Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy
about
Gene therapy of inherited retinal degenerations: prospects and challengesImmunology of AAV-Mediated Gene Transfer in the EyeA comprehensive review of retinal gene therapyAdvancing therapeutic strategies for inherited retinal degeneration: recommendations from the Monaciano SymposiumDevelopment of gene and stem cell therapy for ocular neurodegenerationBiology and therapy of inherited retinal degenerative disease: insights from mouse modelsRetinal degeneration progression changes lentiviral vector cell targeting in the retinaQuantitative Fundus Autofluorescence and Optical Coherence Tomography in PRPH2/RDS- and ABCA4-Associated Disease Exhibiting Phenotypic Overlap.Phospholipid meets all-trans-retinal: the making of RPE bisretinoidsCLINICAL PROGRESS IN INHERITED RETINAL DEGENERATIONS: GENE THERAPY CLINICAL TRIALS AND ADVANCES IN GENETIC SEQUENCING.Effects of organic solvents on two retinal pigment epithelial lipofuscin fluorophores, A2E and all-trans-retinal dimerEnzymatic degradation of A2E, a retinal pigment epithelial lipofuscin bisretinoid.The retinal pigment epithelium in health and diseasePhotoreceptor cell degeneration in Abcr (-/-) miceDefective lipid transport and biosynthesis in recessive and dominant Stargardt macular degenerationGenetic basis of inherited macular dystrophies and implications for stem cell therapy.Successful arrest of photoreceptor and vision loss expands the therapeutic window of retinal gene therapy to later stages of disease.Vector platforms for gene therapy of inherited retinopathies.Analysis of the ABCA4 genomic locus in Stargardt disease.Deuterium enrichment of vitamin A at the C20 position slows the formation of detrimental vitamin A dimers in wild-type rodentsQuantitative fundus autofluorescence in mice: correlation with HPLC quantitation of RPE lipofuscin and measurement of retina outer nuclear layer thicknessGene therapeutic approach using mutation-adapted U1 snRNA to correct a RPGR splice defect in patient-derived cells.Light filtering in a retinal pigment epithelial cell culture model.Clinical characteristics and current therapies for inherited retinal degenerationsNatural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy.EIAV-based retinal gene therapy in the shaker1 mouse model for usher syndrome type 1B: development of UshStat.Gene therapy for ocular diseasesRepublished review: Gene therapy for ocular diseases.Analysis of the ABCA4 gene by next-generation sequencing.Membrane receptors and transporters involved in the function and transport of vitamin A and its derivatives.C1q enhances cone photoreceptor survival in a mouse model of autosomal recessive retinitis pigmentosa.Functional and anatomic consequences of subretinal dosing in the cynomolgus macaqueGene Therapy of ABCA4-Associated Diseases.Gene Therapy for the Retinal Degeneration of Usher Syndrome Caused by Mutations in MYO7AThe bisretinoids of retinal pigment epitheliumX-linked juvenile retinoschisis: clinical diagnosis, genetic analysis, and molecular mechanisms.Predicting Progression of ABCA4-Associated Retinal Degenerations Based on Longitudinal Measurements of the Leading Disease Front.Prospectives for gene therapy of retinal degenerationsGene therapy of inherited retinopathies: a long and successful road from viral vectors to patients.DNA nanoparticle-mediated ABCA4 delivery rescues Stargardt dystrophy in mice.
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Correction of the disease phenotype in the mouse model of Stargardt disease by lentiviral gene therapy
description
2008 nî lūn-bûn
@nan
2008 թուականի Հոկտեմբերին հրատարակուած գիտական յօդուած
@hyw
2008 թվականի հոտեմբերին հրատարակված գիտական հոդված
@hy
2008年の論文
@ja
2008年論文
@yue
2008年論文
@zh-hant
2008年論文
@zh-hk
2008年論文
@zh-mo
2008年論文
@zh-tw
2008年论文
@wuu
name
Correction of the disease phen ...... ase by lentiviral gene therapy
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Correction of the disease phen ...... ase by lentiviral gene therapy
@en
Correction of the disease phen ...... ase by lentiviral gene therapy
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Correction of the disease phen ...... ase by lentiviral gene therapy
@ast
Correction of the disease phen ...... ase by lentiviral gene therapy
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Correction of the disease phen ...... ase by lentiviral gene therapy
@nl
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Correction of the disease phen ...... ase by lentiviral gene therapy
@ast
Correction of the disease phen ...... ase by lentiviral gene therapy
@en
Correction of the disease phen ...... ase by lentiviral gene therapy
@nl
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Correction of the disease phen ...... ase by lentiviral gene therapy
@en
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J R Sparrow
J Zernant-Rajang
R Allikmets
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10.1038/GT.2008.78
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2008-10-01T00:00:00Z
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1006240059