(Q34497332)

2 August 2017

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title

AAV-mediated gene transfer for hemophilia. (English)

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2 August 2017

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High KA

1

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2 August 2017

language of work or name

English

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publication date

1 December 2001

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Annals of the New York Academy of Sciences

2 August 2017

published in

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2 August 2017

volume

953

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2 August 2017

page(s)

64-74

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Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease

2 August 2017

cites work

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

Posttranslational modifications of recombinant myotube-synthesized human factor IX.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

A factor IX-deficient mouse model for hemophilia B gene therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

Deduced amino acid sequence of mouse blood-coagulation factor IX.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

Transient immunomodulation with anti-CD40 ligand antibody and CTLA4Ig enhances persistence and secondary adenovirus-mediated gene transfer into mouse liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

Gene delivery to skeletal muscle results in sustained expression and systemic delivery of a therapeutic protein

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

Stable gene transfer and expression of human blood coagulation factor IX after intramuscular injection of recombinant adeno-associated virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

The use of adeno-associated virus to circumvent the maturation-dependent viral transduction of muscle fibers

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

Identification of the endothelial cell binding site for factor IX.

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

Adeno-associated virus vectors can be efficiently produced without helper virus

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

Recombinant factor VIII for the treatment of previously untreated patients with hemophilia A. Safety, efficacy, and development of inhibitors. Kogenate Previously Untreated Patient Study Group

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

Gene therapy for the hemophilias

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

Efficient lentiviral transduction of liver requires cell cycling in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1111%2FJ.1749-6632.2001.TB11361.X

10.1111/J.1749-6632.2001.TB11361.X

21 January 2018

Identifiers

DOI

1 reference

2 August 2017

1 reference