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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 trialHuman RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 yearA comprehensive review of retinal gene therapyGene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 AchromatopsiaTargeting the Nrf2 Signaling Pathway in the Retina With a Gene-Delivered Secretable and Cell-Penetrating PeptideGene therapy prevents photoreceptor death and preserves retinal function in a Bardet-Biedl syndrome mouse model.AAV-mediated cone rescue in a naturally occurring mouse model of CNGA3-achromatopsiaTreatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial.Development of an anti-angiogenic therapeutic model combining scAAV2-delivered siRNAs and noninvasive photoacoustic imaging of tumor vasculature developmentREPEATABILITY AND LONGITUDINAL ASSESSMENT OF FOVEAL CONE STRUCTURE IN CNGB3-ASSOCIATED ACHROMATOPSIA.Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model.The human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.Organizational motifs for ground squirrel cone bipolar cellsImaging the response of the retina to electrical stimulation with genetically encoded calcium indicators.Maintaining ocular safety with light exposure, focusing on devices for optogenetic stimulation.Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse.Genetically targeted binary labeling of retinal neurons.Restoration of visual function in P23H rhodopsin transgenic rats by gene delivery of BiP/Grp78Self-complementary AAV5 vector facilitates quicker transgene expression in photoreceptor and retinal pigment epithelial cells of normal mouse.Role of connective tissue growth factor in the retinal vasculature during development and ischemiaGene therapy with mitochondrial heat shock protein 70 suppresses visual loss and optic atrophy in experimental autoimmune encephalomyelitis.γ-Secretase inhibition of murine choroidal neovascularization is associated with reduction of superoxide and proinflammatory cytokines.In vivo knockdown of Piccolino disrupts presynaptic ribbon morphology in mouse photoreceptor synapses.Self-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency.Improvement and decline in vision with gene therapy in childhood blindness.Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.DICER1/Alu RNA dysmetabolism induces Caspase-8-mediated cell death in age-related macular degeneration.Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile.RNAi-mediated gene suppression in a GCAP1(L151F) cone-rod dystrophy mouse modelEvaluation of lateral spread of transgene expression following subretinal AAV-mediated gene delivery in dogsSafety and effects of the vector for the Leber hereditary optic neuropathy gene therapy clinical trial.Diabetic eNOS-knockout mice develop accelerated retinopathy.DICER1 deficit induces Alu RNA toxicity in age-related macular degenerationSTAT3 promotes survival of mutant photoreceptors in inherited photoreceptor degeneration modelsEfficiency and safety of AAV-mediated gene delivery of the human ND4 complex I subunit in the mouse visual systemPseudo-fovea formation after gene therapy for RPE65-LCA.Gene delivery of a viral anti-inflammatory protein to combat ocular inflammationXIAP therapy increases survival of transplanted rod precursors in a degenerating host retina.Recombinant AAV-mediated BEST1 transfer to the retinal pigment epithelium: analysis of serotype-dependent retinal effects.
P50
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P50
description
researcher
@en
wetenschapper
@nl
հետազոտող
@hy
name
William W Hauswirth
@ast
William W Hauswirth
@en
William W Hauswirth
@es
William W Hauswirth
@nl
type
label
William W Hauswirth
@ast
William W Hauswirth
@en
William W Hauswirth
@es
William W Hauswirth
@nl
prefLabel
William W Hauswirth
@ast
William W Hauswirth
@en
William W Hauswirth
@es
William W Hauswirth
@nl
P106
P1153
7005825295
P31
P496
0000-0002-3244-4947