Restoration of plasma von willebrand factor deficiency is sufficient to correct thrombus formation after gene therapy for severe von willebrand disease

Arteriosclerosis, Thrombosis, and Vascular Biology

Volumn 28, Issue 9, 2008, Pages 1621-1626

  De Meyer, Simon F ^a   Vandeputte, Nele ^a   Pareyn, Inge ^a   Petrus, Inge ^a   Lenting, Peter J ^b   Chuah, Marinee K L ^a   Vandendriessche, Thierry ^a   Deckmyn, Hans ^a   Vanhoorelbeke, Karen ^a  

^a UNIVERSITY OF LEUVEN   (Belgium)

^b UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

Author keywords

Gene therapy; Hemostasis; Liver; Von Willebrand disease; Von Willebrand factor

Indexed keywords

ALPHA 1 ANTITRYPSIN; BLOOD CLOTTING FACTOR 8; FERRIC CHLORIDE; HERPESVIRUS VECTOR; VON WILLEBRAND FACTOR; FERRIC ION; SERPINA1 PROTEIN, HUMAN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BLEEDING TIME; BLOOD LEVEL; CONTROLLED STUDY; CYTOMEGALOVIRUS; DISEASE MODEL; DISEASE SEVERITY; DRUG TARGETING; GENE EXPRESSION; GENE TRANSFER; HYDRODYNAMICS; INJECTION; LIVER; LIVER CELL; MOLECULAR WEIGHT; MOUSE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; PRIORITY JOURNAL; PROMOTER REGION; PROTEIN BLOOD LEVEL; PROTEIN EXPRESSION; THROMBOCYTE ADHESION; THROMBOCYTE AGGREGATION; THROMBOGENESIS; THROMBOSIS; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VON WILLEBRAND DISEASE; WILD TYPE; ANIMAL; BLOOD; CHEMICALLY INDUCED DISORDER; FEASIBILITY STUDY; GENE THERAPY; GENETICS; HOSPITALIZATION; HUMAN; METABOLISM; METHODOLOGY; MOUSE MUTANT; THROMBOCYTE ADHESIVENESS; TIME;

ALPHA 1-ANTITRYPSIN; ANIMALS; BLEEDING TIME; CYTOMEGALOVIRUS; DISEASE MODELS, ANIMAL; FACTOR VIII; FEASIBILITY STUDIES; FERRIC COMPOUNDS; GENE THERAPY; GENE TRANSFER TECHNIQUES; HUMANS; LIVER; MICE; MICE, KNOCKOUT; PLATELET ADHESIVENESS; PLATELET AGGREGATION; PROMOTER REGIONS (GENETICS); SEVERITY OF ILLNESS INDEX; THROMBOSIS; TIME FACTORS; VON WILLEBRAND DISEASE; VON WILLEBRAND FACTOR;

EID: 51649122550     PISSN: 10795642     EISSN: None     Source Type: Journal    
DOI: 10.1161/ATVBAHA.108.168369     Document Type: Article

References (32)

