Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency. - Wikidata - awgldk - TriplyDB

Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency.

Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency.

http://www.wikidata.org/entity/Q34461299

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AAV-mediated gene therapy in mouse models of recessive retinal degeneration A comprehensive review of retinal gene therapy Structural Characterization of the Dual Glycan Binding Adeno-Associated Virus Serotype 6 AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsia The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.Gene therapy regenerates protein expression in cone photoreceptors in Rpe65(R91W/R91W) mice.Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse.Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy Differential proteomics and functional research following gene therapy in a mouse model of Leber congenital amaurosis Discovery Genetics - The History and Future of Spontaneous Mutation Research.Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.Vector platforms for gene therapy of inherited retinopathies.Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosis Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse.The frequency-response electroretinogram distinguishes cone and abnormal rod function in rd12 mice.Exome capture sequencing identifies a novel mutation in BBS4 Vitreal delivery of AAV vectored Cnga3 restores cone function in CNGA3-/-/Nrl-/- mice, an all-cone model of CNGA3 achromatopsia Trans-Corneal Subretinal Injection in Mice and Its Effect on the Function and Morphology of the Retina.Effects of Subretinal Gene Transfer at Different Time Points in a Mouse Model of Retinal Degeneration.Human galectin 3 binding protein interacts with recombinant adeno-associated virus type 6 AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis.Explant cultures of Rpe65-/- mouse retina: a model to investigate cone opsin trafficking.Transient photoreceptor deconstruction by CNTF enhances rAAV-mediated cone functional rescue in late stage CNGB3-achromatopsia.Cone phosphodiesterase-6α' restores rod function and confers distinct physiological properties in the rod phosphodiesterase-6β-deficient rd10 mouse Complementation test of Rpe65 knockout and tvrm148.Persistence of non-viral vector mediated RPE65 expression: case for viability as a gene transfer therapy for RPE-based diseases Advances in gene therapy technologies to treat retinitis pigmentosa.The Degeneration and Apoptosis Patterns of Cone Photoreceptors in rd11 Mice.AAV-mediated lysophosphatidylcholine acyltransferase 1 (Lpcat1) gene replacement therapy rescues retinal degeneration in rd11 mice.The Rpe65 rd12 allele exerts a semidominant negative effect on vision in mice Gene therapy in animal models of autosomal dominant retinitis pigmentosa Advances in AAV vector development for gene therapy in the retina.Convergence of Human Genetics and Animal Studies: Gene Therapy for X-Linked Retinoschisis.Repurposing an orally available drug for the treatment of geographic atrophy.From compliment to insult: genetics of the complement system in physiology and disease in the human retina.Gene delivery to the retina: from mouse to man.Rare Diseases: Drug Discovery and Informatics Resource.Delivery of CR2-fH Using AAV Vector Therapy as Treatment Strategy in the Mouse Model of Choroidal Neovascularization.RPE65 gene therapy slows cone loss in Rpe65-deficient dogs.Advanced Ocular Injection Techniques for Therapy Approaches.

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P2860

Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency.

http://www.wikidata.org/entity/Q34461299

description

2010 nî lūn-bûn

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2010 թուականի Մարտին հրատարակուած գիտական յօդուած

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2010 թվականի մարտին հրատարակված գիտական հոդված

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2010年の論文

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P2860

Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosis

Vision 1 year after gene therapy for Leber's congenital amaurosis

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

Safety and efficacy of gene transfer for Leber's congenital amaurosis

Mutations in RPE65 cause Leber's congenital amaurosis

Effect of gene therapy on visual function in Leber's congenital amaurosis

Restoration of cone vision in a mouse model of achromatopsia

Speed, spatial, and temporal tuning of rod and cone vision in mouse

Study of rod- and cone-driven oscillatory potentials in mice

Human cone photoreceptor dependence on RPE65 isomerase

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