Engineered factor IX variants bypass FVIII and correct hemophilia A phenotype in mice.

@article{Milanov2012EngineeredFI,
  title={Engineered factor IX variants bypass FVIII and correct hemophilia A phenotype in mice.},
  author={Peter Milanov and Lacramioara Ivanciu and Daniela Abriss and Patricia Quade-Lyssy and Wolfgang Miesbach and Sonja Rosa Alesci and Torsten Tonn and Manuel Grez and Erhard Seifried and Joerg Schuettrumpf},
  journal={Blood},
  year={2012},
  volume={119 2},
  pages={
          602-11
        }
}
The complex of the serine protease factor IX (FIX) and its cofactor, factor VIII (FVIII), is crucial for propagation of the intrinsic coagulation cascade. Absence of either factor leads to hemophilia, a disabling disorder marked by excessive hemorrhage after minor trauma. FVIII is the more commonly affected protein, either by X-chromosomal gene mutations or in autoimmune-mediated acquired hemophilia. Whereas substitution of FVIII is the mainstay of hemophilia A therapy, treatment of patients… CONTINUE READING

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