Phenotypic correction and long-term expression of factor VIII in hemophilic mice by immunotolerization and nonviral gene transfer using the Sleeping Beauty transposon system

Blood

Volumn 105, Issue 7, 2005, Pages 2691-2698

  Ohlfest, John R ^b   Frandsen, Joel L ^b   Fritz, Sabine ^b   Lobitz, Paul D ^b   Perkinson, Scott G ^b   Clark, Karl J ^b   Nelsestuen, Gary ^b   Key, Nigel S ^b   McIvor, R Scott ^b   Hackett, Perry B ^b   Largaespada, David A ^a  

^a UNIVERSITY OF MINNESOTA   (United States)

^b NONE

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; COMPLEMENTARY DNA; GENOMIC DNA; PLASMID DNA; PLASMID VECTOR; RECOMBINANT BLOOD CLOTTING FACTOR 8; TRANSPOSASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIBODY PRODUCTION; ARTICLE; BLOOD CLOTTING TIME; CONTROLLED STUDY; ENZYME LINKED IMMUNOSORBENT ASSAY; GENE TRANSFER; HELA CELL; HEMOPHILIA A; HUMAN; HUMAN CELL; IMMUNOLOGICAL TOLERANCE; IN VITRO STUDY; MOUSE; NEWBORN; NONHUMAN; NONVIRAL GENE THERAPY; PHENOTYPE; POLYMERASE CHAIN REACTION; PRIORITY JOURNAL; TRANSGENE; TRANSPOSON;

EID: 20144386644     PISSN: 00064971     EISSN: None     Source Type: Journal    
DOI: 10.1182/blood-2004-09-3496     Document Type: Article

References (51)

