endothelial growth factor in a murine model of age-related macular degeneration.Understanding hereditary diseases using the dog and human as companion model systems.Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors.Suprachoroidal electrotransfer: a nonviral gene delivery method to transfect the choroid and the retina without detaching the retina.Novel Methodology for Creating Macaque Retinas with Sortable Photoreceptors and Ganglion Cells.Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-CB-hRS1, a Recombinant Adeno-Associated Virus Vector Expressing Retinoschisin.Safety and Biodistribution Evaluation in Cynomolgus Macaques of rAAV2tYF-PR1.7-hCNGB3, a Recombinant AAV Vector for Treatment of Achromatopsia Transient photoreceptor deconstruction by CNTF enhances rAAV-mediated cone functional rescue in late stage CNGB3-achromatopsia.Optimized allotopic expression of the human mitochondrial ND4 prevents blindness in a rat model of mitochondrial dysfunction.Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics.Rescue of sight by gene therapy - closer than it may appear.Ocular and systemic safety of a recombinant AAV8 vector for X-linked retinoschisis gene therapy: GLP studies in rabbits and Rs1-KO mice.Nonviral ocular gene therapy: assessment and future directions.Functional study of two biochemically unusual mutations in GUCY2D Leber congenital amaurosis expressed via adenoassociated virus vector in mouse retinas.The use of canine models of inherited retinal degeneration to test novel therapeutic approaches.An analysis of retinal gene therapy clinical trials.Correction of Monogenic and Common Retinal Disorders with Gene Therapy.

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P2860

Safety in nonhuman primates of ocular AAV2-RPE65, a candidate treatment for blindness in Leber congenital amaurosis.

http://www.wikidata.org/entity/Q40292682

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