Sleeping Beauty transposition: Biology and applications for molecular therapy

Molecular Therapy

Volumn 9, Issue 2, 2004, Pages 147-156

  Izsvák, Zsuzsanna ^a,b   Ivics, Zoltán ^a  

^a MAX DELBRÜCK CENTER FOR MOLECULAR MEDICINE   (Germany)

^b INSTITUTE OF BIOCHEMISTRY   (Hungary)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA 1 ANTITRYPSIN; BLOOD CLOTTING FACTOR 9; FUMARYLACETOACETASE; HIGH MOBILITY GROUP B1 PROTEIN; HOST FACTOR 1; LAMININ; LUCIFERASE; NAKED DNA; RETROVIRUS VECTOR; TRANSPOSASE;

BLEEDING DISORDER; COMPLEX FORMATION; DNA BINDING; DNA CONFORMATION; DNA FOOTPRINTING; DNA INTEGRATION; DNA STRUCTURE; DNA TRANSPOSITION; GENE EXPRESSION; GENE INSERTION; GENE TARGETING; GENE TECHNOLOGY; GENE THERAPY; GENE VECTOR; GENETIC MANIPULATION; GENETIC STABILITY; GERM LINE; HEMOPHILIA B; HUMAN; MOUSE; MUTAGENESIS; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NONVIRAL GENE THERAPY; PARASITE; REVIEW; TRANSGENICS; TRANSPOSON; VIRAL GENE DELIVERY SYSTEM;

INVERTEBRATA; TRANSPOSONS; VERTEBRATA;

EID: 1342311458     PISSN: 15250016     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ymthe.2003.11.009     Document Type: Review

References (82)