1. Kay MA, High K. Gene therapy for the hemophilias. Proc Natl Acad Sci U S A. 1999;96:9973-9975.
2. Bohn RL, Aledort LM, Putnam KG, Ewenstein BM, Mogun H, Avorn J. The economic impact of factor VIII inhibitors in patients with haemophilia. Haemophilia. 2004;10:63-68.
3. Nathwani AC, Davidoff AM, Tuddenham EG. Prospects for gene therapy of haemophilia. Haemophilia. 2004;10:309-318.
4. Marshall E. Gene therapy death prompts review of adenovirus vector. Science. 1999;286:2244-2245.
5. Kaiser J. Gene therapy: side effects sideline hemophilia trial. Science. 2004;304:1423-1425.
6. Ritter T, Lehmann M, Volk HD. Improvements in gene therapy: averting the immune response to adenoviral vectors. BioDrugs. 2002;16:3-10.
7. Halbert CL, Rutledge EA, Allen JM, Russell DW, Miller AD. Repeat transduction in the mouse lung by using adeno-associated virus vectors with different serotypes. J Virol. 2000;74:1524-1532.
8. Hu CS, Hong JY, Douglas WL. Principles of safety, efficacy and stability in gene therapy: review. Zhongguo Shi Van Xue Ye Xue Za Zhi. 2004;12:392-396.
9. Miao CH, Ye X, Thompson AR. High-level factor VIII gene expression in vivo achieved by nonviral liver-specific gene therapy vectors. Hum Gene Ther. 2003;14:1297-1305.
10. Olivares EC, Hollis RP, Chalberg TW, Meuse L, Kay MA, Calos MP. Site-specific genomic integration produces therapeutic Factor IX levels in mice. Nat Biotechnol. 2002;20:1124-1128.
11. Yant SR, Meuse L, Chiu W, Ivics Z, Izsvak Z, Kay MA. Somatic integration and long-term transgene expression in normal and haemophilia mice using a DNA transposon system. Nat Genet. 2000;25:35-41.
12. Kreuz W, Becker S, Lenz E, et al. Factor VIII inhibitors in patients with hemophilia A: epidemiology of inhibitor development and induction of immune tolerance for factor VIII. Semin Thromb Hemost. 1995;21:382-389.
13. Kootstra NA, Matsumura R, Verma IM. Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol Ther. 2003;7:623-631.
14. Ye P, Thompson AR, Sarkar R, et al. Naked DNA transfer of factor VIII induced transgene-specific, species-independent immune response in hemophilia A mice. Mol Ther. 2004;10:117-126.
15. Sarkar R, Gao GP, Chirmule N, Tazelaar J, Kazazian HH Jr. Partial correction of murine hemophilia A with neo-antigenic murine factor VIII. Hum Gene Ther. 2000;11:881-894.
16. Pittman DD, Alderman EM, Tomkinson KN, Wang JH, Giles AR, Kaufman RJ. Biochemical, immunological, and in vivo functional characterization of B-domain-deleted factor VIII. Blood. 1993;81: 2925-2935.
17. Madoiwa S, Yamauchi T, Hakamata Y, et al. Induction of immune tolerance by neonatal intravenous injection of human factor VIII in murine hemophilia A. J Thromb Haemost. 2004;2:754-762.
18. Vigdal TJ, Kaufman CD, Izsvak Z, Voytas DF, Ivics Z. Common physical properties of DNA affecting target site selection of sleeping beauty and other Tc1/mariner transposable elements. J Mol Biol. 2002;323:441-452.
19. Ivics Z, Hackett PB, Plasterk RH, Izsvak Z. Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells. Cell. 1997;91:501-510.
20. Niwa H, Yamamura K, Miyazaki J. Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene. 1991;108:193-199.
21. Lin Y, Chang L, Solovey A, Healey JF, Lollar P, Hebbel RP. Use of blood outgrowth endothelial cells for gene therapy for hemophilia A. Blood. 2002;99:457-462.
22. Ohlfest JR, Lobitz PD, Perkinson SG, Largaespada DA. Integration and long-term expression in xenografted human glioblastoma cells using a plasmid-based transposon system. Mol Ther. 2004;10:260-268.
23. Bi L, Lawler AM, Antonarakis SE, High KA, Gearhart JD, Kazazian HH Jr. Targeted disruption of the mouse factor VIII gene produces a model of haemophilia A. Nat Genet. 1995;10:119-121.
24. Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther. 1999;6:1258-1266.
25. Henderson N, Key NS, Christie B, Kisiel W, Foster D, Nelsestuen GL. Response of factor VIII and IX-deficient blood to wild type and high membrane affinity mutant factor VIIa in an in vitro whole blood clotting assay: possible correlation to clinical outcome. Thromb Haemost. 2002;88:98-103.
26. Carlson CM, Dupuy AJ, Fritz S, Roberg-Perez KJ, Fletcher CF, Largaespada DA. Transposon mutagenesis of the mouse germline. Genetics. 2003;165:243-256.
27. Ehrenforth S, Kreuz W, Scharrer I, et al. Incidence of development of factor VIII and factor IX inhibitors in haemophiliacs. Lancet. 1992;339: 594-598.
28. Clayton JP, Gammon GM, Ando DG, Kono DH, Hood L, Sercarz EE. Peptide-specific prevention of experimental allergic encephalomyelitis: neonatal tolerance induced to the dominant T cell determinant of myelin basic protein. J Exp Med. 1989;169:1681-1691.
29. Gammon GM, Oki A, Shastri N, Sercarz EE. Induction of tolerance to one determinant on a synthetic peptide does not affect the response to a second linked determinant: implications for the mechanism of neonatal tolerance induction. J Exp Med. 1986;164:667-672.
30. Gammon G, Dunn K, Shastri N, Oki A, Wilbur S, Sercarz EE. Neonatal T-cell tolerance to minimal immunogenic peptides is caused by clonal inactivation. Nature. 1986;319:413-415.
31. Geurts AM, Yang Y, Clark KJ, et al. Gene transfer into genomes of human cells by the sleeping beauty transposon system. Mol Ther. 2003;8: 108-117.
32. Zayed H, Izsvak Z, Walisko O, Ivics Z. Development of hyperactive sleeping beauty transposon vectors by mutational analysis. Mol Ther. 2004;9: 292-304.
33. Yant SR, Park J, Huang Y, Mikkelsen JG, Kay MA. Mutational analysis of the N-terminal DNA-binding domain of Sleeping Beauty transposase: critical residues for DNA binding and hyperactivity in mammalian cells. Mol Cell Biol. 2004;24:9239-9247.
34. Mikkelsen JG, Yant SR, Meuse L, Huang Z, Xu H, Kay MA. Helper-independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo. Mol Ther. 2003;8:654-665.
35. 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.
36. Rossmanith W, Chabicovsky M, Herkner K, Schulte-Hermann R. Cellular gene dose and kinetics of gene expression in mouse livers transfected by high-volume tail-vein injection of naked DNA. DNA Cell Biol. 2002;21:847-853.
37. Chen ZY, He CY, Meuse L, Kay MA. Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo. Gene Ther. 2004;11: 856-864.
38. Chen ZY, He CY, Ehrhardt A, Kay MA. Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther. 2003;8:495-500.
39. Chao H, Mao L, Bruce AT, Walsh CE. Sustained expression of human factor VIII in mice using a parvovirus-based vector. Blood. 2000;95:1594-1599.
40. Qian J, Collins M, Sharpe AH, Hoyer LW. Prevention and treatment of factor VIII inhibitors in murine hemophilia A. Blood. 2000;95:1324-1329.
41. Wu H, Reding M, Qian J, et al. Mechanism of the immune response to human factor VIII in murine hemophilia A. Thromb Haemost. 2001;85:125-133.
42. Evans GL, Morgan RA. Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A. Proc Natl Acad Sci U S A. 1998;95:5734-5739.
43. Olivares EC, Hollis RP, Calos MP. Phage R4 integrase mediates site-specific integration in human cells. Gene. 2001;278:167-176.
44. Wu X, Li Y, Crise B, Burgess SM. Transcription start regions in the human genome are favored targets for MLV integration. Science. 2003;300: 1749-1751.
45. Miller DG, Rutledge EA, Russell DW. Chromosomal effects of adeno-associated virus vector integration. Nat Genet. 2002;30:147-148.
46. Nakai H, Iwaki Y, Kay MA, Couto LB. Isolation of recombinant adeno-associated virus vector-cellular DNA junctions from mouse liver. J Virol. 1999; 73:5438-5447.
47. Dean DA, Machado-Aranda D, Blair-Parks K, Yeldandi AV, Young JL. Electroporation as a method for high-level nonviral gene transfer to the lung. Gene Ther. 2003;10:1608-1615.
48. Sandri M, Bortoloso E, Nori A, Volpe P. Electrotransfer in differentiated myotubes: a novel, efficient procedure for functional gene transfer. Exp Cell Res. 2003;286:87-95.
49. Zarnitsyn VG, Prausnitz MR. Physical parameters influencing optimization of ultrasound-mediated DNA transfection. Ultrasound Med Biol. 2004;30:527-538.
50. Belur LR, Frandsen JL, Dupuy AJ, et al. Gene insertion and long-term expression in lung mediated by the Sleeping Beauty transposon system. Mol Ther. 2003;8:501-507.
51. Eastman SJ, Baskin KM, Hodges BL, et al. Development of catheter-based procedures for transducing the isolated rabbit liver with plasmid DNA. Hum Gene Ther. 2002;13:2065-2077.