Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer
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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 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 amaurosisA comprehensive review of retinal gene therapyMicroRNA-restricted transgene expression in the retinaIdentification 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 blindnessRetinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.Homozygosity mapping and targeted sanger sequencing reveal genetic defects underlying inherited retinal disease in families from pakistan.Regulation of retinal function but nonrescue of vision in RPE65-deficient dogs treated with doxycycline-regulatable AAV vectorsAAV2 gene therapy readministration in three adults with congenital blindness.RPE65: role in the visual cycle, human retinal disease, and gene therapy.Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice.Gene therapy targeting glaucoma: where are we?Using the NAFX to measure the effectiveness over time of gene therapy in canine LCAAn evolved adeno-associated viral variant enhances gene delivery and gene targeting in neural stem cellsEfficacious and safe tissue-selective controlled gene therapy approaches for the cornea.Leber congenital amaurosis due to RPE65 mutations and its treatment with gene therapySafety of AAV factor IX peripheral transvenular gene delivery to muscle in hemophilia B dogs.My career path for developing gene therapy for blinding diseases: the importance of mentors, collaborators, and opportunities.Transduction of the inner mouse retina using AAVrh8 and AAVrh10 via intravitreal injectionMechanisms of blindness: animal models provide insight into distinct CRX-associated retinopathiesFranklin H. Epstein Lecture. Both ends of the leash--the human links to good dogs with bad genes.Differential proteomics and functional research following gene therapy in a mouse model of Leber congenital amaurosisQLT091001, a 9-cis-retinal analog, is well-tolerated by retinas of mice with impaired visual cycles.Effects of acetazolamide on infantile nystagmus syndrome waveforms: comparisons to contact lenses and convergence in a well-studied subjectTherapeutic challenges to retinitis pigmentosa: from neuroprotection to gene therapy.Gene therapy for choroideremia using an adeno-associated viral (AAV) vector.Reproducible high yields of recombinant adeno-associated virus produced using invertebrate cells in 0.02- to 200-liter cultures.Gene therapy using stem cellsHistopathology and functional correlations in a patient with a mutation in RPE65, the gene for retinol isomerase.Gene therapy using self-complementary Y733F capsid mutant AAV2/8 restores vision in a model of early onset Leber congenital amaurosisWhole genome sequencing in cats, identifies new models for blindness in AIPL1 and somite segmentation in HES7.Halting progressive neurodegeneration in advanced retinitis pigmentosaProspectives for gene therapy of retinal degenerationsRetinal Gene Therapy: Current Progress and Future Prospects.Non-viral therapeutic approaches to ocular diseases: An overview and future directionsProgress and challenges in viral vector manufacturing
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P2860
Reversal of blindness in animal models of leber congenital amaurosis using optimized AAV2-mediated gene transfer
description
2008 nî lūn-bûn
@nan
2008 թուականի Յունուարին հրատարակուած գիտական յօդուած
@hyw
2008 թվականի հունվարին 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
Reversal of blindness in anima ...... ed AAV2-mediated gene transfer
@ast
Reversal of blindness in anima ...... ed AAV2-mediated gene transfer
@en
Reversal of blindness in anima ...... d AAV2-mediated gene transfer.
@nl
type
label
Reversal of blindness in anima ...... ed AAV2-mediated gene transfer
@ast
Reversal of blindness in anima ...... ed AAV2-mediated gene transfer
@en
Reversal of blindness in anima ...... d AAV2-mediated gene transfer.
@nl
prefLabel
Reversal of blindness in anima ...... ed AAV2-mediated gene transfer
@ast
Reversal of blindness in anima ...... ed AAV2-mediated gene transfer
@en
Reversal of blindness in anima ...... d AAV2-mediated gene transfer.
@nl
P2093
P2860
P356
P1433
P1476
Reversal of blindness in anima ...... ed AAV2-mediated gene transfer
@en
P2093
Albert M Maguire
Andras Komaromy
Bernd Hauck
Caroline Zeiss
Daniel Chung
Daniel Hui
Federico Mingozzi
Gregory M Acland
Jean Bennett
P2860
P304
P356
10.1038/SJ.MT.6300389
P577
2008-01-22T00:00:00Z