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Volumn 8, Issue 10, 2013, Pages

Quantitative Analysis of α-L-Iduronidase Expression in Immunocompetent Mice Treated with the Sleeping Beauty Transposon System

Author keywords

[No Author keywords available]

Indexed keywords

CYCLOPHOSPHAMIDE; DNA; LEVO IDURONIDASE; SB100X PROTEIN; TRANSPOSASE; UNCLASSIFIED DRUG;

EID: 84886917002     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0078161     Document Type: Article
Times cited : (18)

References (34)
  • 1
    • 0030662074 scopus 로고    scopus 로고
    • Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells
    • doi: 10.1016/S0092-8674(00)80436-5
    • Ivics Z, Hackett PB, Plasterk RH, Izsvák Z, (1997) Molecular reconstruction of Sleeping Beauty, a Tc1-like transposon from fish, and its transposition in human cells. Cell 91: 501-510. doi:10.1016/S0092-8674(00)80436-5. PubMed: 9390559.
    • (1997) Cell , vol.91 , pp. 501-510
    • Ivics, Z.1    Hackett, P.B.2    Plasterk, R.H.3    Izsvák, Z.4
  • 2
    • 77956668327 scopus 로고    scopus 로고
    • Translating Sleeping Beauty transposition into cellular therapies: victories and challenges
    • doi: 10.1002/bies.201000027
    • Izsvák Z, Hackett PB, Cooper LJ, Ivics Z, (2010) Translating Sleeping Beauty transposition into cellular therapies: victories and challenges. Bioessays 32: 756-767. doi:10.1002/bies.201000027. PubMed: 20652893.
    • (2010) Bioessays , vol.32 , pp. 756-767
    • Izsvák, Z.1    Hackett, P.B.2    Cooper, L.J.3    Ivics, Z.4
  • 3
    • 79955990614 scopus 로고    scopus 로고
    • The Sleeping Beauty transposon system: a non-viral vector for gene therapy
    • doi: 10.1093/hmg/ddr140
    • Aronovich EL, McIvor RS, Hackett PB, (2011) The Sleeping Beauty transposon system: a non-viral vector for gene therapy. Hum Mol Genet 20(R1):: 14-20. doi:10.1093/hmg/ddr140.
    • (2011) Hum Mol Genet , vol.20 , pp. 14-20
    • Aronovich, E.L.1    McIvor, R.S.2    Hackett, P.B.3
  • 4
    • 79955998860 scopus 로고    scopus 로고
    • Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies
    • doi: 10.2174/156652311797415827
    • Hackett PB Jr, Aronovich EL, Hunter D, Urness M, Bell JB, et al. (2011) Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies. Curr Gene Ther 11: 341-349. doi:10.2174/156652311797415827. PubMed: 21888621.
    • (2011) Curr Gene Ther , vol.11 , pp. 341-349
    • Hackett Jr., P.B.1    Aronovich, E.L.2    Hunter, D.3    Urness, M.4    Bell, J.B.5
  • 5
    • 77950525914 scopus 로고    scopus 로고
    • A transposon and transposase system for human application
    • doi: 10.1038/mt.2010.2
    • Hackett PB, Largaespada DA, Cooper LJN, (2010) A transposon and transposase system for human application. Mol Ther 18: 674-683. doi:10.1038/mt.2010.2. PubMed: 20104209.
    • (2010) Mol Ther , vol.18 , pp. 674-683
    • Hackett, P.B.1    Largaespada, D.A.2    Cooper, L.J.N.3
  • 6
    • 0034103009 scopus 로고    scopus 로고
    • Somatic integration and long-term transgene expression in normal and haemophilic mice using a DNA transposon system
    • doi: 10.1038/75568
    • Yant SR, Meuse L, Chiu W, Ivics Z, Izsvak Z, 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. doi:10.1038/75568. PubMed: 10802653.
    • (2000) Nat Genet , vol.25 , pp. 35-41
    • Yant, S.R.1    Meuse, L.2    Chiu, W.3    Ivics, Z.4    Izsvak, Z.5
  • 7
    • 34249090542 scopus 로고    scopus 로고
    • Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses
    • doi: 10.1002/jgm.1028
    • Aronovich EL, Bell JB, Belur LR, Gunther R, Koniar B, et al. (2007) Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses. J Gene Med 9: 403-415. doi:10.1002/jgm.1028. PubMed: 17407189.
