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Minimal modification in the factor VIII B-domain sequence ameliorates the murine hemophilia A phenotype

Recombinant canine B-domain deleted (BDD) factor VIII (FVIII) is predominantly expressed as a single-chain protein and exhibits greater stability after activation compared with human FVIII-BDD. We generated a novel BDD-FVIII variant (FVIII-RH) with an amino acid change at the furin cleavage site wit...

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Main Authors: Siner, Joshua I., Iacobelli, Nicholas P., Sabatino, Denise E., Ivanciu, Lacramiora, Zhou, Shangzhen, Poncz, Mortimer, Camire, Rodney M., Arruda, Valder R.
Formato: Artigo
Idioma:Inglês
Publicado: American Society of Hematology 2013
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Acceso en liña:https://ncbi.nlm.nih.gov/pmc/articles/PMC3663432/
https://ncbi.nlm.nih.gov/pubmed/23372167
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1182/blood-2012-10-464164
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