Altered bioavailability of platelet-derived factor VIII during thrombocytosis reverses phenotypic efficacy in haemophilic mice

Thrombosis and Haemostasis

Volumn 100, Issue 6, 2008, Pages 1111-1122

  Damon, Andrea L ^a   Scudder, Lesley E ^a   Gnatenko, Dmitri V ^a   Sitaraman, Varsha ^a   Hearing, Patrick ^a   Jesty, Jolyon ^a   Bahou, Wadie F ^a  

^a STONY BROOK UNIVERSITY   (United States)

Author keywords

Animal models; Coagulation factors; Gene targeting

Indexed keywords

AMYLOID PRECURSOR PROTEIN; BLOOD CLOTTING FACTOR; BLOOD CLOTTING FACTOR 10A; BLOOD CLOTTING FACTOR 5A; BLOOD CLOTTING FACTOR 8; RECOMBINANT THROMBOCYTE FACTOR 4; THROMBOCYTE FACTOR 4; THROMBOPOIETIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BIOAVAILABILITY; CONCENTRATION RESPONSE; CONTROLLED STUDY; DIAGNOSTIC PROCEDURE; GENE TARGETING; HEMOPHILIA; HEMOSTATIC TAIL SNIP ASSAY; MEGAKARYOCYTE; MOUSE; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN BLOOD LEVEL; PROTEIN CLEAVAGE; PROTEIN DEGRADATION; PROTEIN DOMAIN; PROTEIN EXPRESSION; PROTEIN FUNCTION; THROMBOCYTE; THROMBOCYTOSIS;

AMYLOID BETA-PROTEIN PRECURSOR; ANIMALS; BLOOD COAGULATION; BLOOD COAGULATION TESTS; BLOOD PLATELETS; CYSTEINE ENDOPEPTIDASES; CYTOPLASMIC GRANULES; DISEASE MODELS, ANIMAL; FACTOR VIII; FACTOR XA; GENE THERAPY; HEMOPHILIA A; HUMANS; MEGAKARYOCYTES; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; MICE, TRANSGENIC; NEOPLASM PROTEINS; PHENOTYPE; PLATELET ACTIVATION; PLATELET COUNT; PLATELET FACTOR 4; RATS; THROMBIN; THROMBOCYTOSIS; THROMBOPOIETIN; TIME FACTORS;

EID: 57349147041     PISSN: 03406245     EISSN: None     Source Type: Journal    
DOI: 10.1160/TH08-04-0242     Document Type: Article

References (60)

