A novel approach for gene therapy: Engraftment of fibroblasts containing the artificial chromosome expression system at the site inflammation

Journal of Gene Medicine

Volumn 8, Issue 1, 2006, Pages 63-71

  Adriaansen, Janik ^a   Vervoordeldonk, Margriet J ^a   Vanderbyl, Sandra ^b   de Jong, Gary ^b   Tak, Paul P ^a  

^a UNIVERSITY OF AMSTERDAM   (Netherlands)

^b Chromos Molecular Systems Inc   (Canada)

Author keywords

Adjuvant arthritis; Artificial chromosomes; Ex vivo; Gene therapy; Rats; Rheumatoid arthritis

Indexed keywords

BETA GALACTOSIDASE; RED FLUORESCENT PROTEIN;

ADJUVANT ARTHRITIS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ARTIFICIAL CHROMOSOME; CONTROLLED STUDY; EX VIVO STUDY; FEASIBILITY STUDY; GENE EXPRESSION; GENETIC TRANSFECTION; MALE; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NONVIRAL GENE THERAPY; PRIORITY JOURNAL; RAT; REPORTER GENE; SKIN FIBROBLAST; SYNOVIOCYTE;

ANIMALS; ARTHRITIS, EXPERIMENTAL; CELLS, CULTURED; CHROMOSOMES, ARTIFICIAL, MAMMALIAN; FEASIBILITY STUDIES; FIBROBLASTS; GENE THERAPY; INFLAMMATION; LAC OPERON; MALE; RATS; RATS, INBRED LEW; TRANSFECTION; TRANSGENES;

ANIMALIA; MURINAE;

EID: 31644435920     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.810     Document Type: Article

References (31)

