메뉴 건너뛰기




Volumn 21, Issue 4, 2014, Pages 402-412

Intragenic integration in DLC1 sustains factor VIII expression in primary human cells without insertional oncogenicity

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; GENOMIC DNA; INTEGRASE; PHIC31 INTEGRASE; UNCLASSIFIED DRUG; DLC1 PROTEIN, HUMAN; GUANOSINE TRIPHOSPHATASE ACTIVATING PROTEIN; TUMOR SUPPRESSOR PROTEIN;

EID: 84897577406     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2014.11     Document Type: Article
Times cited : (3)

References (46)
  • 2
    • 19944421384 scopus 로고    scopus 로고
    • Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gam-maretroviral vector
    • Gaspar H, Parsley K, Howe S, King D, Gilmour K, Sinclair J et al. Gene therapy of X-linked severe combined immunodeficiency by use of a pseudotyped gam-maretroviral vector. The Lancet 2004; 364: 2181-2187.
    • (2004) The Lancet , vol.364 , pp. 2181-2187
    • Gaspar, H.1    Parsley, K.2    Howe, S.3    King, D.4    Gilmour, K.5    Sinclair, J.6
  • 4
    • 33645734405 scopus 로고    scopus 로고
    • Correction of X-linked chronic granulomatous disease by gene therapy, augmented by inser-tional activation of MDS1-EVI1, PRDM16 or SETBP1
    • Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U et al. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by inser-tional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med 2006; 12: 401-409.
    • (2006) Nat Med , vol.12 , pp. 401-409
    • Ott, M.G.1    Schmidt, M.2    Schwarzwaelder, K.3    Stein, S.4    Siler, U.5    Koehl, U.6
  • 6
    • 70449427834 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy
    • Cartier N, Hacein-Bey-Abina S, Bartholomae CC, Veres G, Schmidt M, Kutschera I et al. Hematopoietic stem cell gene therapy with a lentiviral vector in X-linked adrenoleukodystrophy. Science 2009; 326: 818-823.
    • (2009) Science , vol.326 , pp. 818-823
    • Cartier, N.1    Hacein-Bey-Abina, S.2    Bartholomae, C.C.3    Veres, G.4    Schmidt, M.5    Kutschera, I.6
  • 7
    • 51349090473 scopus 로고    scopus 로고
    • Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1
    • Hacein-Bey-Abina S, Garrigue A, Wang GP, Soulier J, Lim A, Morillon E et al. Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1. J Clin Invest 2008; 118: 3132-3142.
    • (2008) J Clin Invest , vol.118 , pp. 3132-3142
    • Hacein-Bey-Abina, S.1    Garrigue, A.2    Wang, G.P.3    Soulier, J.4    Lim, A.5    Morillon, E.6
  • 8
    • 51349158298 scopus 로고    scopus 로고
    • Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
    • Howe SJ, Mansour MR, Schwarzwaelder K, Bartholomae C, Hubank M, Kempski H et al. Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients. J Clin Invest 2008; 118: 3143-3150.
    • (2008) J Clin Invest , vol.118 , pp. 3143-3150
    • Howe, S.J.1    Mansour, M.R.2    Schwarzwaelder, K.3    Bartholomae, C.4    Hubank, M.5    Kempski, H.6
  • 9
    • 76249131912 scopus 로고    scopus 로고
    • Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease
    • Stein S, Ott MG, Schultze-Strasser S, Jauch A, Burwinkel B, Kinner A et al. Genomic instability and myelodysplasia with monosomy 7 consequent to EVI1 activation after gene therapy for chronic granulomatous disease. Nat Med 2010; 16: 198-204.
    • (2010) Nat Med , vol.16 , pp. 198-204
    • Stein, S.1    Ott, M.G.2    Schultze-Strasser, S.3    Jauch, A.4    Burwinkel, B.5    Kinner, A.6
  • 10
    • 84655170273 scopus 로고    scopus 로고
