Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.
about
Promising and delivering gene therapies for vision lossGene therapy of inherited retinal degenerations: prospects and challengesImmunology of AAV-Mediated Gene Transfer in the EyeBiology and therapy of inherited retinal degenerative disease: insights from mouse modelsUsher syndrome: Hearing loss, retinal degeneration and associated abnormalities.Myosin7a deficiency results in reduced retinal activity which is improved by gene therapy.Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.The potential of adeno-associated viral vectors for gene delivery to muscle tissue.Vector platforms for gene therapy of inherited retinopathies.Targeted exon sequencing in Usher syndrome type INatural 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 the Retinal Degeneration of Usher Syndrome Caused by Mutations in MYO7AImproved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease.Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids.Single residue AAV capsid mutation improves transduction of photoreceptors in the Abca4-/- mouse and bipolar cells in the rd1 mouse and human retina ex vivo.Effective delivery of large genes to the retina by dual AAV vectors.Usher protein functions in hair cells and photoreceptors.Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A.Providing comprehensive genetic-based ophthalmic care.Stem cells for investigation and treatment of inherited retinal disease.Gene therapies for inherited retinal disorders.Copackaging of multiple adeno-associated viral vectors in a single production step.Gene and cell-based therapies for inherited retinal disorders: An update.In vitro and in vivo rescue of aberrant splicing in CEP290-associated LCA by antisense oligonucleotide delivery.Whole USH2A Gene Sequencing Identifies Several New Deep Intronic Mutations.A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequenceIncreased vulnerability of photoreceptors to aberrant splicing highlight the utility of AON-based therapy for CEP290-LCA.USH2A Gene Editing Using the CRISPR SystemRetinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus.Efficient gene delivery to the cone-enriched pig retina by dual AAV vectors.Gene therapy approaches for prevention of retinal degeneration in Usher syndrome.Dual AAV Vectors for Stargardt Disease.Triple Vectors Expand AAV Transfer Capacity in the Retina.Myosins: Domain Organisation, Motor Properties, Physiological Roles and Cellular Functions.Adeno-associated Virus (AAV) Dual Vector Strategies for Gene Therapy Encoding Large Transgenes.Gene therapy and genome surgery in the retina.High-throughput screening identifies kinase inhibitors that increase dual AAV vectors transduction <i>in vitro</i> and in mouse retina.
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P2860
Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.
description
2013 nî lūn-bûn
@nan
2013年の論文
@ja
2013年論文
@yue
2013年論文
@zh-hant
2013年論文
@zh-hk
2013年論文
@zh-mo
2013年論文
@zh-tw
2013年论文
@wuu
2013年论文
@zh
2013年论文
@zh-cn
name
Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.
@ast
Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.
@en
type
label
Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.
@ast
Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.
@en
prefLabel
Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.
@ast
Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.
@en
P2093
P2860
P356
P1433
P1476
Retinal gene therapy with a large MYO7A cDNA using adeno-associated virus.
@en
P2093
D S Williams
W W Hauswirth
P2860
P2888
P304
P356
10.1038/GT.2013.3
P577
2013-01-24T00:00:00Z
P5875
P6179
1021416355