1. Bathon JM, Martin RW, Fleischmann RM, et al. A comparison of etanercept and methotrexate in patients with early rheumatoid arthritis. N Engl J Med 2000; 343: 1586-1593.
2. Lipsky PE, van der Heijde DM, St Clair EW, et al. Infliximab and methotrexate in the treatment of rheumatoid arthritis. Anti-Tumor Necrosis Factor Trial in Rheumatoid Arthritis with Concomitant Therapy Study Group. N Engl J Med 2000; 343: 1594-1602.
3. Chernajovsky Y, Gould DJ, Podhajcer OL. Gene therapy for autoimmune diseases: quo vadis? Nat Rev Immunol 2004; 4: 800-811.
4. Vervoordeldonk M, Tak PP. Gene therapy in rheumatic diseases. Best Pract Res Clin Rheumatol 2001; 15: 771-788.
5. Check E. Second cancer case halts gene-therapy trials. Nature 2003; 421: 305.
6. Li ZX, Dullmann J, Schiedlmeier B, et al. Murine leukemia induced by retroviral gene marking. Science 2002; 296: 497.
7. Bessis N, GarciaCozar FJ, Boissier MC. Immune responses to gene therapy vectors: influence on vector function and effector mechanisms. Gene Ther 2004; 11: S10-S17.
8. Grimm D, Kay MA. From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy. Curr Gene Ther 2003; 3: 281-304.
9. Grimm D. Production methods for gene transfer vectors based on adeno-associated virus serotypes. Methods 2002; 28: 146-157.
10. Telenius H, Szeles A, Kereso J, et al. Stability of a functional murine satellite DNA-based artificial chromosome across mammalian species. Chromosome Res 1999; 7: 3-7.
11. deJong G, Telenius AH, Telenius H, et al. Mammalian artificial chromosome pilot production facility: large-scale isolation of functional satellite DNA-based artificial chromosomes. Cytometry 1999; 35: 129-133.
12. Hadlaczky G. Satellite DNA-based artificial chromosomes for use in gene therapy. Curr Opin Mol Ther 2001; 3: 125-132.
13. Lindenbaum M, Perkins E, Csonka E, et al. A mammalian artificial chromosome engineering system (ACE System) applicable to biopharmaceutical protein production, transgenesis and gene-based cell therapy. Nucleic Acids Res 2004; 32: e172.
14. de Jong G, Telenius A, Vanderbyl S, et al. Efficient in-vitro transfer of a 60-Mb mammalian artificial chromosome into murine and hamster cells using cationic lipids and dendrimers. Chromosome Res 2001; 9: 475-485.
15. Lipps HJ, Jenke ACW, Nehlsen K, et al. Chromosome-based vectors for gene therapy. Gene 2003; 304: 23-33.
16. Vos JMH. The simplicity of complex MACs. Nat Biotechnol 1997; 15: 1257-1259.
17. Huxley C. Mammalian artificial chromosomes and chromosome transgenics. Trends Genet 1997; 13: 345-347.
18. Stewart S, MacDonald N, Perkins E, et al. Retrofitting of a satellite repeat DNA-based murine artificial chromosome (ACes) to contain loxP recombination sites. Gene Ther 2002; 9: 719-723.
19. Alvarogracia JM, Zvaifler NJ, Brown CB, et al. Cytokines in chronic inflammatory arthritis.6. Analysis of the synovial cells involved in granulocyte-macrophage colony-stimulating factor production and gene expression in rheumatoid arthritis and its regulation by Il-1 and tumor-necrosis-factor-alpha. J Immunol 1991; 146: 3365-3371.
20. Vanderbyl S, MacDonald N, de Jong G. A flow cytometry technique for measuring chromosome-mediated gene transfer. Cytometry 2001; 44: 100-105.
21. Tak PP, Gerlag DM, Aupperle KR, et al. Inhibitor of nuclear factor kappa B kinase beta is a key regulator of synovial inflammation. Arthritis Rheumatism 2001; 44: 1897-1907.
22. Nguyen KHY, Boyle DL, McCormack JE, et al. Direct synovial gene transfer with retroviral vectors in rat adjuvant arthritis. J Rheumatol 1998; 25: 1118-1125.
23. Kraan MC, Smith MD, Weedon H, et al. Measurement of cytokine and adhesion molecule expression in synovial tissue by digital image analysis. Ann Rheum Dis 2001; 60: 296-298.
24. t'Hart BA, Vervoordeldonk M, Heeney JL, et al. Gene therapy in nonhuman primate models of human autoimmune disease. Gene Ther 2003; 10: 890-901.
25. Robbins PD, Evans CH, Chernajovsky Y. Gene therapy for arthritis. Gene Ther 2003; 10: 902-911.
26. Vanderbyl S, MacDonald N, Sidhu S, et al. Transfer and stable transgene expression of mammalian artificial chromosome into bone marrow derived human mesenchymal stem cells. Stem Cells 2004; 22: 324-333.
27. Co DO, Borowski AH, Leung JD, et al. Generation of transgenic mice and germline transmission of a mammalian artificial chromosome introduced into embryos by pronuclear microinjection. Chromosome Res 2000; 8: 183-191.
28. Monteith D, Leung JD, Borowski AH, et al. Pronuclear microinjection of purified artificial chromosomes for generation of transgenic mice: pick-and-inject technique. Methods Mol Biol 2004; 240: 227-242.
29. Evans CH, Robbins P, Ghivizzani SC, et al. Clinical trial to assess the safety, feasibility, and efficacy of transferring a potentially anti-arthritic cytokine to human joints with rheumatoid arthritis. Hum Gene Ther 1996; 7: 1261-1280.
30. Evans CH, Ghivizzani SC, Herndon JH, et al. Clinical trials in the gene therapy of arthritis. Clin Orthop Rel Res 2000; 379: S300-S307.
31. Roth DA, Tawa NE, O'Brien JM, et al. Nonviral transfer of the gene encoding coagulation factor VIII in patients with severe hemophilia A. N Engl J Med 2001; 344: 1735-1742.