1. Ruggeri ZM. Von Willebrand factor. Curr Opin Hematol. 2003;10: 142-149.
2. Sadler JE, Mannucci PM, Berntorp E, Bochkov N, Boulyjenkov V, Ginsburg D, Meyer D, Peake I, Rodeghiero F, Srivastava A. Impact, diagnosis and treatment of von Willebrand disease. Thromb Haemost. 2000;84:160-174.
3. De Meyer SF, Pareyn I, Baert J, Deckmyn H, Vanhoorelbeke K. False positive results in chimeraplasty for von Willebrand Disease. Thromb Res. 2007;119:93-104.
4. Lenting PJ, de Groot PG, De Meyer SF, Vanhoorelbeke K, Pruss C, Lillicrap D, Marx I, Denis CV. Correction of the bleeding time in von Willebrand factor (vWF)-deficient mice using murine vWF. Blood. 2007; 109:2267-2268.
5. Pergolizzi RG, Jin GC, Chan D, Pierre L, Bussel J, Ferris B, Leopold PL, Crystal RG. Correction of a murine model of von Willebrand disease by gene transfer. Blood. 2006;108:862-869.
6. De Meyer SF, Vanhoorelbeke K, Chuah MK, Pareyn I, Gillijns V, Hebbel RP, Collen D, Deckmyn H, VandenDriessche T. Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with, lentiviral vectors expressing von Willebrand factor. Blood. 2006;107: 4728-4736.
7. Kay MA, Li Q, Liu TJ, Leland F, Toman C, Finegold M, Woo SL. Hepatic gene therapy: persistent expression of human, alpha 1-antitrypsin in mice after direct gene delivery in vivo. Hum Gene Ther. 1992;3: 641-647.
8. Schmidt EV, Christoph. G, Zeller R, Leder P. The cytomegalovirus enhancer: a pan-active control element in transgenic mice. Mol Cell Biol. 1990;10:4406-4411.
9. Miao CH, Ohashi K, Patijn GA, Meuse L, Ye X, Thompson AR, Kay MA. Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro. Mol Ther. 2000;1:522-532.
10. Miao CH, Thompson AR, Loeb K, Ye X. Long-term and therapeutic-level hepatic gene expression of human factor IX after naked plasmid transfer in vivo. Mol Ther. 2001;3:947-957.
11. Denis C, Methia N, Frenette PS, Rayburn H, Ullman-Cullere M, Hynes RO, Wagner DD. A mouse model of severe von Willebrand disease: defects in hemostasis and thrombosis. Proc Natl Acad Sci USA. 1998;95: 9524-9529.
12. Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther. 1999;6:1258-1266.
13. Zhang G, Budker V, Wolff JA. High levels of foreign gene expression in hepatocytes after tail vein injections of naked plasmid DNA. Hum Gene Ther. 1999;10:1735-1737.
14. Roussi J, Turecek PL, Andre P, Bonneau M, Pignaud G, Sollier CBD, Schlokat U, Dorner F, Schwarz HP, Drouet L. Effects of human recombinant, plasma-derived and porcine von Willebrand factor in pigs with severe von Willebrand disease. Blood Coagul Fibrin. 1998;9:361-372.
15. Turecek PL, Pichler L, Auer W, Eder G, Varadi K, Mitterer A, Mundt W, Schlokat U, Dorner F, Drouet LO, Roussi J, van Mourik JA, Schwarz HP. Evidence for extracellular processing of pro-von Willebrand factor after infusion in animals with and without severe von Willebrand disease. Blood. 1999;94:1637-1647.
16. Turecek PL, Gritsch H, Pichler L, Auer W, Fischer B, Mitterer A, Mundt W, Schlokat U, Dorner F, Brinkman HJ, van Mourik JA, Schwarz HP. In vivo characterization of recombinant von Willebrand factor in dogs with von Willebrand disease. Blood. 1997;90:3555-3567.
17. Fischer BE, Schlokat U, Mitterer A, Reiter M, Mundt W, Turecek PL, Schwarz HP, Domer F. Structural analysis of recombinant von Willebrand factor produced at industrial scale fermentation of transformed CHO cells co-expressing recombinant furin. FEBS Lett. 1995;375: 259-262.
18. VandenDriessche T, Vanslembrouck V, Goovaerts I, Zwinnen H, Vanderhaeghen ML, Collen D, Chuah MK. Long-term expression, of human coagulation factor VIII and correction of hemophilia A after in vivo retroviral gene transfer in factor VITI- deficient mice. Proc Natl Acad Sci USA. 1999;96:10379-10384.
