Article
Catalytic domain modification and viral gene delivery of activated factor VII confers hemostasis at reduced expression levels and vector doses in vivo.
Blood - 14 Apr 2011
Margaritis Paris, Roy Elise, Faella Armida, Downey Harre D, Ivanciu Lacramioara, Pavani Giulia, Zhou Shangzhen, Bunte Ralph M, High Katherine A
Abstract excerpt
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 mFVIIa-DVQ) with modified catalytic domains, based on recombinant human FVIIa (rhFVIIa) variants. Using purified...
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