An engineered EBV vector expressing human factor VIII and von Willebrand factor in cultured B-cells

Journal of Gene Medicine

Volumn 6, Issue 7, 2004, Pages 760-768

  Basu, Utpal ^a   Banerjee, Subrata ^a  

^a SAHA INSTITUTE OF NUCLEAR PHYSICS   (India)

Author keywords

EBV; Gene therapy; Hematopoietic cells; Hemophilia A; von Willebrand factor

Indexed keywords

BLOOD CLOTTING FACTOR 8; COMPLEMENTARY DNA; THYMIDINE KINASE; VIRUS VECTOR; VON WILLEBRAND FACTOR;

ARTICLE; B LYMPHOCYTE; CELL BASED GENE THERAPY; CELL CULTURE; CONTROLLED STUDY; ENZYME LINKED IMMUNOSORBENT ASSAY; EPSTEIN BARR VIRUS; GENE AMPLIFICATION; GENE CONSTRUCT; GENE DELETION; GENE DELIVERY SYSTEM; GENE EXPRESSION; GENE TRANSFER; GENETIC ENGINEERING; GENETIC TRANSDUCTION; GENETIC TRANSFECTION; HEMATOPOIETIC CELL; HEMOPHILIA A; HUMAN; HUMAN CELL; IMMUNOHISTOCHEMISTRY; IN VITRO STUDY; PRIORITY JOURNAL; REPORTER GENE; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; RNA ISOLATION; VIRAL GENE DELIVERY SYSTEM;

B-LYMPHOCYTES; BASE SEQUENCE; CELLS, CULTURED; DNA PRIMERS; DNA, COMPLEMENTARY; ENZYME-LINKED IMMUNOSORBENT ASSAY; FACTOR VIII; GENETIC ENGINEERING; HERPESVIRUS 4, HUMAN; HUMANS; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; VON WILLEBRAND FACTOR;

HUMAN HERPESVIRUS 4;

EID: 13644267946     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.571     Document Type: Article

References (35)