19. Follenzi A, Battaglia M, Lombardo A, Annoni A, Roncarolo MG, Naldini L. Targeting lentiviral vector expression to hepatocytes limits transgenespecific immune response and establishes long-term expression of human antihemophilic factor IX in mice. Blood. 2004;103:3700-3709.
20. VandenDriessche T, Thorrez L, Naldini L, Follenzi A, Moons L, Berneman Z, Collen D, Chuah MK. Lentiviral vectors containing the human immunodeficiency virus type-1 central polypurine tract can efficiently transduce nondividing hepatocytes and antigen-presenting cells in vivo. Blood. 2002;100:813-822.
21. Snyder RO, Miao C, Meuse L, Tubb J, Donahue BA, Lin HF, Stafford DW, Patel S, Thompson AR, Nichols T, Read MS, Bellinger DA, Brinkhous KM, Kay MA. Correction of hemophilia B in canine and murine models using recombinant adeno-associated viral vectors. Nat Med. 1999;5:64-70.
22. Chuah MK, Schiedner G, Thorrez L, Brown B, Johnston M, Gillijns V, Hertel S, Van Rooijen N, Lillicrap D, Collen D, VandenDriessche T, Kochanek S. Therapeutic factor VIII levels and negligible toxicity in mouse and dog models of hemophilia A following gene therapy with high-capacity adenoviral vectors. Blood. 2003;101:1734-1743.
23. 22a Khorsandi SE, Bachellier P, Weber JC, Greget M, Jaeck D, Zacharoulis D, Rountas C, Helmy S, Helmy A, Al-Waracky M, Salama H, Jiao L, Nicholls J, Davies AJ, Levicar N, Jensen S, Habib N. Minimally invasive and selective hydrodynamic gene therapy of liver segments in the pig and human. Cancer Gene Ther. 2008;15:225-230.
24. Herweijer H, Zhang G, Subbotin VM, Budker V, Williams P, Wolff JA. Time course of gene expression after plasmid DNA gene transfer to the liver. J Gene Med. 2001;3:280-291.
25. Nichols TC, Bellinger DA, Reddick RL, Smith SV, Koch GG, Davis K, Sigman J, Brinkhous KM, Griggs TR, Read MS. The roles of von Willebrand factor and factor VIII in arterial thrombosis: studies in canine von Willebrand disease and hemophilia A. Blood. 1993;81:2644-2651.
26. Nichols TC, Samama CM, Bellinger DA, Roussi J, Reddick RL, Bonneau M, Read MS, Bailliart O, Koch GG, Vaiman M. Function of von Willebrand factor after crossed bone marrow transplantation between normal and von Willebrand disease pigs: effect on arterial thrombosis in chimeras. Proc Natl Acad Sci USA. 1995;92:2455-2459.
27. Andre P, Brouland JP, Roussi J, Bonneau M, Pignaud G, Bal dit SC, Hainaud P, Vaiman M, Drouet L. Role of plasma and platelet von Willebrand factor in arterial thrombogenesis and hemostasis in the pig. Exp Hematol. 1998;26:620-626.
28. Budde U, Metzner HJ, Muller HG. Comparative analysis and classification of von Willebrand factor/factor VIII concentrates: impact on treatment of patients with von Willebrand disease. Semin Thromb Hemost. 2006;32:626-635.
29. Mannucci PM. How I treat patients with von Willebrand disease. Blood. 2001;97:1915-1919.
30. Mannucci PM, Chediak J, Hanna W, Byrnes J, Ledford M, Ewenstein BM, Retzios AD, Kapelan BA, Schwartz RS, Kessler C. Treatment of von Willebrand disease with a high-purity factor VIII/von Willebrand factor concentrate: a prospective, multicenter study. Blood 2002;99:450-456.
31. De Caterina R, Lanza M, Manca G, Strata GB, Maffei S, Salvatore L. Bleeding time and bleeding: an analysis of the relationship of the bleeding time test with parameters of surgical bleeding. Blood. 1994;84: 3363-3370.
32. Dobrkovska A, Krzensk U, Chediak JR. Pharmacokinetics, efficacy and safety of Humate-P in von Willebrand disease. Haemophilia. 1998;4 Suppl 3:33-39.