AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects (Q37146095)

18 August 2017

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title

AAV-mediated gene transfer for the treatment of hemophilia B: problems and prospects (English)

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18 August 2017

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N C Hasbrouck

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18 August 2017

K A High

series ordinal

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18 August 2017

publication date

24 April 2008

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18 August 2017

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18 August 2017

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18 August 2017

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18 August 2017

page(s)

870-875

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Vector integration is nonrandom and clustered and influences the fate of lymphopoiesis in SCID-X1 gene therapy

18 August 2017

cites work

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https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Multilineage hematopoietic reconstitution without clonal selection in ADA-SCID patients treated with stem cell gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo

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https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Haemophilia prophylaxis in young patients--a long-term follow-up

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Hemophilia presenting with intracranial hemorrhage. An approach to the infant with intracranial bleeding and coagulopathy

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Prophylaxis versus episodic treatment to prevent joint disease in boys with severe hemophilia

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Expression of human factor IX in mice after injection of genetically modified myoblasts

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https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Expression of human factor IX in rat capillary endothelial cells: toward somatic gene therapy for hemophilia B

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https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Evidence for gene transfer and expression of factor IX in haemophilia B patients treated with an AAV vector.

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https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Long-term correction of canine hemophilia B by gene transfer of blood coagulation factor IX mediated by adeno-associated viral vector

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

AAV-mediated factor IX gene transfer to skeletal muscle in patients with severe hemophilia B.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Influence of vector dose on factor IX-specific T and B cell responses in muscle-directed gene therapy

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Muscle-directed gene transfer and transient immune suppression result in sustained partial correction of canine hemophilia B caused by a null mutation

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Risk and prevention of anti-factor IX formation in AAV-mediated gene transfer in the context of a large deletion of F9.

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https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Uniform scale-independent gene transfer to striated muscle after transvenular extravasation of vector

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

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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https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

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

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Modulation of tolerance to the transgene product in a nonhuman primate model of AAV-mediated gene transfer to liver.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Evidence of multiyear factor IX expression by AAV-mediated gene transfer to skeletal muscle in an individual with severe hemophilia B.

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Heparin binding directs activation of T cells against adeno-associated virus serotype 2 capsid

1 reference

https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo

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https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Observed incidence of tumorigenesis in long-term rodent studies of rAAV vectors.

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https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

AAV vector integration sites in mouse hepatocellular carcinoma.

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https://api.crossref.org/works/10.1038%2FGT.2008.71

7 January 2021

Identifiers

DOI

10.1038/GT.2008.71

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Dimensions Publication ID

18 August 2017

1034467993

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