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1 an adenoviral vector expressing human blood coagulation factor IX.
2 xicity and promote the delivery of the human coagulation factor IX and alpha-galactosidase genes into
3 n with functional outcome; and two proteins (coagulation factor IX and complement C5) positively asso
5 in patients with haemophilia B (defective in coagulation factor IX) and the Marfan syndrome (defectiv
6 is caused by the absence of functional blood coagulation factor IX, and is an excellent candidate for
7 gene on the X chromosome that encodes blood coagulation factor IX, and is predicted to alter RNA spl
8 o demonstrate a polymorphism of human plasma coagulation factor IX antigen in double antibody solid-p
9 g-term expression (>5 months) of human blood coagulation factor IX at levels that were therapeutic in
11 products for the treatment of hemophilia B (coagulation factor IX deficiency) and one for hemophilia
12 We have used gene targeting to develop a coagulation factor IX-deficient (factor IX-knockout) mou
13 lia B is caused by the absence of functional coagulation factor IX (F.IX) and represents an important
14 sociated viral (AAV) vector expressing blood coagulation factor IX (F.IX) can result in long-term exp
17 g disorder hemophilia B [deficiency in blood coagulation factor IX (F.IX)] by gene replacement therap
19 at antigenic epitopes derived from an ARF in coagulation factor IX (F9) cDNA can induce CTL reactivit
20 n particular, we target a promoterless human coagulation factor IX (F9) gene to the liver-expressed m
21 a B (HB) replacement therapy is evaluated by coagulation factor IX (FIX) activity in plasma, although
22 d efficient induction of immune tolerance to coagulation factor IX (FIX) by direct intramuscular inje
23 emonstrated induction of immune tolerance to coagulation factor IX (FIX) by hepatic adeno-associated
25 associated viral (AAV) vector encoding blood coagulation factor IX (FIX) into skeletal muscle results
26 d mice with combined deficiencies of Plg and coagulation factor IX (fIX) or XI (fXI) to determine the
27 ion and most importantly the activity of the coagulation factor IX (FIX) produced by mature hepatocyt
28 nced the expression and clotting activity of coagulation factor IX (FIX) to nearly 2-fold that of imm
29 ssion and function, we designed an optimized coagulation factor IX (FIX) variant and used multiple me
30 safety of recombinant fusion protein linking coagulation factor IX (FIX) with albumin (rIX-FP) which,
35 onsidering the enzymatic domain of activated coagulation factor IX (FIXa) is homologous to those of t
36 ient to maintain therapeutic levels of human coagulation Factor IX for more than six months in mice u
38 are the result of missense mutations in the coagulation factor IX gene and defective circulating fac
39 cin resistance marker gene (neo) and a human coagulation factor IX genomic construct designed for exp
41 tion of immune tolerance to a secreted human coagulation factor IX (hF.IX) antigen by adeno-associate
43 arried out a systematic study on human blood coagulation factor IX (hFIX) and anti-coagulant protein
46 er-specific promoter-1 encoding either human coagulation factors IX (hFIX) or X (hFX) into Macaca fas
51 hilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent bleeding.
52 closely related fragment, the Gla domain of coagulation factor IX, the structure of which had previo