(Q35140784)

6 August 2017

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title

Gene therapy progress and prospects: gene therapy for the hemophilias (English)

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6 August 2017

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Christopher E Walsh

1

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6 August 2017

publication date

1 June 2003

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6 August 2017

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6 August 2017

volume

10

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6 August 2017

issue

12

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6 August 2017

page(s)

999-1003

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AAV vectors: is clinical success on the horizon?

6 August 2017

cites work

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https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Several log increase in therapeutic transgene delivery by distinct adeno-associated viral serotype vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Sustained and complete phenotype correction of hemophilia B mice following intramuscular injection of AAV1 serotype vectors

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Bone marrow stromal cells as a genetic platform for systemic delivery of therapeutic proteins in vivo: human factor IX model

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Use of blood outgrowth endothelial cells for gene therapy for hemophilia A.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Long-term persistence of human bone marrow stromal cells transduced with factor VIII-retroviral vectors and transient production of therapeutic levels of human factor VIII in nonmyeloablated immunodeficient mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Use of recombinant factor VIIa in hereditary bleeding disorders

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Induction of tolerance to human factor VIII in mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Dangerous liaisons: the role of "danger" signals in the immune response to gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A.

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Sustained high-level expression of human factor IX (hFIX) after liver-targeted delivery of recombinant adeno-associated virus encoding the hFIX gene in rhesus macaques

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Messenger RNA repair and restoration of protein function by spliceosome-mediated RNA trans-splicing

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

Pluripotency of mesenchymal stem cells derived from adult marrow

21 January 2018

1 reference

https://api.crossref.org/works/10.1038%2FSJ.GT.3302024

21 January 2018

Identifiers

DOI

10.1038/SJ.GT.3302024

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6 August 2017

1 reference