(Q37944999)

25 August 2017

title

Novel adeno-associated viral vectors for retinal gene therapy (English)

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25 August 2017

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L H Vandenberghe

1

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25 August 2017

A Auricchio

series ordinal

2

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25 August 2017

publication date

13 October 2011

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25 August 2017

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25 August 2017

volume

19

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25 August 2017

issue

2

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25 August 2017

page(s)

162-168

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Use of adeno-associated virus as a mammalian DNA cloning vector: transduction of neomycin resistance into mammalian tissue culture cells

25 August 2017

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Exchange of surface proteins impacts on viral vector cellular specificity and transduction characteristics: the retina as a model

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Novel caprine adeno-associated virus (AAV) capsid (AAV-Go.1) is closely related to the primate AAV-5 and has unique tropism and neutralization properties

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21 January 2018

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The atomic structure of adeno-associated virus (AAV-2), a vector for human gene therapy.

21 January 2018

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Next generation of adeno-associated virus 2 vectors: point mutations in tyrosines lead to high-efficiency transduction at lower doses

21 January 2018

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High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors

21 January 2018

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Novel properties of tyrosine-mutant AAV2 vectors in the mouse retina

21 January 2018

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Long-term retinal function and structure rescue using capsid mutant AAV8 vector in the rd10 mouse, a model of recessive retinitis pigmentosa.

21 January 2018

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Artificial evolution with adeno-associated viral libraries

21 January 2018

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A novel adeno-associated viral variant for efficient and selective intravitreal transduction of rat Müller cells

21 January 2018

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Inner limiting membrane barriers to AAV-mediated retinal transduction from the vitreous

21 January 2018

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Adeno-associated virus gene transfer to mouse retina

21 January 2018

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Dosage thresholds for AAV2 and AAV8 photoreceptor gene therapy in monkey

21 January 2018

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Effect of gene therapy on visual function in Leber's congenital amaurosis

21 January 2018

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Inhibition of choroidal neovascularization in a nonhuman primate model by intravitreal administration of an AAV2 vector expressing a novel anti-VEGF molecule

21 January 2018

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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 trial

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2011.151

Safety and efficacy of gene transfer for Leber's congenital amaurosis

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2011.151

Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial

21 January 2018

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Gene therapy for red-green colour blindness in adult primates

21 January 2018

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Gene therapy rescues cone function in congenital achromatopsia

21 January 2018

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rAAV2/5 gene-targeting to rods:dose-dependent efficiency and complications associated with different promoters

21 January 2018

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Recombinant adeno-associated virus serotype 4 mediates unique and exclusive long-term transduction of retinal pigmented epithelium in rat, dog, and nonhuman primate after subretinal delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Subretinal delivery of adeno-associated virus serotype 2 results in minimal immune responses that allow repeat vector administration in immunocompetent mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Gene therapy in the second eye of RPE65-deficient dogs improves retinal function

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Safety and efficacy of subretinal readministration of a viral vector in large animals to treat congenital blindness

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2011.151

Stable rAAV-mediated transduction of rod and cone photoreceptors in the canine retina

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2011.151

Stable transgene expression in rod photoreceptors after recombinant adeno-associated virus-mediated gene transfer to monkey retina

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2011.151

Prevalence of mutations causing retinitis pigmentosa and other inherited retinopathies

21 January 2018

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https://api.crossref.org/works/10.1038%2FGT.2011.151

Gene transfer into the mouse retina mediated by an adeno-associated viral vector

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Restoration of photoreceptor ultrastructure and function in retinal degeneration slow mice by gene therapy.

21 January 2018

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Gene replacement therapy rescues photoreceptor degeneration in a murine model of Leber congenital amaurosis lacking RPGRIP.

