Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year - Test2 - elhamkhani - TriplyDB

Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

http://www.wikidata.org/entity/Q24634801

about

Felix d’Herelle and our microbial future Microphthalmia-associated transcription factor (MITF) promotes differentiation of human retinal pigment epithelium (RPE) by regulating microRNAs-204/211 expression Gene therapy rescues cone function in congenital achromatopsia.Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration Clinical development of gene therapy: results and lessons from recent successes Manufacturing of recombinant adeno-associated viral vectors for clinical trials Parvovirus glycan interactions Regenerative medicine for the special senses: restoring the inputs.Promising and delivering gene therapies for vision loss Gene therapy of inherited retinal degenerations: prospects and challenges AAV-mediated gene therapy in mouse models of recessive retinal degeneration A comprehensive review of retinal gene therapy Canine CNGA3 Gene Mutations Provide Novel Insights into Human Achromatopsia-Associated Channelopathies and Treatment Successful amelioration of mitochondrial optic neuropathy using the yeast NDI1 gene in a rat animal model MicroRNA-restricted transgene expression in the retina Bicistronic lentiviruses containing a viral 2A cleavage sequence reliably co-express two proteins and restore vision to an animal model of LCA1 Structural Insights into Adeno-Associated Virus Serotype 5 Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness Retinal Neuroprotective Effects of Flibanserin, an FDA-Approved Dual Serotonin Receptor Agonist-Antagonist Whirlin replacement restores the formation of the USH2 protein complex in whirlin knockout photoreceptors Development and utility of an internal threshold control (ITC) real-time PCR assay for exogenous DNA detection Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.Assessment of Rod, Cone, and Intrinsically Photosensitive Retinal Ganglion Cell Contributions to the Canine Chromatic Pupillary Response Gene therapy regenerates protein expression in cone photoreceptors in Rpe65(R91W/R91W) mice.Scalable downstream strategies for purification of recombinant adeno- associated virus vectors in light of the properties.Optogenetics.At the frontier of vision research: the National Eye Institute celebrates 40 years Inhibition of choroidal neovascularization in a nonhuman primate model by intravitreal administration of an AAV2 vector expressing a novel anti-VEGF molecule.Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse.Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapy The retinal pigment epithelium in health and disease Directed evolution of adeno-associated virus for enhanced gene delivery and gene targeting in human pluripotent stem cells.Long-term gene therapy causes transgene-specific changes in the morphology of regenerating retinal ganglion cells.Loss of Raf-1 kinase inhibitory protein delays early-onset severe retinal ciliopathy in Cep290rd16 mouse.The potential of nanomedicine therapies to treat neovascular disease in the retina.Diagnostic challenges in retinitis pigmentosa: genotypic multiplicity and phenotypic variability.Efficient transduction and optogenetic stimulation of retinal bipolar cells by a synthetic adeno-associated virus capsid and promoter.Expression of wild-type Rp1 protein in Rp1 knock-in mice rescues the retinal degeneration phenotype Adeno-associated virus: a key to the human genome?

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P2860

Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

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2009 nî lūn-bûn

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2009 թուականի Սեպտեմբերին հրատարակուած գիտական յօդուած

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2009 թվականի սեպտեմբերին հրատարակված գիտական հոդված

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2009年の論文

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2009年論文

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Human RPE65 gene therapy for L ...... rovements and safety at 1 year

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P2860

Latent adeno-associated virus infection elicits humoral but not cell-mediated immune responses in a nonhuman primate model

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

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

Human cone photoreceptor dependence on RPE65 isomerase

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.

Identifying photoreceptors in blind eyes caused by RPE65 mutations: Prerequisite for human gene therapy success.

Leber congenital amaurosis: genes, proteins and disease mechanisms.

Retinal laminar architecture in human retinitis pigmentosa caused by Rhodopsin gene mutations

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