Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.
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Lentiviral gene transfer of RPE65 rescues survival and function of cones in a mouse model of Leber congenital amaurosisRetinitis pigmentosaGene therapy rescues cone function in congenital achromatopsia.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 trialGene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administrationThe human visual cortex responds to gene therapy-mediated recovery of retinal functionSafety and efficacy of gene transfer for Leber's congenital amaurosisHuman RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 yearNovel RDH12 mutations associated with Leber congenital amaurosis and cone-rod dystrophy: biochemical and clinical evaluationsGenetic manipulation for inherited neurodegenerative diseases: myth or reality?Recent advances and future prospects in choroideremiaPromising and delivering gene therapies for vision lossA comprehensive review of retinal gene therapyLentiviral expression of retinal guanylate cyclase-1 (RetGC1) restores vision in an avian model of childhood blindnessCanine CNGA3 Gene Mutations Provide Novel Insights into Human Achromatopsia-Associated Channelopathies and TreatmentGene Augmentation Therapy Restores Retinal Function and Visual Behavior in a Sheep Model of CNGA3 AchromatopsiaComparative analysis of DNA nanoparticles and AAVs for ocular gene deliveryMicroRNA-restricted transgene expression in the retinaA partial structural and functional rescue of a retinitis pigmentosa model with compacted DNA nanoparticlesIdentification of key residues determining isomerohydrolase activity of human RPE65Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trialSafety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindnessMetabolic basis of visual cycle inhibition by retinoid and nonretinoid compounds in the vertebrate retinaGenetic and phenotypic variations of inherited retinal diseases in dogs: the power of within- and across-breed studiesThe proteome of the mouse photoreceptor sensory cilium complexHomozygosity mapping and targeted sanger sequencing reveal genetic defects underlying inherited retinal disease in families from pakistan.The use of surface modified poly(glycerol-co-sebacic acid) in retinal transplantationHuman cone photoreceptor dependence on RPE65 isomeraseGene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years.Successful gene therapy in the RPGRIP1-deficient dog: a large model of cone-rod dystrophy.AAV2 gene therapy readministration in three adults with congenital blindness.Development and use of DNA archives at veterinary teaching hospitals to investigate the genetic basis of disease in dogsGene therapy regenerates protein expression in cone photoreceptors in Rpe65(R91W/R91W) mice.Efficient non-viral ocular gene transfer with compacted DNA nanoparticles.Canine and human visual cortex intact and responsive despite early retinal blindness from RPE65 mutation.Adeno-associated viral vectors engineered for macrolide-adjustable transgene expression in mammalian cells and miceReversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transferProgressive retinal atrophy in the Border Collie: a new XLPRA.A study of candidate genes for day blindness in the standard wire haired dachshundOcular delivery of compacted DNA-nanoparticles does not elicit toxicity in the mouse retina
P2860
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P2860
Long-term restoration of rod and cone vision by single dose rAAV-mediated gene transfer to the retina in a canine model of childhood blindness.
description
2005 nî lūn-bûn
@nan
2005年の論文
@ja
2005年論文
@yue
2005年論文
@zh-hant
2005年論文
@zh-hk
2005年論文
@zh-mo
2005年論文
@zh-tw
2005年论文
@wuu
2005年论文
@zh
2005年论文
@zh-cn
name
Long-term restoration of rod a ...... model of childhood blindness.
@en
type
label
Long-term restoration of rod a ...... model of childhood blindness.
@en
prefLabel
Long-term restoration of rod a ...... model of childhood blindness.
@en
P2093
P2860
P1433
P1476
Long-term restoration of rod a ...... model of childhood blindness.
@en
P2093
Albert M Maguire
Artur V Cideciyan
Gregory M Acland
Gustavo D Aguirre
Jean Bennett
Jeannette Bennicelli
Nadine S Dejneka
Samuel G Jacobson
Susan E Pearce-Kelling
Tomas S Aleman
P2860
P304
P356
10.1016/J.YMTHE.2005.08.008
P577
2005-10-14T00:00:00Z