(Q45859498)

17 December 2017

title

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

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17 December 2017

main subject

hemophilia

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coagulation factor IX

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17 December 2017

Catherine Manno

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17 December 2017

Erich V de Paula

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Glenn F Pierce

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17 December 2017

Margareth C Ozelo

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17 December 2017

Joseph A Vargas

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17 December 2017

Peter Smith

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17 December 2017

Jürg Sommer

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17 December 2017

Alvin Luk

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17 December 2017

Katherine A High

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17 December 2017

Valder R Arruda

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17 December 2017

publication date

5 July 2006

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17 December 2017

published in

Molecular Therapy

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17 December 2017

volume

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17 December 2017

issue

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17 December 2017

page(s)

452-455

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17 December 2017

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

cites work

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

Posttranslational modifications of recombinant myotube-synthesized human factor IX.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

Direct intramuscular injection with recombinant AAV vectors results in sustained expression in a dog model of hemophilia

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

Safety and efficacy of factor IX gene transfer to skeletal muscle in murine and canine hemophilia B models by adeno-associated viral vector serotype 1.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

Regional intravascular delivery of AAV-2-F.IX to skeletal muscle achieves long-term correction of hemophilia B in a large animal model

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

Haemophilia B: database of point mutations and short additions and deletions--eighth edition

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

Identification of the endothelial cell binding site for factor IX.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

Circulating and binding characteristics of wild-type factor IX and certain Gla domain mutants in vivo

7 January 2021

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https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

Factor IX variants improve gene therapy efficacy for hemophilia B.

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

Quantification of adeno-associated virus particles and empty capsids by optical density measurement

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

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

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FJ.YMTHE.2006.05.004

10.1016/J.YMTHE.2006.05.004

7 January 2021

Identifiers

DOI

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17 December 2017

1 reference

17 December 2017