Phenotypic correction of von Willebrand disease type 3 blood-derived endothelial cells with lentiviral vectors expressing von Willebrand factor

Blood

Volumn 107, Issue 12, 2006, Pages 4728-4736

  De Meyer, Simon F ^c   Vanhoorelbeke, Karen ^c   Chuah, Marinee K ^c   Pareyn, Inge ^c   Gillijns, Veerle ^c   Hebbel, Robert P ^c   Collen, Désiré ^c   Deckmyn, Hans ^b   VandenDriessche, Thierry ^a  

^a DEPARTMENT OF PLANT SYSTEMS BIOLOGY   (Belgium)

^b UNIVERSITY OF LEUVEN   (Belgium)

^c NONE

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; COLLAGEN; GLYCOPROTEIN IB ALPHA; LENTIVIRUS VECTOR; RISTOCETIN; VON WILLEBRAND FACTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL BASED GENE THERAPY; CELL ISOLATION; CELL STRAIN COS7; CONTROLLED STUDY; CYTOMEGALOVIRUS; DISEASE CLASSIFICATION; DOG; DRUG BINDING; DRUG MECHANISM; DRUG SYNTHESIS; ENDOTHELIUM CELL; GENETIC TRANSDUCTION; GENETIC TRANSFECTION; GENOTYPE; HUMAN; HUMAN CELL; IMMUNOFLUORESCENCE; NONHUMAN; PERIPHERAL BLOOD MONONUCLEAR CELL; PHENOTYPE; POINT MUTATION; PRIORITY JOURNAL; PROTEIN BINDING; PROTEIN EXPRESSION; PROTEIN FUNCTION; PROTEIN SYNTHESIS; PROTEIN TRANSPORT; TARGET CELL; VIRAL GENE DELIVERY SYSTEM; VON WILLEBRAND DISEASE;

EID: 33745100551     PISSN: 00064971     EISSN: 00064971     Source Type: Journal    
DOI: 10.1182/blood-2005-09-3605     Document Type: Article

References (43)