1. Dobbelstein, M. (2003). Viruses in therapy - royal road or dead end? Virus Res. 92: 219-221.
2. Thomas, C. E., Ehrhardt, A., and Kay, M. A. (2003). Progress and problems with the use of viral vectors for gene therapy. Nat. Rev. Genet. 4: 346-358.
3. Gene therapy clinical trials. J. Gene Med. http://www.wiley.co.uk/wileychi/genmed/clinical/.
4. Check, E. (2002). A tragic setback. Nature 420: 116-118.
5. Abdallah, B., Sachs, L., and Demeneix, B. A. (1995). Non-viral gene transfer: applications in developmental biology and gene therapy. Biol. Cell 85: 1-7.
6. Herweijer, H., and Wolff, J. A. (2003). Progress and prospects: naked DNA gene transfer and therapy. Gene Ther. 10: 453-458.
7. Niidome, T., and Huang, L. (2002). Gene therapy progress and prospects: nonviral vectors. Gene Ther. 9: 1647-1652.
8. Hartl, D. L., Lozovskaya, E. R., Nurminsky, D. I., and Lohe, A. R. (1997). What restricts the activity of mariner-like transposable elements? Trends Genet. 13: 197-201.
9. Moran, J. V., and Gilbert, N. (2002). Mammalian LINE-1 retrotransposons and related elements. In: Mobile DNA, vol. II (N. L. Craig, R. Craigie, M. Gellert, A. M. Lambowitz, Eds.), (2002). pp. 836-869. Washington, DC: ASM Press.
10. International Human Genome Sequencing Consortium (2001). Initial sequencing and analysis of the human genome. Nature 409: 860-921.
11. Brouha, B., et al. (2003). Hot L1s account for the bulk of retrotransposition in the human population. Proc. Natl. Acad. Sci. USA 100: 5280-5285.
12. Kazazian, H. H. (1999). An estimated frequency of endogenous insertional mutations in humans. Nat. Genet. 22: 130.
13. Dewannieux, M., Esnault, C., and Heidmann, T. (2003). LINE-mediated retrotransposition of marked Alu sequences. Nat. Genet. 35: 41-48.
14. Deininger, P. L., and Batzer, M. A. (1999). Alu repeats and human disease. Mol. Genet. Metab. 67: 183-193.
15. Cooley, L., Kelley, R., and Spradling, A. (1988). Insertional mutagenesis of the Drosophila genome with single P elements. Science 239: 1121-1128.
16. Plasterk, R. H. (1996). The Tc1/mariner transposon family. Curr. Top. Microbiol. Immunol. 204: 125-143.
17. Ivics, Z., Izsvák, Z., Minter, A., and Hackett, P. B. (1996). Identification of functional domains and evolution of Tc1-like transposable elements. Proc. Natl. Acad. Sci. USA 93: 5008-5013.
18. Rio, D. C., Barnes, G., Laski, F. A., Rine, J., and Rubin, G. M. (1988). Evidence for Drosophila P element transposase activity in mammalian cells and yeast. J. Mol. Biol. 200: 411-415.
19. Raz, E., van Luenen, H. G., Schaerringer, B., Plasterk, R. H. A., and Driever, W. (1998). Transposition of the nematode Caenorhabditis elegans Tc3 element in the zebrafish Danio rerio. Curr. Biol. 8: 82-88.
20. Zagoraiou, L., et al. (2001). In vivo transposition of Minos, a Drosophila mobile element, in mammalian tissues. Proc. Natl. Acad. Sci. USA 98: 11474-11478.
21. Ivics, Z., Hackett, P. B., Plasterk, R. H., and Izsvák, Z. (1997). Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells. Cell 91: 501-510.
22. Fischer, S. E., Wienholds, E., and Plasterk, R. H. (2001). Regulated transposition of a fish transposon in the mouse germ line. Proc. Natl. Acad. Sci. USA 98: 6759-6764.
23. Plasterk, R. H., Izsvák, Z., and Ivics, Z. (1999). Resident aliens: the Tc1/mariner superfamily of transposable elements. Trends Genet. 15: 326-332.
24. Mikkelsen, J. G., et al. (2003). Helper-independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo. Mol. Ther. 8: 654-665.
25. Izsvák, Z., Ivics, Z., and Plasterk, R. H. (2000). Sleeping Beauty, a wide host-range transposon vector for genetic transformation in vertebrates. J. Mol. Biol. 302: 93-102.
26. Dupuy, A. J., et al. (2002). Mammalian germ-line transgenesis by transposition. Proc. Natl. Acad. Sci. USA 99: 4495-4499.
27. Izsvák, Z., et al. (2002). Involvement of a bifunctional, paired-like DNA-binding domain and a transpositional enhancer in Sleeping Beauty transposition. J. Biol. Chem. 277: 34581-34588.
28. Doak, T. G., Doerder, F. P., Jahn, C. L., and Herrick, G. (1994). A proposed superfamily of transposase genes: transposon-like elements in ciliated protozoa and a common "D35E" motif. Proc. Natl. Acad. Sci. USA 91: 942-946.
29. Katzman, M., and Sudol, M. (1995). Mapping domains of retroviral integrase responsible for viral DNA specificity and target site selection by analysis of chimeras between human immunodeficiency virus type 1 and visna virus integrases. J. Virol. 69: 5687-5696.