    • Safe harbours for the integration of new DNA in the human genome
    • Sadelain M, Papapetrou EP, Bushman FD. Safe harbours for the integration of new DNA in the human genome. Nat Rev Cancer 2011; 12: 51-58.
    • (2011) Nat Rev Cancer , vol.12 , pp. 51-58
    • Sadelain, M.1    Papapetrou, E.P.2    Bushman, F.D.3
  • 12
    • 0038618975 scopus 로고    scopus 로고
    • PhiC31 integrase-mediated nonviral genetic correction of junctional epidermolysis bul-losa
    • Ortiz-Urda S, Thyagarajan B, Keene DR, Lin Q, Calos MP, Khavari PA et al. PhiC31 integrase-mediated nonviral genetic correction of junctional epidermolysis bul-losa. Hum Gene Ther 2003; 14: 923-928.
    • (2003) Hum Gene Ther , vol.14 , pp. 923-928
    • Ortiz-Urda, S.1    Thyagarajan, B.2    Keene, D.R.3    Lin, Q.4    Calos, M.P.5    Khavari, P.A.6
  • 14
    • 33744786230 scopus 로고    scopus 로고
    • Phage phiC31 integrase-mediated genomic integration of the common cytokine receptor gamma chain in human T-cell lines
    • Ishikawa Y, Tanaka N, Murakami K, Uchiyama T, Kumaki S, Tsuchiya S et al. Phage phiC31 integrase-mediated genomic integration of the common cytokine receptor gamma chain in human T-cell lines. J Gene Med 2006; 8: 646-653.
    • (2006) J Gene Med , vol.8 , pp. 646-653
    • Ishikawa, Y.1    Tanaka, N.2    Murakami, K.3    Uchiyama, T.4    Kumaki, S.5    Tsuchiya, S.6
  • 16
    • 80051786231 scopus 로고    scopus 로고
    • Long-term phenotypic correction in factor IX knockout mice by using FC31 integrase-mediated gene therapy
    • Keravala A, Chavez CL, Hu G, Woodard LE, Monahan PE, Calos MP et al. Long-term phenotypic correction in factor IX knockout mice by using FC31 integrase-mediated gene therapy. Gene Therapy 2011; 18: 842-848.
    • (2011) Gene Therapy , vol.18 , pp. 842-848
    • Keravala, A.1    Chavez, C.L.2    Hu, G.3    Woodard, L.E.4    Monahan, P.E.5    Calos, M.P.6
  • 17
    • 77954244026 scopus 로고    scopus 로고
    • Biosafety assessment of site-directed transgene integration in human umbilical cord-lining cells
    • Sivalingam J, Krishnan S, Ng WH, Lee SS, Phan TT, Kon OL et al. Biosafety assessment of site-directed transgene integration in human umbilical cord-lining cells. Mol Ther 2010; 18: 1346-1356.
    • (2010) Mol Ther , vol.18 , pp. 1346-1356
    • Sivalingam, J.1    Krishnan, S.2    Ng, W.H.3    Lee, S.S.4    Phan, T.T.5    Kon, O.L.6
  • 18
    • 80053554900 scopus 로고    scopus 로고
    • Long-term expression of human coagulation factor VIII in a tolerant mouse model using the jC31 integrase system
    • Chavez CL, Keravala A, Chu JN, Farruggio AP, Cuéllar VE, Voorberg J et al. Long-term expression of human coagulation factor VIII in a tolerant mouse model using the jC31 integrase system. Hum Gene Ther 2012; 23: 390-398.
    • (2012) Hum Gene Ther , vol.23 , pp. 390-398
    • Chavez, C.L.1    Keravala, A.2    Chu, J.N.3    Farruggio, A.P.4    Cuéllar, V.E.5    Voorberg, J.6
  • 19
    • 31044452085 scopus 로고    scopus 로고
    • Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration
    • Bertoni C, Jarrahian S, Wheeler TM, Li Y, Olivares EC, Calos MP et al. Enhancement of plasmid-mediated gene therapy for muscular dystrophy by directed plasmid integration. Proc Natl Acad Sci USA 2006; 103: 419-424.
    • (2006) Proc Natl Acad Sci USA , vol.103 , pp. 419-424
    • Bertoni, C.1    Jarrahian, S.2    Wheeler, T.M.3    Li, Y.4    Olivares, E.C.5    Calos, M.P.6