1. Kaufman RJ, Antonarakis SE. Structure, biology, and genetics of factor VIII. In Hematology: Basic Principles and Practice. Hoffman R, Benz EJ, Shattil SJ, Furie B, Cohen HJ (eds). Churchill Livingstone: New York, 2000; 1850-1868.
2. Kazazian HH, Tuddenham EGD, Antonarakis SE. Hemophilia A and parahemophilia: deficiencies of coagulation factors VIII and V. In The Metabolic and Molecular Bases of Inherited Disease, Scriver CR, Beaudet AL, Sly WS, Valle D (eds). McGraw-Hill Inc.: New York, 1994; 3241-3267.
3. Lenting PJ, van Mourik JA, Mertens K. The life cycle of coagulation factor VIII in view of its structure and function. Blood 1998; 92: 3983-3996.
4. Lynch CM. Gene therapy of hemophilia. Curr Opin Mol Ther 1999; 1: 493-499.
5. Van Damme A, VandenDriessche T, Collen D, Chuah MKL. Bone marrow stromal cells as targets for gene therapy. Curr Gene Ther 2002; 2: 1-15.
6. Chuah MKL, VandenDriessche T, Morgan RA. Development and analysis of retroviral vectors expressing human factor VIII as a potential gene therapy for hemophilia A. Hum Gene Ther 1995; 6 1363-1377.
7. Hoeben RC, Einerhard MP, Briet E, Van Ormondt H, Valerio D, Van Der Eb AJ. Toward gene therapy for hemophilia A: retrovirus-mediated transfer of a FVIII gene into murine haematopoietic progenitor cells. Thromb Haemost 1992; 67: 341-345.
8. Tonn T, Herder C, Becker S, Seifried E, Grez M. Generation and characterization of human hematopoietic cell lines expressing factor VIII. J. Hematother Stem Cell Res 2002; 11: 695-704.
9. Banerjee S, Livanos E, Vos JMH. Therapeutic gene delivery in human B-lymphoblastoid cells by engineered non-transforming infectious Epstein-Barr virus. Nat Med 1995; 1: 1303-1308.
10. Sun TQ, Livanos E, Vos JMH. Engineering a mini-herpesvirus as general strategy to transduce up to 180kb of functional self-replicating human mini-chromosomes. Gene Ther 1996; 3: 1081-1088.
11. White RE, Wade-Martins R, James MR. Infectious delivery of 120-kilobase genomic DNA by an Epstein-Barr virus amplicon vector. Mol Ther 2002; 5: 427-435.
12. Sun TQ, Vos JMH. Packaging of 200kb engineered DNA as infectious Epstein Barr virus. Int J Genome Res 1992; 1: 45-57.
13. Shizuya H, Bitten BW, Slepak T, et al. Cloning and stable maintenance of 300 kilobase pair fragments of human DNA in Escherichia coli using an F-factor based vector. Proc Natl Acad Sci USA 1992; 89: 8794-8797.
14. Wagner MJ, Sharp JA, Summers WC. Nucleotide sequence of the thymidine kinase gene of herpes simplex virus type 1. Proc Natl Acad Sci U S A 1981; 78: 1441-1445.
15. Stel HV, van der Kwast TH, Veerman ECL. Detection of factor VIII/coagulant antigen in human liver tissue. Nature 1983; 303 530-532.
16. Gruppo RA, Brown D, Wilkes MM, Navickis RJ. Comparative effectiveness of full-length and B-domain deleted factor VIII for prophylaxis-a meta-analysis. Hemophilia 2003; 9: 251-260.
17. Savard M, Belanger C, Tardif M, Gourde P, Flamand L, Gosselin J. Infection of primary human monocytes by Epstein-Barr virus. J Virol 2000; 74: 2612-2619.
18. Larochelle B, Flamand L, Gourde P, Beauchamp D, Gosselin J. Epstein-Barr virus infects and induces apoptosis in human neutrophils. Blood 1998; 92: 291-299.
19. Guan M, Romano G, Henderson EE. In situ RT-PCR detection of Epstein-Barr virus immediate-early transcripts in CD4+ and CD8 + T lymphocytes. Anticancer Res 1998; 18: 3171-3180.
20. Stoll SM, Sclimenti CR, Baba EJ, Meuse L, Kay MA, Calos MP. Epstein-Barr virus/human vector provides high-level, long-term expression of alpha1-antitrypsin in mice. Mol Ther 2001; 4: 122-129.
21. Cui FD, Kishida T, Ohashi S, et al. Highly efficient gene transfer into murine liver achieved by intravenous administration of naked Epstein-Barr virus (EBV)-based plasmid vectors. Gene Ther 2001; 8: 1508-1513.
22. Li Z, Dullmann J, Schiedlmeier B, et al. Murine leukemia induced by retroviral gene marking. Science 2002; 296 497.
23. Hacein-Bey-Abina S, von Kalle C, Schmidt M, et al. A serious adverse event after successful gene therapy for severe combined immunodeficiency. N Eng J Med 2003; 348: 255-256.
24. Hammerschmidt W. Herpesvirus vectors come of age. Curr Opin Mol Ther 2000; 2: 532-539.
25. Janssens W, Chuah MK, Naldini L, et al. Efficiency of oncoretroviral and lentiviral gene transfer into primary mouse and human B-lymphocytes is pseudotype dependent. Hum Gene Ther 2003; 14 263-276.
26. Bovia F, Salmon P, Matthes T, et al. Efficient transduction of primary human B lymphocytes and nondividing myeloma B cells with HIV-1-derived lentiviral vectors. Blood 2003; 101: 1727-1733.
27. Rickinson AB, Kieff E. Epstein Barr virus. In Virology, Fields BN, Knipe DM, Howley PM (eds). Lippincott Raven: Philadelphia. 1996; 2397-2446.
28. Thorley-Lawson DA. EBV persistence in vivo. Invading and avoiding the immune response. In Herpesviruses and Immunity, Medveczky PG, Friedman H, Bendinelli M (eds). Plenum Press: New York. 1998; 207-229.
29. Levitskaya J, Coram M, Levitsky V, et al. Inhibition of antigen processing by the internal repeat region of the Epstein-Barr nuclear antigen-1. Nature 1995; 375: 685-688.
30. Tsimbouri P, Drotar ME, Coy JL, Wilson JB. bcl-xL and RAG genes are induced and the response to IL-2 enhanced in EmuEBNA-1 transgenic mouse lymphocytes. Oncogene 2002; 21: 5182-5187.
31. Chuang TC, Way TD, Lin YS, Lee YC, Law SL, Kao MC. The Epstein-Barr virus nuclear antigen-1 may act as a transforming suppressor of the HER2/neu oncogene. FEBS Lett 2002; 532: 135-142.
32. Delecluse HJ, Pich D, Hilsendegen T, Baum C, Hammerschmidt W. A first-generation packaging cell line for Epstein-Barr virus-derived vectors. Proc Natl Acad Sci U S A 1999; 96: 5188-5193.
33. + cells by transfecting EBV-based episomal vectors. FEBS Lett 1998; 441: 39-42.
34. Behrmann M, Pasi J, Saint-Remy JM, Kotitschke R, Kloft M. Von Willebrand factor modulates factor VIII immunogenicity: comparative study of different factor VIII concentrates in a haemophilia A mouse model. Thromb Haemost 2002; 8: 221-229.
35. Kootstra NA, Matsumura R, Verma. IM. Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol Ther 2003; 7: 623-631.