Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates (original) (raw)

• Ivics, Z., Hackett, P.B., Plasterk, R.H. & Izsvák, Z. Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells. Cell 91, 501–510 (1997).
  Article CAS Google Scholar
• Mates, L., Izsvak, Z. & Ivics, Z. Technology transfer from worms and flies to vertebrates: transposition-based genome manipulations and their future perspectives. Genome Biol. 8 (Suppl 1), S1 (2007).
  Article Google Scholar
• Ivics, Z. & Izsvak, Z. Transposons for gene therapy! Curr. Gene Ther. 6, 593–607 (2006).
  Article CAS Google Scholar
• Geurts, A.M. et al. Gene transfer into genomes of human cells by the Sleeping Beauty transposon system. Mol. Ther. 8, 108–117 (2003).
  Article CAS Google Scholar
• Zayed, H., Izsvak, Z., Walisko, O. & Ivics, Z. Development of hyperactive Sleeping Beauty transposon vectors by mutational analysis. Mol. Ther. 9, 292–304 (2004).
  Article CAS Google Scholar
• Baus, J., Liu, L., Heggestad, A.D., Sanz, S. & Fletcher, B.S. Hyperactive transposase mutants of the Sleeping Beauty transposon. Mol. Ther. 12, 1148–1156 (2005).
  Article CAS Google Scholar
• Yant, S.R., Park, J., Huang, Y., Mikkelsen, J.G. & Kay, M.A. 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. 24, 9239–9247 (2004).
  Article CAS Google Scholar
• Cadinanos, J. & Bradley, A. Generation of an inducible and optimized piggyBac transposon system. Nucleic Acids Res. 35, e87 (2007).
  Article Google Scholar
• Wilson, M.H., Coates, C.J. & George, A.L. Jr. PiggyBac Transposon-mediated Gene Transfer in Human Cells. Mol. Ther. 15, 139–145 (2007).
  Article CAS Google Scholar
• Wang, W. et al. Chromosomal transposition of PiggyBac in mouse embryonic stem cells. Proc. Natl. Acad. Sci. USA 105, 9290–9295 (2008).
  Article CAS Google Scholar
• Izsvak, Z. & Ivics, Z. Sleeping beauty transposition: biology and applications for molecular therapy. Mol. Ther. 9, 147–156 (2004).
  Article CAS Google Scholar
• Liu, G., Aronovich, E.L., Cui, Z., Whitley, C.B. & Hackett, P.B. Excision of Sleeping Beauty transposons: parameters and applications to gene therapy. J. Gene Med. 6, 574–583 (2004).
  Article CAS Google Scholar
• Mingozzi, F. et al. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. J. Clin. Invest. 111, 1347–1356 (2003).
  Article CAS Google Scholar
• Conner, D.A. Transgenic mouse production by zygote injection. Curr. Protoc. Mol. Biol. Chapter 23, Unit 23.9 (2004).
  PubMed Google Scholar
• Dupuy, A.J. et al. Mammalian germ-line transgenesis by transposition. Proc. Natl. Acad. Sci. USA 99, 4495–4499 (2002).
  Article CAS Google Scholar
• Horie, K. et al. Characterization of Sleeping Beauty transposition and its application to genetic screening in mice. Mol. Cell. Biol. 23, 9189–9207 (2003).
  Article CAS Google Scholar
• Kitada, K. et al. Transposon-tagged mutagenesis in the rat. Nat. Methods 4, 131–133 (2007).
  Article CAS Google Scholar
• Lu, B. et al. Generation of rat mutants using a coat color-tagged Sleeping Beauty transposon system. Mamm. Genome 18, 338–346 (2007).
  Article CAS Google Scholar
• Dupuy, A.J., Fritz, S. & Largaespada, D.A. Transposition and gene disruption in the male germline of the mouse. Genesis 30, 82–88 (2001).
  Article CAS Google Scholar
• Yusa, K., Takeda, J. & Horie, K. Enhancement of Sleeping Beauty transposition by CpG methylation: possible role of heterochromatin formation. Mol. Cell. Biol. 24, 4004–4018 (2004).
  Article CAS Google Scholar
• Geurts, A.M. et al. Gene mutations and genomic rearrangements in the mouse as a result of transposon mobilization from chromosomal concatemers. PLoS Genet. 2, e156 (2006).
  Article Google Scholar
• Manno, C.S. et al. Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat. Med. 12, 342–347 (2006).
  Article CAS Google Scholar
• Hollis, R.P. et al. Stable gene transfer to human CD34(+) hematopoietic cells using the Sleeping Beauty transposon. Exp. Hematol. 34, 1333–1343 (2006).