30. Junop, M. S., and Haniford, D. B. (1997). Factors responsible for target site selection in Tn10 transposition: a role for the DDE motif in target DNA capture. EMBO J. 16: 2646-2655.
31. van Luenen, H. G., Colloms, S. D., and Plasterk, R. H. (1994). The mechanism of transposition of Tc3 in C. elegans. Cell 79: 293-301.
32. Smit, A. F., and Riggs, A. D. (1996). Tiggers and DNA transposon fossils in the human genome. Proc. Natl. Acad. Sci. USA 93: 1443-1448.
33. Robertson, H. M. (1996). Members of the pogo superfamily of DNA-mediated transposons in the human genome. Mol. Gen. Genet. 252: 761-7661.
34. Robertson, H. M., and Martos, R. (1997). Molecular evolution of the second ancient human mariner transposon, Hsmar2, illustrates patterns of neutral evolution in the human genome. Gene 205: 219-228.
35. Robertson, H. M., and Zumpano, K. L. (1997). Molecular evolution of an ancient mariner transposon, Hsmar1, in the human genome. Gene 205: 203-217.
36. Zayed, H., Izsvák, Z., Khare, D., Heinemann, U., and Ivics, Z. (2003). The DNA-bending protein HMGB1 is a cellular cofactor of Sleeping Beauty transposition. Nucleic Acids Res. 31: 2313-2322.
37. Bustin, M. (1999). Regulation of DNA-dependent activities by the functional motifs of the high-mobility-group chromosomal proteins. Mol. Cell. Biol. 19: 5237-5246.
38. Costello, E., Saudan, P., Winocour, E., Pizer, L., and Beard, P. (1997). High mobility group chromosomal protein 1 binds to the adeno-associated virus replication protein (Rep) and promotes Rep-mediated site-specific cleavage of DNA, ATPase activity and transcriptional repression. EMBO J. 17: 5943-5954.
39. Li, L., et al. (2000). Retroviral cDNA integration: stimulation by HMG I family proteins. J. Virol. 74: 10965-10974.
40. Geurts, A. M., et al. (2003). Gene transfer into genomes of human cells by the Sleeping Beauty transposon system. Mol. Ther. 8: 108-117.
41. Berg, D. E., Berg, C. M., Sasakawa, C., Zhou, M., and Reznikoff, W. S. (1984). Bacterial transposon Tn5: evolutionary inferences. Mol. Biol. Evol. 1: 411-422.
42. Kleckner, N., Chalmers, R. M., Kwon, D., Sakai, J., and Bolland, S. (1996). Tn10 and IS10 transposition and chromosome rearrangements: mechanism and regulation in vivo and in vitro. Curr. Top. Microbiol. Immunol. 204: 49-82.
43. Dupuy, A. J., Fritz, S., and Largaespada, D. A. (2001). Transposition and gene disruption in the male germline of the mouse. Genesis 30: 82-88.
44. Luo, G., Ivics, Z., Izsvák, Z., and Bradley, A. (1998). Chromosomal transposition of a Tc1/mariner-like element in mouse embryonic stem cells. Proc. Natl. Acad. Sci. USA 95: 10769-10773.
45. Vigdal, T. J., Kaufman, C. D., Izsvák, Z., Voytas, D. F., and Ivics, Z. (2002). Common physical properties of DNA affecting target site selection of Sleeping Beauty and other Tc1/mariner transposable elements. J. Mol. Biol. 323: 441-452.
46. Craig, N. L. (1997). Target site selection in transposition. Annu. Rev. Biochem. 66: 437-474.
47. Kirchner, J., Connolly, C. M., and Sandmeyer, S. B. (1995). Requirement of RNA polymerase III transcription factors for in vitro position-specific integration of a retroviruslike element. Science 267: 1488-1491.
48. Zou, S., Ke, N., Kim, J. M., and Voytas, D. F. (1996). The Saccharomyces retrotransposon Ty5 integrates preferentially into regions of silent chromatin at the telomeres and mating loci. Genes Dev. 10: 634-645.
49. van Luenen, H. G., and Plasterk, R. H. (1994). Target site choice of the related transposable elements Tc1 and Tc3 of Caenorhabditis elegans. Nucleic Acids Res. 22: 262-269.
50. Yant, S. R., et al. (2000). Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system. Nat. Genet. 25: 35-41.
51. Belur, L. R., et al. (2003). Gene insertion and long-term expression in lung mediated by the Sleeping Beauty transposon system. Mol. Ther. 8: 501-507.
52. Horie, K., et al. (2001). Efficient chromosomal transposition of a Tc1/mariner-like transposon Sleeping Beauty in mice. Proc. Natl. Acad. Sci. USA 98: 9191-9196.
53. Montini, E., et al. (2002). In vivo correction of murine tyrosinemia type I by DNA-mediated transposition. Mol. Ther. 6: 759-769.
54. Ortiz-Urda, S., et al. (2003). Sustainable correction of junctional epidermolysis bullosa via transposon-mediated nonviral gene transfer. Gene Ther. 10: 1099-1104.