    • (2007) J Gene Med , vol.9 , pp. 403-415
    • Aronovich, E.L.1    Bell, J.B.2    Belur, L.R.3    Gunther, R.4    Koniar, B.5
  • 8
    • 67649844281 scopus 로고    scopus 로고
    • Systemic correction of storage disease in MPS I NOD/SCID mice using the Sleeping Beauty transposon system
    • doi: 10.1038/mt.2009.87
    • Aronovich EL, Bell JB, Khan SA, Belur LR, Gunther R, et al. (2009) Systemic correction of storage disease in MPS I NOD/SCID mice using the Sleeping Beauty transposon system. Mol Ther 17: 1136-1144. doi:10.1038/mt.2009.87. PubMed: 19384290.
    • (2009) Mol Ther , vol.17 , pp. 1136-1144
    • Aronovich, E.L.1    Bell, J.B.2    Khan, S.A.3    Belur, L.R.4    Gunther, R.5
  • 9
    • 0041315884 scopus 로고    scopus 로고
    • Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo
    • doi: 10.1016/S1525-0016(03)00168-0
    • Chen ZY, He CY, Ehrhardt A, Kay MA, (2003) Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther 8: 495-500. doi:10.1016/S1525-0016(03)00168-0. PubMed: 12946323.
    • (2003) Mol Ther , vol.8 , pp. 495-500
    • Chen, Z.Y.1    He, C.Y.2    Ehrhardt, A.3    Kay, M.A.4
  • 11
    • 10744227295 scopus 로고    scopus 로고
    • Healing the wounds inflicted by Sleeping Beauty transposition by double-strand break repair in mammalian somatic cells
    • doi: 10.1016/S1097-2765(03)00524-0
    • Izsvák Z, Stüwe EE, Fiedler D, Katzer A, Jeggo PA, et al. (2004) Healing the wounds inflicted by Sleeping Beauty transposition by double-strand break repair in mammalian somatic cells. Mol Cell 13: 279-290. doi:10.1016/S1097-2765(03)00524-0. PubMed: 14759372.
    • (2004) Mol Cell , vol.13 , pp. 279-290
    • Izsvák, Z.1    Stüwe, E.E.2    Fiedler, D.3    Katzer, A.4    Jeggo, P.A.5
  • 12
    • 0033179759 scopus 로고    scopus 로고
    • Resident aliens: the Tc1/mariner superfamily of transposable elements
    • doi: 10.1016/S0168-9525(99)01777-1
    • Plasterk RHA, Izsvák Z, Ivics Z, (1999) Resident aliens: the Tc1/mariner superfamily of transposable elements. Trends Genet 15: 326-332. doi:10.1016/S0168-9525(99)01777-1. PubMed: 10431195.
    • (1999) Trends Genet , vol.15 , pp. 326-332
    • Plasterk, R.H.A.1    Izsvák, Z.2    Ivics, Z.3
  • 13
    • 0042700227 scopus 로고    scopus 로고
    • Gene transfer into genomes of human cells by the Sleeping Beauty transposon system
    • doi: 10.1016/S1525-0016(03)00099-6
    • Geurts AM, Yang Y, Clark KJ, Liu G, Cui Z, et al. (2003) Gene transfer into genomes of human cells by the Sleeping Beauty transposon system. Mol Ther 8: 108-117. doi:10.1016/S1525-0016(03)00099-6. PubMed: 12842434.
    • (2003) Mol Ther , vol.8 , pp. 108-117
    • Geurts, A.M.1    Yang, Y.2    Clark, K.J.3    Liu, G.4    Cui, Z.5
  • 14
    • 4744374701 scopus 로고    scopus 로고
    • Mutational analysis of the N-terminal DNA-binding domain of Sleeping Beauty transposase: critical residues for DNA binding and hyperactivity in mammalian cells
    • doi: 10.1128/MCB.24.20.9239-9247.2004
    • Yant SR, Park J, Huang Y, Mikkelsen JG, Kay MA, (2004) 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. doi:10.1128/MCB.24.20.9239-9247.2004. PubMed: 15456893.
    • (2004) Mol Cell Biol , vol.24 , pp. 9239-9247
    • Yant, S.R.1    Park, J.2    Huang, Y.3    Mikkelsen, J.G.4    Kay, M.A.5
  • 15
    • 67349250885 scopus 로고    scopus 로고
    • Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates
    • doi: 10.1038/ng.343
    • Mátés L, Chuah MK, Belay E, Jerchow B, Manoj N, et al. (2009) Molecular evolution of a novel hyperactive Sleeping Beauty transposase enables robust stable gene transfer in vertebrates. Nat Genet 41: 753-761. doi:10.1038/ng.343. PubMed: 19412179.