1. Hoyer LW. Hemophilia A. N Engl J Med 1994; 330: 38-47.
2. Gitschier J, et al. Characterization of the human factor VIII gene. Nature 1984; 312: 326-330.
3. Toole JJ, et al. Molecular cloning of a cDNA encoding human antihaemophilic factor. Nature 1984; 312: 342-346.
4. Wood WI, et al. Expression of active human factor VIII from recombinant DNA clones. Nature 1984; 312: 330-336.
5. Vehar GA, et al. Structure of human factor VIII. Nature 1984; 312: 337-342.
6. Eaton D, et al. Proteolytic processing of human factor VIII: correlation of specific cleavages by thrombin, factor Xa, and activated protein C with activation and inactivation of factor VIII coagulant activity. Biochem 1986; 83: 5939-5942.
7. Fulcher CA, Zimmerman TS. Characterization of the human factor VIII procoagulant protein with a heterologous precipitating antibody. Proc Nat Acad Sci USA 1982; 79: 1648-1652.
8. Pittman D, Kaufman R. Proteolytic requirements for thrombin activation of anti-hemophilic factor (factor VIII). Proc Nat Acad Sci USA 1988; 85: 2429-2433.
9. Bahou WF, et al. A monoclonal antibody to von Willebrand factor (vWF) inhibits factor VIII binding. Localization of its antigenic determinant to a non adecapeptide at the amino terminus of the mature vWF polypeptide. J Clin Invest 1989; 84: 56-61.
10. Foster PA, et al. A major factor VIII binding domain resides within the amino-terminal 292 amino acid residues of von Willebrand factor. J Biol Chem 1987; 262: 8443-8446.
11. Foster PA, et al. An immunogenic region within residues Vall670-Glu684 of the factor VIII light chain induces antibodies which inhibit binding of factor VIII to von Willebrand factor. J Biol Chem 1988; 263: 5230-5234.
12. Kaufman RJ, et al. Effect of von Willebrand factor coexpression on the synthesis and secretion of factor VIII in Chinese hamster ovary cells. Molec Cell Biol 1989; 9: 1233-1242.
13. Fay PJ, et al. Activated protein C-catalyzed inactivation of human factor VIII and factor VIIIa: identification of cleavage sites and correlation of proteolysis with cofactor activity. J Biol Chem 1991; 266: 20139-20145.
14. Fulcher CA, et al. Proteolytic inactivation of human factor VIII procoagulant protein by activated human protein C and its analogy with factor V. Blood 1984; 63: 486-489.
15. Bihoreau N, et al. Structural and functional characterization of factor VIII-dII, a new recombinant factor VIII lacking most of the B-domain. Biochem J 1991; 277: 23-31.
16. Eaton DL, et al. Construction and characterization of an active factor VIII variant lacking the central onethird of the molecule. Biochem 1986; 25: 8344-8347.
17. Toole JJ, et al. A large region (95 kDa) of human factor VIII is dispensable for in vitro procoagulant activity. Proc Nat Acad Sci USA 1986; 83: 5939-5942.
18. Gilbert GE, Arena AA. Activation of the factor VIIIa-factor IXa enzyme complex of blood coagulation by membranes containing phosphatidyl-L-serine. J Biol Chem 1996; 271: 11120-11125.
19. Butenas S, Mann KG. Blood coagulation. Biochemistry (Mosc) 2002; 67: 3-12.
20. Mathur A, et al. Interaction of factor IXa with factor VIIIa. Effects of protease domain Ca2+ binding site, proteolysis in the autolysis loop, phospholipid, and factor X. J Biol Chem 1997; 272: 23418-23426.
21. van Dieijen G, et al. The role of phospholipid and factor VIIIa in the activation of bovine factor X. J Biol Chem 1981; 256: 3433-3442.
22. Cawthern KM, et al. Blood coagulation in hemophilia A and hemophilia C. Blood 1998; 91: 4581-4592.
23. Butenas S, et al. Platelets and phospholipids in tissue factor-initiated thrombin generation. Thromb Haemost 2001; 86: 660-667.
24. Yarovoi HV, et al. Factor VIII ectopically expressed in platelets: efficacy in hemophilia A treatment. Blood 2003; 102: 4006-4013.
25. Shi Q, et al. Factor VIII ectopically targeted to platelets is therapeutic in hemophilia A with high-titer inhibitory antibodies. J Clin Invest 2006; 116: 1974-1982.
26. Wilcox DA, et al. Induction of megakaryocytes to synthesize and store a releasable pool of human factor VIII. J Thromb Haemost 2003; 1: 2477-2489.
27. Jesty J, Bluestein D. Acetylated prothrombin as a substrate in the measurement of the procoagulant activity of platelets: elimination of the feedback activation of platelets by thrombin. Analyt Biochem 1999; 272: 64-70.
28. Gould WR, et al. Unique in vivo modifications of coagulation factor V produce a physically and functionally distinct platelet-derived cofactor: characterization of purified platelet-derived factor V/Va. J Biol Chem 2004; 279: 2383-2393.
29. Bahou WF, et al. A shear-restricted pathway of platelet procoagulant activity is regulated by IQGAP1. J Biol Chem 2004; 279: 22571-22577.
