Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations
about
Smelling the roses and seeing the light: gene therapy for ciliopathiesAAV-mediated gene therapy in mouse models of recessive retinal degenerationImmunology of AAV-Mediated Gene Transfer in the EyeBiology and therapy of inherited retinal degenerative disease: insights from mouse modelsRestoration of vision in the pde6β-deficient dog, a large animal model of rod-cone dystrophyDeploying RNA and DNA with Functionalized Carbon NanotubesWhirlin replacement restores the formation of the USH2 protein complex in whirlin knockout photoreceptorsThe human rhodopsin kinase promoter in an AAV5 vector confers rod- and cone-specific expression in the primate retina.Successful gene therapy in the RPGRIP1-deficient dog: a large model of cone-rod dystrophy.Gene therapy restores vision in rd1 mice after removal of a confounding mutation in Gpr179Functional and behavioral restoration of vision by gene therapy in the guanylate cyclase-1 (GC1) knockout mouse.AAV's anatomy: roadmap for optimizing vectors for translational successEarly alteration of retinal neurons in Aipl1-/- animals.The Leber congenital amaurosis protein, AIPL1, is needed for the viability and functioning of cone photoreceptor cellsRod Outer Segment Development Influences AAV-Mediated Photoreceptor Transduction After Subretinal Injection.Replacement gene therapy with a human RPGRIP1 sequence slows photoreceptor degeneration in a murine model of Leber congenital amaurosis.Leber congenital amaurosis associated with AIPL1: challenges in ascribing disease causation, clinical findings, and implications for gene therapy.Scavenger receptors and their potential as therapeutic targets in the treatment of cardiovascular diseaseLong-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.A single intravenous AAV9 injection mediates bilateral gene transfer to the adult mouse retina.AAV-mediated photoreceptor transduction of the pig cone-enriched retina.Gene therapy for ocular diseasesViral-mediated vision rescue of a novel AIPL1 cone-rod dystrophy model.Republished review: Gene therapy for ocular diseases.Long-term preservation of cone photoreceptors and restoration of cone function by gene therapy in the guanylate cyclase-1 knockout (GC1KO) mouse.Residual electroretinograms in young Leber congenital amaurosis patients with mutations of AIPL1Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosisC1q enhances cone photoreceptor survival in a mouse model of autosomal recessive retinitis pigmentosa.A long-term efficacy study of gene replacement therapy for RPGR-associated retinal degenerationEvidence for baseline retinal pigment epithelium pathology in the Trp1-Cre mouse.Effects of Subretinal Gene Transfer at Different Time Points in a Mouse Model of Retinal Degeneration.Gene therapy of inherited retinopathies: a long and successful road from viral vectors to patients.Long-term rescue of cone photoreceptor degeneration in retinitis pigmentosa 2 (RP2)-knockout mice by gene replacement therapy.Photoreceptor sensory cilia and ciliopathies: focus on CEP290, RPGR and their interacting proteins.AAV-mediated gene therapy in the guanylate cyclase (RetGC1/RetGC2) double knockout mouse model of Leber congenital amaurosis.Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa.Zinc-finger nuclease-mediated gene correction using single AAV vector transduction and enhancement by Food and Drug Administration-approved drugs.Advances in Gene Therapy for Diseases of the Eye.Combined rod and cone transduction by adeno-associated virus 2/8.
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P2860
Gene therapy with a promoter targeting both rods and cones rescues retinal degeneration caused by AIPL1 mutations
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
Gene therapy with a promoter t ...... tion caused by AIPL1 mutations
@ast
Gene therapy with a promoter t ...... tion caused by AIPL1 mutations
@en
Gene therapy with a promoter t ...... ion caused by AIPL1 mutations.
@nl
type
label
Gene therapy with a promoter t ...... tion caused by AIPL1 mutations
@ast
Gene therapy with a promoter t ...... tion caused by AIPL1 mutations
@en
Gene therapy with a promoter t ...... ion caused by AIPL1 mutations.
@nl
prefLabel
Gene therapy with a promoter t ...... tion caused by AIPL1 mutations
@ast
Gene therapy with a promoter t ...... tion caused by AIPL1 mutations
@en
Gene therapy with a promoter t ...... ion caused by AIPL1 mutations.
@nl
P2093
P2860
P356
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Gene therapy with a promoter t ...... tion caused by AIPL1 mutations
@en
P2093
M A Sandberg
O V Bulgakov
P2860
P2888
P304
P356
10.1038/GT.2009.104
P577
2009-08-27T00:00:00Z