    • (2009) Nat Genet , vol.41 , pp. 753-761
    • Mátés, L.1    Chuah, M.K.2    Belay, E.3    Jerchow, B.4    Manoj, N.5
  • 16
    • 0036317929 scopus 로고    scopus 로고
    • Structure-function analysis of the inverted terminal repeats of the Sleeping Beauty transposon
    • doi: 10.1016/S0022-2836(02)00237-1
    • Cui Z, Geurts AM, Liu G, Kaufman CD, Hackett PB, (2002) Structure-function analysis of the inverted terminal repeats of the Sleeping Beauty transposon. J Mol Biol 318: 1221-1235. doi:10.1016/S0022-2836(02)00237-1. PubMed: 12083513.
    • (2002) J Mol Biol , vol.318 , pp. 1221-1235
    • Cui, Z.1    Geurts, A.M.2    Liu, G.3    Kaufman, C.D.4    Hackett, P.B.5
  • 17
    • 1342290203 scopus 로고    scopus 로고
    • Development of hyperactive Sleeping Beauty transposon vectors by mutational analysis
    • doi: 10.1016/j.ymthe.2004.06.690
    • Zayed H, Izsvák Z, Walisko O, Ivics Z, (2004) Development of hyperactive Sleeping Beauty transposon vectors by mutational analysis. Mol Ther 9: 292-304. doi:10.1016/j.ymthe.2004.06.690. PubMed: 14759813.
    • (2004) Mol Ther , vol.9 , pp. 292-304
    • Zayed, H.1    Izsvák, Z.2    Walisko, O.3    Ivics, Z.4
  • 18
    • 84860162273 scopus 로고    scopus 로고
    • The Sleeping Beauty transposon toolbox
    • doi: 10.1007/978-1-61779-603-6_13
    • Ammar I, Izsvák Z, Ivics Z, (2012) The Sleeping Beauty transposon toolbox. Methods Mol Biol 859: 229-240. doi:10.1007/978-1-61779-603-6_13. PubMed: 22367875.
    • (2012) Methods Mol Biol , vol.859 , pp. 229-240
    • Ammar, I.1    Izsvák, Z.2    Ivics, Z.3
  • 19
    • 77957552575 scopus 로고    scopus 로고
    • Duration of expression and activity of Sleeping Beauty transposase in mouse liver following hydrodynamic DNA delivery
    • doi: 10.1038/mt.2010.152
    • Bell JB, Aronovich EL, Schreifels JM, Beadnell TC, Hackett PB, (2010) Duration of expression and activity of Sleeping Beauty transposase in mouse liver following hydrodynamic DNA delivery. Mol Ther 18: 1796-1802. doi:10.1038/mt.2010.152. PubMed: 20628359.
    • (2010) Mol Ther , vol.18 , pp. 1796-1802
    • Bell, J.B.1    Aronovich, E.L.2    Schreifels, J.M.3    Beadnell, T.C.4    Hackett, P.B.5
  • 20
    • 69949184405 scopus 로고    scopus 로고
    • Strategies to modulate immune responses: a new frontier for gene therapy
    • doi: 10.1038/mt.2009.150
    • Arruda VR, Favaro P, Finn JD, (2009) Strategies to modulate immune responses: a new frontier for gene therapy. Mol Ther 17: 1492-1503. doi:10.1038/mt.2009.150. PubMed: 19584819.
    • (2009) Mol Ther , vol.17 , pp. 1492-1503
    • Arruda, V.R.1    Favaro, P.2    Finn, J.D.3
  • 21
    • 0030008178 scopus 로고    scopus 로고
    • Liver-directed gene therapy: a retroviral vector with a complete LTR and the ApoE enhancer-alpha 1-antitrypsin promoter dramatically increases expression of human alpha 1-antitrypsin in vivo
    • doi: 10.1089/hum.1996.7.5-637
    • Okuyama T, Huber RM, Bowling W, Pearline R, Kennedy SC, et al. (1996) Liver-directed gene therapy: a retroviral vector with a complete LTR and the ApoE enhancer-alpha 1-antitrypsin promoter dramatically increases expression of human alpha 1-antitrypsin in vivo. Hum Gene Ther 7: 637-645. doi:10.1089/hum.1996.7.5-637. PubMed: 8845389.