30. Camire RM, et al. Secretable human platelet-derived factor V originates from the plasma pool. Blood 1998; 92: 3035-3041.
31. Nesheim ME, et al. The contribution of bovine Factor V and Factor Va to the activity of prothrombinase. J Biol Chem 1979; 254: 10952-10962.
32. Kane WH, et al. Activation of coagulation factor V by a platelet protease. J Clin Invest 1982; 70: 1092-1100.
33. Tracy PB, Mann KG. Abnormal formation of the prothrombinase complex: factor V deficiency and related disorders. Hum Pathol 1987; 18: 162-169.
34. Miletich JP, et al. Patients with congenital factor V deficiency have decreased factor Xa binding sites on their platelets. J Clin Invest 1978; 62: 824-831.
35. Borchgrevink CF, Owren PA. The hemostatic effect of normal platelets in hemophilia and factor V deficiency. The importance of clotting factors adsorbed on platelets for normal hemostasis. Acta Med Scand 1961; 170: 375-383.
36. Ravid K, et al. Selective targeting of gene products with the megakaryocyte platelet factor 4 promoter. Proc Nat Acad Sci USA 1991; 88: 1521-1525.
37. Gnatenko D, et al. Human factor VIII can be packaged and functionally expressed in an adeno-associated virus background: applicability to hemophilia A gene therapy. Br J Haematol 1999; 104: 27-36.
38. Xu F, et al. Protease nexin-2/amyloid beta-protein precursor limits cerebral thrombosis. Proc Nat Acad Sci USA 2005; 102: 18135-18140.
39. Bi L, et al. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nature Genetics 1995; 10: 119-121.
40. Schmidt VA, et al. Development of hepatocellular carcinoma in Iqgap2-deficient mice is IQGAP1 dependent. Mol Cell Biol 2008; 28: 1489-1502.
41. Heid CA, et al. Real time quantitative PCR. Genome Res 1996; 6: 986-994.
42. Gnatenko DV, et al. Transcript profiling of human platelets using microarray and serial analysis of gene expression. Blood 2003; 101: 2285-2293.
43. Jesty J. The kinetics of inhibition of thrombin by antithrombin in the presence of components of the hemostatic system. Blood 1985; 66: 1189-1195.
44. Morrison SA, Jesty J. Tissue factor-dependent activation of tritium-labeled factor IX and factor X in human plasma. Blood 1984; 63: 1338-1347.
45. Jesty J, Godfrey HP. Parlin, a general microcomputer program for parallel-line analysis of bioassays. Am J Clin Pathol 1986; 85: 485-489.
46. Pittman DD, et al. Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII. Blood 1993; 81: 2925-2935.
47. Schmidt VA, et al. IQGAP2 functions as a GTP-dependent effector protein in thrombin-induced platelet cytoskeletal reorganization. Blood 2003; 101: 3021-3028.
48. Kufrin D, et al. Antithrombotic thrombocytes: ectopic expression of urokinase-type plasminogen activator in platelets. Blood 2003; 102: 926-933.
49. Yang TL, et al. Rescue of fatal neonatal hemorrhage in factor V deficient mice by low level transgene expression. Thromb Haemost 2000; 83: 70-77.
50. Bucki R, et al. Calcium induces phospholipid redistribution and microvesicle release in human erythrocyte membranes by independent pathways. Biochemistry 1998; 37: 15383-15391.
51. Gaffet P, et al. Transverse redistribution of phospholipids during human platelet activation: evidence for a vectorial outflux specific to aminophospholipids. Biochemistry 1995; 34: 6762-6769.
52. Gnatenko DV, et al. Expression of therapeutic levels of factor VIII in hemophilia A mice using a novel adeno/adeno-associated hybrid virus. Thromb Haemost 2004; 92: 317-327.
53. Tracy PB, et al. Radioimmunoassay of factor V in human plasma and platelets. Blood 1982; 60: 59-63.
54. Monkovic DD, Tracy PB. Functional characterization of human platelet-released factor V and its activation by factor Xa and thrombin. J Biol Chem 1990; 265: 17132-17140.
55. Denis MM, et al. Escaping the nuclear confines: signal-dependent pre-mRNA splicing in anucleate platelets. Cell 2005; 122: 379-391.
56. Sun H, et al. The murine platelet and plasma factor V pools are biosynthetically distinct and sufficient for minimal hemostasis. Blood 2003; 102: 2856-2861.
57. Kahr WH, et al. Platelets from patients with the Quebec platelet disorder contain and secrete abnormal amounts of urokinase-type plasminogen activator. Blood 2001; 98: 257-265.
58. Pipe SW, et al. Differential interaction of coagulation factor VIII and factor V with protein chaperones calnexin and calreticulin. J Biol Chem 1998; 273: 8537-8544.
59. Nguyen HG, et al. Conditional overexpression of transgenes in megakaryocytes and platelets in vivo. Blood 2005; 106: 1559-1564.
60. Kaluzhny Y, et al. A selective effect of Mpl ligand on mRNA stabilization during megakaryocyte differentiation. FEBS letters 2002; 527: 279-283.