1. Ruggeri ZM. Structure of von Willebrand factor and its function in platelet adhesion and thrombus formation. Best Pract Res Clin Haematol. 2001;14:257-279.
2. Kaufmann JE, Oksche A, Wollheim CB, et al. Vasopressin-induced von Willebrand factor secretion from endothelial cells involves V2 receptors and cAMP. J Clin Invest. 2000;106:107-116.
3. Rodeghiero F, Castaman G. Treatment of von Willebrand disease. Semin Hematol. 2005;42:29-35.
4. Mannucci PM, Bettega D, Cattaneo M. Patterns of development of tachyphylaxis in patients with haemophilia and von Willebrand disease after repeated doses of desmopressin (DDAVP). Br J Haematol. 1992;82:87-93.
5. Bond L, Bevan D. Myocardial infarction in a patient with hemophilia treated with DDAVP [letter]. N Engl J Med. 1988;318:121.
6. Byrnes JJ, Larcada A, Moake JL. Thrombosis following desmopressin for uremic bleeding. Am J Hematol. 1988;28:63-65.
7. Mendolicchio GL, Ruggeri ZM. New perspectives on von Willebrand factor functions in hemostasis and thrombosis. Semin Hematol. 2005;42:5-14.
8. Lin Y, Chang L, Solovey A, et al. Use of blood outgrowth endothelial cells for gene therapy for hemophilia A. Blood. 2002;99:457-462.
9. Chuah MK, Schiedner G, Thorrez L, et al. 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.
10. Chuah MK, Collen D, VandenDriessche T. Biosafety of adenoviral vectors. Curr Gene Ther. 2003;3:527-543.
11. High KA, Manno CS, Sabatino DE, et al. Immune responses to AAV and to factor IX in a phase I study of AAV-mediated, liver-directed gene transfer for hemophilia B. Blood. 2003;102:154A-155A.
12. Raper SE, Chirmule N, Lee FS, et al. Fatal systemic inflammatory response syndrome in a ornithine transcarbamylase deficient patient following adenoviral gene transfer. Mol Genet Metab. 2003;80:148-158.
13. VandenDriessche T, Thorrez L, Naldini L, et al. 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.
14. Herder C, Tonn T, Oostendorp R, et al. Sustained expansion and transgene expression of coagulation factor VIII-transduced cord blood-derived endothelial progenitor cells. Arterioscler Thromb Vasc Biol. 2003;23:2266-2272.
15. De Meyer SF, Pareyn I, Baert J, Deckmyn H, Vanhoorelbeke K. False positive results in chimeraplasty for von Willebrand disease. Thromb Res. Prepublished on January 31, 2006, as DOI 10.1016/j.thrombres.2005.12.009.
16. Rieger M, Schwarz HP, Turecek PL, et al. Identification of mutations in the canine von Willebrand factor gene associated with type III von Willebrand disease. Thromb Haemost. 1998;80:332-337.
17. Janssens W, Chuah MK, Naldini L, et al. Efficiency of onco-retroviral and lentiviral gene transfer into primary mouse and human B-lymphocytes is pseudotype dependent. Hum Gene Ther. 2003;14:263-276.
18. VandenDriessche T, Naldini L, Collen D, Chuah MK. Oncoretroviral and lentiviral vector-mediated gene therapy. Methods Enzymol. 2002;346:573-589.
19. Vanhoorelbeke K, Cauwenberghs N, Vauterin S, et al. A reliable and reproducible ELISA method to measure ristocetin cofactor activity of von Willebrand factor. Thromb Haemost. 2000;83:107-113.
20. Cauwenberghs N, Vanhoorelbeke K, Vauterin S, et al. Epitope mapping of inhibitory antibodies against platelet glycoprotein Ibalpha reveals interaction between the leucine-rich repeat N-terminal and C-terminal flanking domains of glycoprotein Ibalpha. Blood. 2001;98:652-660.
21. Ulrichts H, Vanhoorelbeke K, Cauwenberghs S, et al. Von Willebrand factor but not alpha-thrombin binding to platelet glycoprotein Ib alpha is influenced by the HPA-2 polymorphism. Arterioscler Thromb Vasc Biol. 2003;23:1302-1307.
22. Ruggeri ZM, Zimmerman TS. The complex multimeric composition of factor VIII/von Willebrand factor. Blood. 1981;57:1140-1143.
23. Goudemand J, Mazurier C, Parquet-Gernez A, Goudemand M. HLA antigen and Willebrand's disease. Pathol Biol. 1977;25:241-243.
24. Turecek PL, Gritsch H, Pichler L, et al. In vivo characterization of recombinant von Willebrand factor in dogs with von Willebrand disease. Blood. 1997;90:3555-3567.
25. Roussi J, Turecek PL, Andre P, et al. Effects of human recombinant, plasma-derived and porcine von Willebrand factor in pigs with severe von Willebrand disease. Blood Coagul Fibrinolysis. 1998;9:361-372.
26. Turecek PL, Pichler L, Auer W, et al. 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.
27. Liu ML, Winther BL, Kay MA. Pseudotransduction of hepatocytes by using concentrated pseudotyped vesicular stomatitis virus G glycoprotein (VSV-G)-Moloney murine leukemia virus-derived retrovirus vectors: comparison of VSV-G and amphotropic vectors for hepatic gene transfer. J Virol. 1996;70:2497-2502.
28. Van Damme A, Thorrez L, Ma L, et al. Efficient lentiviral transduction and improved engraftment of human bone marrow mesenchymal cells. Stem Cells. Prepublished on December 9, 2005, as DOI 10.1634/stemcells.2003-0106.
29. Federici AB, Castaman G, Mannucci PM. Guidelines for the diagnosis and management of von Willebrand disease in Italy. Haemophilia. 2002;8:607-621.
30. Fischer BE, Schlokat U, Mitterer A, et al. 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.
31. Verweij CL, Hart M, Pannekoek H. Proteolytic cleavage of the precursor of von Willebrand factor is not essential for multimer formation. J Biol Chem. 1988;263:7921-7924.
32. Mayadas TN, Wagner DD. In vitro multimerization of von Willebrand factor is triggered by low pH: importance of the propolypeptide and free sulfhydryls. J Biol Chem. 1989;264:13497-13503.
33. Wagner DD, Mayadas T, Marder VJ. Initial glycosylation and acidic pH in the Golgi apparatus are required for multimerization of von Willebrand factor. J Cell Biol. 1986;102:1320-1324.
34. Wagner D. Storage and secretion of von Willebrand factor. In: Ruggeri ZM, Zimmerman TS, eds. Coagulation and Bleeding Disorders: The Role of FVIII and VWF. New York: Marcel Dekker; 1989:161-180.
35. Haberichter SL, Merricks EP, Fahs SA, et al. Reestablishment of VWF-dependent Weibel-Palade bodies in VWD endothelial cells. Blood. 2005;105:145-152.
36. Do H, Healey JF, Waller EK, Lollar P. Expression of factor VIII by murine liver sinusoidal endothelial cells. J Biol Chem. 1999;274:19587-19592.
37. Huber D, Cramer EM, Kaufmann JE, et al. Tissue-type plasminogen activator (t-PA) is stored in Weibel-Palade bodies in human endothelial cells both in vitro and in vivo. Blood. 2002;99:3637-3645.
38. Romani dW, de Leeuw HP, Rondaij MG, et al. Von Willebrand factor targets IL-8 to Weibel-Palade bodies in an endothelial cell line. Exp Cell Res. 2003;286:67-74.
39. Rosenberg JB, Greengard JS, Montgomery RR. Genetic induction of a releasable pool of factor VIII in human endothelial cells. Arterioscler Thromb Vasc Biol. 2000;20:2689-2695.
40. Wolff B, Burns AR, Middleton J, Rot A. Endothelial cell "memory" of inflammatory stimulation: human venular endothelial cells store interleukin 8 in Weibel-Palade bodies. J Exp Med. 1998;188:1757-1762.
41. Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science. 2003;302:415-419.
42. Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N Engl J Med. 2003;348:255-256.
43. Chung JH, Whiteley M, Felsenfeld G. A 5′ element of the chicken beta-globin domain serves as an insulator in human erythroid cells and protects against position effect in Drosophila. Cell. 1993;74:505-514.