    • (1996) Hum Gene Ther , vol.7 , pp. 637-645
    • Okuyama, T.1    Huber, R.M.2    Bowling, W.3    Pearline, R.4    Kennedy, S.C.5
  • 22
    • 0034202522 scopus 로고    scopus 로고
    • 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
    • doi: 10.1006/mthe.2000.0075
    • Miao CH, Ohashi K, Patijn GA, Meuse L, Ye X, et al. (2000) 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. doi:10.1006/mthe.2000.0075. PubMed: 10933977.
    • (2000) Mol Ther , vol.1 , pp. 522-532
    • Miao, C.H.1    Ohashi, K.2    Patijn, G.A.3    Meuse, L.4    Ye, X.5
  • 23
    • 33644820684 scopus 로고    scopus 로고
    • Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response
    • doi: 10.1038/nm1358
    • Manno CS, Pierce GF, Arruda VR, Glader B, Ragni M, et al. (2006) Successful transduction of liver in hemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 12: 342-347. doi:10.1038/nm1358. PubMed: 16474400.
    • (2006) Nat Med , vol.12 , pp. 342-347
    • Manno, C.S.1    Pierce, G.F.2    Arruda, V.R.3    Glader, B.4    Ragni, M.5
  • 24
    • 9144274389 scopus 로고    scopus 로고
    • Excision of Sleeping Beauty transposons: parameters and applications to gene therapy
    • PubMed: 15133768
    • Liu G, Cui Z, Aronovich EL, Whitley CB, Hackett PB, (2003) Excision of Sleeping Beauty transposons: parameters and applications to gene therapy. J Gene Med 6: 574-583. PubMed: 15133768.
    • (2003) J Gene Med , vol.6 , pp. 574-583
    • Liu, G.1    Cui, Z.2    Aronovich, E.L.3    Whitley, C.B.4    Hackett, P.B.5
  • 25
    • 39849089752 scopus 로고    scopus 로고
    • Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation
    • doi: 10.1038/sj.mt.6300399
    • Chen ZY, Riu E, He CY, Xu H, Kay MA, (2008) Silencing of episomal transgene expression in liver by plasmid bacterial backbone DNA is independent of CpG methylation. Mol Ther 16: 548-556. doi:10.1038/sj.mt.6300399. PubMed: 18253155.
    • (2008) Mol Ther , vol.16 , pp. 548-556
    • Chen, Z.Y.1    Riu, E.2    He, C.Y.3    Xu, H.4    Kay, M.A.5
  • 26
    • 79952187756 scopus 로고    scopus 로고
    • Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I
    • doi: 10.1038/mt.2010.249
    • Osborn MJ, McElmurry RT, Lees CJ, DeFeo AP, Chen ZY, et al. (2011) Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I. Mol Ther 19: 450-460. doi:10.1038/mt.2010.249. PubMed: 21081900.
    • (2011) Mol Ther , vol.19 , pp. 450-460
    • Osborn, M.J.1    McElmurry, R.T.2    Lees, C.J.3    DeFeo, A.P.4    Chen, Z.Y.5
  • 27
    • 84871919467 scopus 로고    scopus 로고
    • Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level
    • doi: 10.1038/mt.2012.244
    • Gracey Maniar LE, Maniar JM, Chen ZY, Lu J, Fire AZ, et al. (2013) Minicircle DNA vectors achieve sustained expression reflected by active chromatin and transcriptional level. Mol Ther 21: 131-138. doi:10.1038/mt.2012.244. PubMed: 23183534.
    • (2013) Mol Ther , vol.21 , pp. 131-138
    • Gracey Maniar, L.E.1    Maniar, J.M.2    Chen, Z.Y.3    Lu, J.4    Fire, A.Z.5
  • 28
    • 84877015804 scopus 로고    scopus 로고
    • A Mini-intronic Plasmid (MIP): A Novel Robust Transgene Expression Vector In Vivo and In Vitro
    • doi: 10.1038/mt.2013.33
    • Lu J, Zhang F, Kay MA, (2013) A Mini-intronic Plasmid (MIP): A Novel Robust Transgene Expression Vector In Vivo and In Vitro. Mol Ther 21: 954-963. doi:10.1038/mt.2013.33. PubMed: 23459514.
