Vision 1 year after gene therapy for Leber's congenital amaurosis
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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 administrationClinical Trials in Retinal DystrophiesCloning humans? Biological, ethical, and social considerationsRegenerative medicine for the special senses: restoring the inputs.Current prospects and challenges for epilepsy gene therapyGene therapy of inherited retinal degenerations: prospects and challengesAAV-mediated gene therapy in mouse models of recessive retinal degenerationImmunology of AAV-Mediated Gene Transfer in the EyeA comprehensive review of retinal gene therapyGene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 AchromatopsiaMicroRNA-restricted transgene expression in the retinaWhat Is Next for Retinal Gene Therapy?Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trialRetinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.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.Passive immunization against HIV/AIDS by antibody gene transfer.A flow cytometric approach to study the mechanism of gene delivery to cells by gemini-lipid nanoparticles: an implication for cell membrane nanoporationMacular function in macular degenerations: repeatability of microperimetry as a potential outcome measure for ABCA4-associated retinopathy trials.Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.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.GFAP-driven GFP expression in activated mouse Müller glial cells aligning retinal blood vessels following intravitreal injection of AAV2/6 vectorsEvidence for the failure of adeno-associated virus serotype 5 to package a viral genome > or = 8.2 kb.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 therapyGene therapy in ophthalmology.Gene augmentation for adRP mutations in RHO.Viral and nonviral delivery systems for gene deliveryExpression of wild-type Rp1 protein in Rp1 knock-in mice rescues the retinal degeneration phenotypeSelf-complementary AAV-mediated gene therapy restores cone function and prevents cone degeneration in two models of Rpe65 deficiency.Blue cone monochromacy: visual function and efficacy outcome measures for clinical trials.Bestrophin 1 and retinal disease.Vector platforms for gene therapy of inherited retinopathies.Recombinant vectors based on porcine adeno-associated viral serotypes transduce the murine and pig retinaTemporal interactions during paired-electrode stimulation in two retinal prosthesis subjects.Gene therapy rescues cone structure and function in the 3-month-old rd12 mouse: a model for midcourse RPE65 leber congenital amaurosisAltered expression of retinal molecular markers in the canine RPE65 model of Leber congenital amaurosisAssessment of tropism and effectiveness of new primate-derived hybrid recombinant AAV serotypes in the mouse and primate retina
P2860
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P2860
Vision 1 year after gene therapy for Leber's congenital amaurosis
description
2009 nî lūn-bûn
@nan
2009 թուականի Օգոստոսին հրատարակուած գիտական յօդուած
@hyw
2009 թվականի օգոստոսին հրատարակված գիտական հոդված
@hy
2009年の論文
@ja
2009年論文
@yue
2009年論文
@zh-hant
2009年論文
@zh-hk
2009年論文
@zh-mo
2009年論文
@zh-tw
2009年论文
@wuu
name
Vision 1 year after gene therapy for Leber's congenital amaurosis
@ast
Vision 1 year after gene therapy for Leber's congenital amaurosis
@en
Vision 1 year after gene therapy for Leber's congenital amaurosis
@nl
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label
Vision 1 year after gene therapy for Leber's congenital amaurosis
@ast
Vision 1 year after gene therapy for Leber's congenital amaurosis
@en
Vision 1 year after gene therapy for Leber's congenital amaurosis
@nl
prefLabel
Vision 1 year after gene therapy for Leber's congenital amaurosis
@ast
Vision 1 year after gene therapy for Leber's congenital amaurosis
@en
Vision 1 year after gene therapy for Leber's congenital amaurosis
@nl
P2093
P2860
P3181
P356
P1476
Vision 1 year after gene therapy for Leber's congenital amaurosis
@en
P2093
Alejandro J Roman
Alexander Sumaroka
Artur V Cideciyan
Barry J Byrne
Elizabeth A M Windsor
Samuel G Jacobson
Sanford L Boye
Shalesh Kaushal
Sharon B Schwartz
Thomas J Conlon
P2860
P3181
P356
10.1056/NEJMC0903652
P407
P577
2009-08-13T00:00:00Z