Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A. - Test2 - elhamkhani - TriplyDB

Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A.

Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A.

http://www.wikidata.org/entity/Q37710671

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Adeno-associated Virus as a Mammalian DNA Vector Promising and delivering gene therapies for vision loss Gene therapy of inherited retinal degenerations: prospects and challenges Usher syndrome: Hearing loss, retinal degeneration and associated abnormalities.Gene therapy: charting a future course--summary of a National Institutes of Health Workshop, April 12, 2013.Vector platforms for gene therapy of inherited retinopathies.Gene Therapy for the Retinal Degeneration of Usher Syndrome Caused by Mutations in MYO7A Improved dual AAV vectors with reduced expression of truncated proteins are safe and effective in the retina of a mouse model of Stargardt disease.Nanoparticle-motivated gene delivery for ophthalmic application Copackaged AAV9 Vectors Promote Simultaneous Immune Tolerance and Phenotypic Correction of Pompe Disease.Expressing Transgenes That Exceed the Packaging Capacity of Adeno-Associated Virus Capsids.Correction of Monogenic and Common Retinal Disorders with Gene Therapy.Copackaging of multiple adeno-associated viral vectors in a single production step.Gene and cell-based therapies for inherited retinal disorders: An update.Hereditary Retinal Dystrophy.Emerging Gene Therapies for Genetic Hearing Loss.A comparison of AAV strategies distinguishes overlapping vectors for efficient systemic delivery of the 6.2 kb Dysferlin coding sequence Evaluating Efficiencies of Dual AAV Approaches for Retinal Targeting.Retinal gene therapy using adeno-associated viral vectors: multiple applications for a small virus.Dual AAV Vectors for Stargardt Disease.Triple Vectors Expand AAV Transfer Capacity in the Retina.Adeno-associated Virus (AAV) Dual Vector Strategies for Gene Therapy Encoding Large Transgenes.Gene therapy and genome surgery in the retina.Cochlear Gene Therapy for Sensorineural Hearing Loss: Current Status and Major Remaining Hurdles for Translational Success.

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Dual adeno-associated virus vectors result in efficient in vitro and in vivo expression of an oversized gene, MYO7A.

http://www.wikidata.org/entity/Q37710671

description

article científic

@ca

article scientifique

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articolo scientifico

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artigo científico

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bilimsel makale

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scientific article published on April 2014

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vedecký článok

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vetenskaplig artikel

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videnskabelig artikel

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vědecký článek

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name

Dual adeno-associated virus ve ...... n of an oversized gene, MYO7A.

@en

Dual adeno-associated virus ve ...... n of an oversized gene, MYO7A.

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type

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Dual adeno-associated virus ve ...... n of an oversized gene, MYO7A.

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Dual adeno-associated virus ve ...... n of an oversized gene, MYO7A.

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prefLabel

Dual adeno-associated virus ve ...... n of an oversized gene, MYO7A.

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Dual adeno-associated virus ve ...... n of an oversized gene, MYO7A.

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Human gene therapy methods

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Dual adeno-associated virus ve ...... on of an oversized gene, MYO7A

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Frank M Dyka

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Sanford L Boye

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Shannon E Boye

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Vince A Chiodo

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P2860

Defective myosin VIIA gene responsible for Usher syndrome type 1B

Circular intermediates of recombinant adeno-associated virus have defined structural characteristics responsible for long-term episomal persistence in muscle tissue.

Expression in cochlea and retina of myosin VIIa, the gene product defective in Usher syndrome type 1B

Human myosin VIIA responsible for the Usher 1B syndrome: a predicted membrane-associated motor protein expressed in developing sensory epithelia

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 trial

Gene therapy for Leber's congenital amaurosis is safe and effective through 1.5 years after vector administration

Human RPE65 gene therapy for Leber congenital amaurosis: persistence of early visual improvements and safety at 1 year

Usher syndrome: animal models, retinal function of Usher proteins, and prospects for gene therapy

The many different cellular functions of MYO7A in the retina

A comprehensive review of retinal gene therapy

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166-177

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scholarly article

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10.1089/HGTB.2013.212

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2

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25

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William W Hauswirth

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2014-04-01T00:00:00Z

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260379954

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24568220

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vector-borne disease

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3991978

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