  Article CAS Google Scholar
• Conneally, E., Eaves, C.J. & Humphries, R.K. Efficient retroviral-mediated gene transfer to human cord blood stem cells with in vivo repopulating potential. Blood 91, 3487–3493 (1998).
  CAS PubMed Google Scholar
• Miyoshi, H., Smith, K.A., Mosier, D.E., Verma, I.M. & Torbett, B.E. Transduction of human CD34+ cells that mediate long-term engraftment of NOD/SCID mice by HIV vectors. Science 283, 682–686 (1999).
  Article CAS Google Scholar
• Salmon, P. et al. High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors. Blood 96, 3392–3398 (2000).
  CAS PubMed Google Scholar
• Follenzi, A., Ailles, L.E., Bakovic, S., Geuna, M. & Naldini, L. Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat. Genet. 25, 217–222 (2000).
  Article CAS Google Scholar
• VandenDriessche, T. 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 100, 813–822 (2002).
  Article CAS Google Scholar
• Zufferey, R., Donello, J.E., Trono, D. & Hope, T.J. Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J. Virol. 73, 2886–2892 (1999).
  CAS PubMed PubMed Central Google Scholar
• Piacibello, W. et al. Lentiviral gene transfer and ex vivo expansion of human primitive stem cells capable of primary, secondary, and tertiary multilineage repopulation in NOD/SCID mice. Nonobese diabetic/severe combined immunodeficient. Blood 100, 4391–4400 (2002).
  Article CAS Google Scholar
• Bushman, F. et al. Genome-wide analysis of retroviral DNA integration. Nat. Rev. Microbiol. 3, 848–858 (2005).
  Article CAS Google Scholar
• Schroder, A.R. et al. HIV-1 integration in the human genome favors active genes and local hotspots. Cell 110, 521–529 (2002).
  Article CAS Google Scholar
• Liu, G. et al. Target-site preferences of Sleeping Beauty transposons. J. Mol. Biol. 346, 161–173 (2005).
  Article CAS Google Scholar
• Vigdal, T.J., Kaufman, C.D., Izsvák, Z., Voytas, D.F. & Ivics, Z. Common physical properties of DNA affecting target site selection of Sleeping Beauty and other Tc1/mariner transposable elements. J. Mol. Biol. 323, 441–452 (2002).
  Article CAS Google Scholar
• Ivics, Z. et al. Targeted Sleeping Beauty transposition in human cells. Mol. Ther. 15, 1137–1144 (2007).
  Article CAS Google Scholar
• Yant, S.R., Huang, Y., Akache, B. & Kay, M.A. Site-directed transposon integration in human cells. Nucleic Acids Res. 35, e50 (2007).
  Article Google Scholar
• Walisko, O. et al. Transcriptional activities of the Sleeping Beauty transposon and shielding its genetic cargo with insulators. Mol. Ther. 16, 359–369 (2008).
  Article CAS Google Scholar
• Dalsgaard, T. et al. Shielding of sleeping beauty DNA transposon-delivered transgene cassettes by heterologous insulators in early embryonal cells. Mol. Ther. 17, 121–130 (2009).
  Article CAS Google Scholar
• Mikkelsen, J.G. et al. Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo. Mol. Ther. 8, 654–665 (2003).
  Article CAS Google Scholar
• Ohlfest, J.R. et al. 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 105, 2691–2698 (2005).
  Article CAS Google Scholar
• Yant, S.R. et al. Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system. Nat. Genet. 25, 35–41 (2000).
  Article CAS Google Scholar
• Stemmer, W.P. DNA shuffling by random fragmentation and reassembly: in vitro recombination for molecular evolution. Proc. Natl. Acad. Sci. USA 91, 10747–10751 (1994).
  Article CAS Google Scholar
• Miao, C.H. et al. Inclusion of the hepatic locus control region, an intron, and untranslated region increases and stabilizes hepatic factor IX gene expression in vivo but not in vitro. Mol. Ther. 1, 522–532 (2000).
  Article CAS Google Scholar
• Ishikawa, F. et al. Development of functional human blood and immune systems in NOD/SCID/IL2 receptor {gamma} chain(null) mice. Blood 106, 1565–1573 (2005).
  Article CAS Google Scholar
• Nagy, A., Gertsenstein, M., Vintersten, K. & Behringer, R. Manipulating the Mouse Embryo: A Laboratory Manual (Cold Spring Harbor Laboratory Press, Cold Spring Harbor, New York, 2003).
  Google Scholar
• Moore, J.C., Jin, H.M., Kuchner, O. & Arnold, F.H. Strategies for the in vitro evolution of protein function: enzyme evolution by random recombination of improved sequences. J. Mol. Biol. 272, 336–347 (1997).
  Article CAS Google Scholar