    • (2013) Mol Ther , vol.21 , pp. 954-963
    • Lu, J.1    Zhang, F.2    Kay, M.A.3
  • 29
    • 32944458256 scopus 로고    scopus 로고
    • Sleeping Beauty-mediated transposition and long-term expression in vivo: use of the Lox/Cre recombinase system to distinguish transposition-specific expression
    • doi: 10.1016/j.ymthe.2005.10.015
    • Score PR, Belur LR, Frandsen JL, Geurts JL, Yamaguchi T, et al. (2006) Sleeping Beauty-mediated transposition and long-term expression in vivo: use of the Lox/Cre recombinase system to distinguish transposition-specific expression. Mol Ther 13: 617-624. doi:10.1016/j.ymthe.2005.10.015. PubMed: 16356773.
    • (2006) Mol Ther , vol.13 , pp. 617-624
    • Score, P.R.1    Belur, L.R.2    Frandsen, J.L.3    Geurts, J.L.4    Yamaguchi, T.5
  • 30
    • 0014468316 scopus 로고
    • Intraperitoneal injection of mice
    • PubMed: 5775909
    • Miner NA, Koehler J, Greenaway L, (1969) Intraperitoneal injection of mice. Appl Microbiol 17: 250-251. PubMed: 5775909.
    • (1969) Appl Microbiol , vol.17 , pp. 250-251
    • Miner, N.A.1    Koehler, J.2    Greenaway, L.3
  • 31
    • 34547585101 scopus 로고    scopus 로고
    • Implications of experimental technique for analysis and interpretation of data from animal experiments: outliers and increased variability resulting from failure of intraperitoneal injection procedures
    • doi: 10.1258/002367707781282802
    • Gaines Das R, North D, (2007) Implications of experimental technique for analysis and interpretation of data from animal experiments: outliers and increased variability resulting from failure of intraperitoneal injection procedures. Lab Anim 41: 312-320. doi:10.1258/002367707781282802. PubMed: 17640458.
    • (2007) Lab Anim , vol.41 , pp. 312-320
    • Gaines Das, R.1    North, D.2
  • 32
    • 77249179341 scopus 로고    scopus 로고
    • Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons
    • doi: 10.1089/hum.2009.128
    • Podetz-Pedersen KM, Bell JB, Steele TW, Wilber A, Shier WT, et al. (2010) Gene expression in lung and liver after intravenous infusion of polyethylenimine complexes of Sleeping Beauty transposons. Hum Gene Ther 21: 210-220. doi:10.1089/hum.2009.128. PubMed: 19761403.
    • (2010) Hum Gene Ther , vol.21 , pp. 210-220
    • Podetz-Pedersen, K.M.1    Bell, J.B.2    Steele, T.W.3    Wilber, A.4    Shier, W.T.5
  • 33
    • 39149118329 scopus 로고    scopus 로고
    • Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injection
    • doi: 10.1038/nprot.2007.471
    • Bell JB, Podetz-Pedersen KM, Aronovich EL, Belur LR, McIvor RS, et al. (2007) Preferential delivery of the Sleeping Beauty transposon system to livers of mice by hydrodynamic injection. Nat Protoc 2: 3153-3165. doi:10.1038/nprot.2007.471. PubMed: 18079715.
    • (2007) Nat Protoc , vol.2 , pp. 3153-3165
    • Bell, J.B.1    Podetz-Pedersen, K.M.2    Aronovich, E.L.3    Belur, L.R.4    McIvor, R.S.5
  • 34
    • 34848831191 scopus 로고    scopus 로고
    • Characterization of an immunodeficient mouse model of mucopolysaccharidosis type 1 suitable for preclinical testing of human stem cell and gene therapy
    • doi: 10.1016/j.brainresbull.2007.07.018
    • Garcia-Rivera MF, Colvin-Wanshura LE, Nelson MS, Nan Z, Khan SA, et al. (2007) Characterization of an immunodeficient mouse model of mucopolysaccharidosis type 1 suitable for preclinical testing of human stem cell and gene therapy. Brain Res Bullet 74: 429-438. doi:10.1016/j.brainresbull.2007.07.018.
    • (2007) Brain Res Bullet , vol.74 , pp. 429-438
    • Garcia-Rivera, M.F.1    Colvin-Wanshura, L.E.2    Nelson, M.S.3    Nan, Z.4    Khan, S.A.5


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.