55. Scherdin, U., Rhodes, K., and Breindl, M. (1990). Transcriptionally active genome regions are preferred targets for retrovirus integration. J. Virol. 64: 907-912.
56. Schroder, A. R., et al. (2002). HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110: 521-529.
57. Nakai, H., et al. (2003). AAV serotype 2 vectors preferentially integrate into active genes in mice. Nat. Genet. 34: 297-302.
58. Miller, D. G., Rutledge, E. A., and Russell, D. W. (2002). Chromosomal effects of adenoassociated virus vector integration. Nat. Genet. 30: 147-148.
59. Wiegand, T. W., and Reznikoff, W. S. (1992). Characterization of two hypertransposing Tn5 mutants. J. Bacteriol. 174: 1229-1239.
60. Kunze, R., et al. (1993). Dominant transposition-deficient mutants of maize Activator (Ac) transposase. Proc. Natl. Acad. Sci. USA 90: 7094-7098.
61. Lampe, D. J., Grant, T. E., and Robertson, H. M. (11998). Factors affecting transposition of the Himar1 mariner transposon in vitro. Genetics 149: 179-187.
62. Lohe, A. R., and Hartl, D. L. (1996). Autoregulation of mariner transposase activity by overproduction and dominant-negative complementation. Mol. Biol. Evol. 13: 549-555.
63. Vos, J. C., van Luenen, H. G., and Plasterk, R. H. (1993). Characterization of the Caenorhabditis elegans Tc1 transposase in vivo and in vitro. Genes Dev. 7: 1244-1253.
64. Braam, L. A., Goryshin, I. Y., and Reznikoff, W. S. (1999). A mechanism for Tn5 inhibition: carboxyl-terminal dimerization. J. Biol. Chem. 274: 86-92.
65. Simmons, M. J., and Bucholz, L. M. (1985). Transposase titration in Drosophila melanogaster: a model of cytotype in the P-M system of hybrid dysgenesis. Proc. Natl. Acad. Sci. USA 82: 8119-8123.
66. Lin, M. T., Pulkkinen, L., Uitto, J., and Yoon, K. (2000). The gene gun: current applications in cutaneous gene therapy. Int. J. Dermatol. 39: 161-170.
67. Kumar, V. V., Singh, R. S., and Chaudhuri, A. (2003). Cationic transfection lipids in gene therapy: successes, set-backs, challenges and promises. Curr. Med. Chem. 10: 1297-1306.
68. Lemkine, G. F., and Demeneix, B. A. (2001). Polyethylenimines for in vivo gene delivery. Curr. Opin. Mol. Ther. 3: 178-182.
69. Yant, S. R., et al. (2002). Transposition from a gutless adeno-transposon vector stabilizes transgene expression in vivo. Nat. Biotechnol. 20: 999-1005.
70. Lieber, A., Steinwaerder, D. S., Carlson, C. A., and Kay, M. A. (1999). Integrating adenovirus-adeno-associated virus hybrid vectors devoid of all viral genes. J. Virol. 73: 9314-9324.
71. Soifer, H., et al. (2001). Stable integration of transgenes delivered by a retrotransposon-adenovirus hybrid vector. Hum. Gene Ther. 12: 1417-1428.
72. Plasterk, R. H., and Ketting, R. F. (2000). The silence of the genes. Curr. Opin. Genet. Dev. 10: 562-567.
73. Garrick, D., Fiering, S., Martin, D. I., and Whitelaw, E. (1998). Repeat-induced gene silencing in mammals. Nat. Genet. 18: 56-59.
74. Henikoff, S. (1998). Conspiracy of silence among repeated transgenes. Bioessays 20: 532-535.
75. Koprek, T., et al. (2001). Transposon-mediated single-copy gene delivery leads to increased transgene expression stability in barley. Plant Physiol. 125: 1354-1362.
76. Hacein-Bey-Abina, S., et al. (2003). A serious adverse event after successful gene therapy for X-linked severe combined immunodeficiency. N. Engl. J. Med. 348: 255-256.
77. Hacein-Bey-Abina, S., et al. (2003). LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302: 415-419.
78. Ye, X., et al. (2003). Insulation from viral transcriptional regulatory elements enables improvement to hepatoma-specific gene expression from adenovirus vectors. Biochem. Biophys. Res. Commun. 307: 759-764.
79. Emery, D. W., et al. (2000). A chromatin insulator protects retrovirus vectors from chromosomal position effects. Proc. Natl. Acad. Sci. USA 97: 9150-9155.
80. Zhu, Y., Dai, J., Fuerst, P. G., and Voytas, D. F. (2003). Controlling integration specificity of a yeast retrotransposon. Proc. Natl. Acad. Sci. USA 100: 5891-5895.
81. Portlock, J. L., et al. (2003). Site-specific genomic strategies for gene therapy. Curr. Opin. Mol. Ther. 5: 376-382.
82. Miskey, C., Izsvk, Z., Plasterk, R. H., and Ivics, Z. (2003). The Frog Prince: a reconstructed transposon from Rana pipiens with high transpositional activity in vertebrate cells. Nucleic Acids Res. 31: 6873-6881.