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Engineered factor IX variants bypass FVIII and correct hemophilia A phenotype in mice.
Blood. 2012 Jan 12;119(2):602-11. doi: 10.1182/blood-2011-05-353672. Epub 2011 Oct 26.
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Expression of biologically active human clotting factor IX in Drosophila S2 cells: γ-carboxylation of a human vitamin K-dependent protein by the insect enzyme.
Biotechnol Prog. 2012 Jan-Feb;28(1):45-51. doi: 10.1002/btpr.723. Epub 2011 Oct 19.
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Stable and high-level production of recombinant Factor IX in human hepatic cell line.
Biotechnol Appl Biochem. 2011 Jul-Aug;58(4):243-9. doi: 10.1002/bab.32. Epub 2011 Aug 9.
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Hepatic control elements promote long-term expression of human coagulation factor IX gene in hydrodynamically transfected mice.
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Prolonged half-life and preserved enzymatic properties of factor IX selectively PEGylated on native N-glycans in the activation peptide.
Blood. 2011 Aug 25;118(8):2333-41. doi: 10.1182/blood-2011-02-336172. Epub 2011 Jun 23.
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Enhanced pharmacokinetic properties of a glycoPEGylated recombinant factor IX: a first human dose trial in patients with hemophilia B.
Blood. 2011 Sep 8;118(10):2695-701. doi: 10.1182/blood-2011-02-335596. Epub 2011 May 9.
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Good manufacturing practice production of self-complementary serotype 8 adeno-associated viral vector for a hemophilia B clinical trial.
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A commentary on the differences in pharmacokinetics between recombinant and plasma-derived factor IX and their implications for dosing.
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Long-term safety and efficacy following systemic administration of a self-complementary AAV vector encoding human FIX pseudotyped with serotype 5 and 8 capsid proteins.
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