21 January 2018

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Retinoschisin gene therapy and natural history in the Rs1h-KO mouse: long-term rescue from retinal degeneration

21 January 2018

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Gene therapy for retinitis pigmentosa and Leber congenital amaurosis caused by defects in AIPL1: effective rescue of mouse models of partial and complete Aipl1 deficiency using AAV2/2 and AAV2/8 vectors

21 January 2018

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Cross-packaging of a single adeno-associated virus (AAV) type 2 vector genome into multiple AAV serotypes enables transduction with broad specificity

21 January 2018

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Virus-mediated transduction of murine retina with adeno-associated virus: effects of viral capsid and genome size.

21 January 2018

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Adeno-associated virus type 5: transduction efficiency and cell-type specificity in the primate retina.

21 January 2018

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Prolonged recovery of retinal structure/function after gene therapy in an Rs1h-deficient mouse model of x-linked juvenile retinoschisis

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Restoration of cone vision in a mouse model of achromatopsia

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

AAV-mediated gene therapy for retinal degeneration in the rd10 mouse containing a recessive PDEbeta mutation

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Retinal degeneration mutants in the mouse

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Assessment of ocular transduction using single-stranded and self-complementary recombinant adeno-associated virus serotype 2/8.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

AAV-mediated photoreceptor transduction of the pig cone-enriched retina

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Subretinal delivery of recombinant AAV serotype 8 vector in dogs results in gene transfer to neurons in the brain

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Long-term and age-dependent restoration of visual function in a mouse model of CNGB3-associated achromatopsia following gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Serotype-dependent packaging of large genes in adeno-associated viral vectors results in effective gene delivery in mice.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Little vector, big gene transduction: fragmented genome reassembly of adeno-associated virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

AAV-Mediated gene transfer slows photoreceptor loss in the RCS rat model of retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Preclinical studies on specific gene therapy for recessive retinal degenerative diseases

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Functional and structural recovery of the retina after gene therapy in the RPE65 null mutation dog.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Restoration of vision in RPE65-deficient Briard dogs using an AAV serotype 4 vector that specifically targets the retinal pigmented epithelium

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Cellular tropism and transduction properties of seven adeno-associated viral vector serotypes in adult retina after intravitreal injection

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Adeno-associated virus type 6 (AAV6) vectors mediate efficient transduction of airway epithelial cells in mouse lungs compared to that of AAV2 vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

GFAP-driven GFP expression in activated mouse Müller glial cells aligning retinal blood vessels following intravitreal injection of AAV2/6 vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Müller cells in the healthy and diseased retina

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Transduction of the inner mouse retina using AAVrh8 and AAVrh10 via intravitreal injection

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Molecular evolution of adeno-associated virus for enhanced glial gene delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Tailoring the AAV vector capsid for gene therapy.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Gene therapy in ophthalmology: validation on cultured retinal cells and explants from postmortem human eyes

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Ectopic expression of a microbial-type rhodopsin restores visual responses in mice with photoreceptor degeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Light-activated channels targeted to ON bipolar cells restore visual function in retinal degeneration

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Genetic reactivation of cone photoreceptors restores visual responses in retinitis pigmentosa

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Vascular bed-targeted in vivo gene delivery using tropism-modified adeno-associated viruses

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Molecular signatures of disease brain endothelia provide new sites for CNS-directed enzyme therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

Exploiting and antagonizing microRNA regulation for therapeutic and experimental applications

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

AAV-mediated gene replacement, either alone or in combination with physical and pharmacological agents, results in partial and transient protection from photoreceptor degeneration associated with betaPDE deficiency

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

inferred from DOI database lookup

7 January 2021

based on heuristic

Long-term preservation of cones and improvement in visual function following gene therapy in a mouse model of leber congenital amaurosis caused by guanylate cyclase-1 deficiency.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

inferred from DOI database lookup

7 January 2021

based on heuristic

Membrane-associated heparan sulfate proteoglycan is a receptor for adeno-associated virus type 2 virions

1 reference

https://api.crossref.org/works/10.1038%2FGT.2011.151

inferred from DOI database lookup

7 January 2021

based on heuristic

Identifiers

DOI

10.1038/GT.2011.151

1 reference

Dimensions Publication ID

25 August 2017

1045543244

0 references

1 reference