Tailoring the AAV vector capsid for gene therapy. (Q37340302)

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17 January 2020

review article

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title

Tailoring the AAV vector capsid for gene therapy (English)

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17 January 2020

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2

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17 January 2020

author name string

L H Vandenberghe

series ordinal

1

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17 January 2020

G Gao

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3

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17 January 2020

language of work or name

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4 December 2008

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17 January 2020

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17 January 2020

volume

16

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17 January 2020

page(s)

311-319

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17 January 2020

issue

3

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https://scigraph.springernature.com/pub.10.1038/gt.2008.170

17 January 2020

exact match

0 references

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Long-term pharmacologically regulated expression of erythropoietin in primates following AAV-mediated gene transfer.

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Sustained expression of therapeutic level of factor IX in hemophilia B dogs by AAV-mediated gene therapy in liver

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7 January 2021

Biology of AAV serotype vectors in liver-directed gene transfer to nonhuman primates.

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Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models

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7 January 2021

Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response

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7 January 2021

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

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

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

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7 January 2021

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

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7 January 2021

Identification of PDGFR as a receptor for AAV-5 transduction

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7 January 2021

Sustained AAV-mediated dystrophin expression in a canine model of Duchenne muscular dystrophy with a brief course of immunosuppression

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7 January 2021

Percutaneous transendocardial delivery of self-complementary adeno-associated virus 6 achieves global cardiac gene transfer in canines.

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7 January 2021

Multiyear therapeutic benefit of AAV serotypes 2, 6, and 8 delivering factor VIII to hemophilia A mice and dogs

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7 January 2021

Sustained correction of disease in naive and AAV2-pretreated hemophilia B dogs: AAV2/8-mediated, liver-directed gene therapy

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7 January 2021

Long-term efficacy of adeno-associated virus serotypes 8 and 9 in hemophilia a dogs and mice

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Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

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7 January 2021

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

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Rapid uncoating of vector genomes is the key to efficient liver transduction with pseudotyped adeno-associated virus vectors

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Endosomal processing limits gene transfer to polarized airway epithelia by adeno-associated virus.

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Sustained phenotypic correction of hemophilia B dogs with a factor IX null mutation by liver-directed gene therapy

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7 January 2021

Self-complementary adeno-associated virus vectors containing a novel liver-specific human factor IX expression cassette enable highly efficient transduction of murine and nonhuman primate liver

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7 January 2021

Unrestricted hepatocyte transduction with adeno-associated virus serotype 8 vectors in mice

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7 January 2021

Immune responses to adeno-associated virus vectors

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7 January 2021

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7 January 2021

CD8(+) T-cell responses to adeno-associated virus capsid in humans

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Cross-presentation of adeno-associated virus serotype 2 capsids activates cytotoxic T cells but does not render hepatocytes effective cytolytic targets

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7 January 2021

Pre-existing AAV capsid-specific CD8+ T cells are unable to eliminate AAV-transduced hepatocytes

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7 January 2021

Adeno-associated virus type 2 (AAV2) capsid-specific cytotoxic T lymphocytes eliminate only vector-transduced cells coexpressing the AAV2 capsid in vivo

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7 January 2021