Catalytic domain modification and viral gene delivery of activated factor VII confers hemostasis at reduced expression levels and vector doses in vivo

Catalytic domain variants of activated factor VII (FVIIa) with enhanced hemostatic properties are highly attractive for the treatment of bleeding disorders via gene-based therapy. To explore this in a hemophilic mouse model, we characterized 2 variants of murine activated FVII (mFVIIa-VEAY and mFVII...

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Detalhes bibliográficos
Main Authors: Margaritis, Paris, Roy, Elise, Faella, Armida, Downey, Harre D., Ivanciu, Lacramioara, Pavani, Giulia, Zhou, Shangzhen, Bunte, Ralph M., High, Katherine A.
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Hematology 2011
Assuntos:
Gene Therapy
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3087527/
https://ncbi.nlm.nih.gov/pubmed/21325603
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2010-09-309732
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