  • 20
    • 0035008830 scopus 로고    scopus 로고
    • Site-specific genomic integration in mammalian cells mediated by phage phiC31 integrase
    • Thyagarajan B, Olivares EC, Hollis RP, Ginsburg DS, Calos MP. Site-specific genomic integration in mammalian cells mediated by phage phiC31 integrase. Mol Cell Biol 2001; 21: 3926-3934.
    • (2001) Mol Cell Biol , vol.21 , pp. 3926-3934
    • Thyagarajan, B.1    Olivares, E.C.2    Hollis, R.P.3    Ginsburg, D.S.4    Calos, M.P.5
  • 21
    • 67649840877 scopus 로고    scopus 로고
    • Directed engineering of a high-expression chimeric transgene as a strategy for gene therapy of hemophilia A
    • Doering CB, Denning G, Dooriss K, Gangadharan B, Johnston JM, Kerstann KW et al. Directed engineering of a high-expression chimeric transgene as a strategy for gene therapy of hemophilia A. Mol Ther 2009; 17: 1145-1154.
    • (2009) Mol Ther , vol.17 , pp. 1145-1154
    • Doering, C.B.1    Denning, G.2    Dooriss, K.3    Gangadharan, B.4    Johnston, J.M.5    Kerstann, K.W.6
  • 22
  • 23
    • 40149083557 scopus 로고    scopus 로고
    • Deleted in liver cancer-1 (DLC-1): A tumor suppressor not just for liver
    • Liao YC, Lo SH. Deleted in liver cancer-1 (DLC-1): a tumor suppressor not just for liver. Int J Biochem Cell Biol 2008; 40: 843-847.
    • (2008) Int J Biochem Cell Biol , vol.40 , pp. 843-847
    • Liao, Y.C.1    Lo, S.H.2
  • 24
    • 32344448765 scopus 로고    scopus 로고
    • Regulation of recombinant monoclonal antibody production in chinese hamster ovary cells: A comparative study of gene copy number, mRNA level, and protein expression
    • Jiang Z, Huang Y, Sharfstein ST. Regulation of recombinant monoclonal antibody production in chinese hamster ovary cells: a comparative study of gene copy number, mRNA level, and protein expression. Biotechnol Prog 2006; 22: 313-318.
    • (2006) Biotechnol Prog , vol.22 , pp. 313-318
    • Jiang, Z.1    Huang, Y.2    Sharfstein, S.T.3
  • 25
  • 26
    • 0032524031 scopus 로고    scopus 로고
    • Cloning, characterization, and chromosomal localization of a gene frequently deleted in human liver cancer (DLC-1) homologous to rat RhoGAP
    • Yuan BZ, Miller MJ, Keck CL, Zimonjic DB, Thorgeirsson SS, Popescu NC et al. Cloning, characterization, and chromosomal localization of a gene frequently deleted in human liver cancer (DLC-1) homologous to rat RhoGAP. Cancer Res 1998; 58: 2196-2199.
    • (1998) Cancer Res , vol.58 , pp. 2196-2199
    • Yuan, B.Z.1    Miller, M.J.2    Keck, C.L.3    Zimonjic, D.B.4    Thorgeirsson, S.S.5    Popescu, N.C.6
  • 27
    • 79955466717 scopus 로고    scopus 로고
    • A novel isoform of the 8p22 tumor suppressor gene DLC1 suppresses tumor growth and is frequently silenced in multiple common tumors
    • Low JSW, Tao Q, Ng KM, Goh HK, Shu XS, Woo WL et al. A novel isoform of the 8p22 tumor suppressor gene DLC1 suppresses tumor growth and is frequently silenced in multiple common tumors. Oncogene 2011; 30: 1923-1935.
    • (2011) Oncogene , vol.30 , pp. 1923-1935
    • Jsw, L.1    Tao, Q.2    Ng, K.M.3    Goh, H.K.4    Shu, X.S.5    Woo, W.L.6
  • 28
    • 33746124221 scopus 로고    scopus 로고
    • Phi c31 integrase induces chromosomal aberrations in primary human fibroblasts
    • Liu J, Jeppesen I, Nielsen K, Jensen TG. Phi c31 integrase induces chromosomal aberrations in primary human fibroblasts. Gene Therapy 2006; 13: 1188-1190.
    • (2006) Gene Therapy , vol.13 , pp. 1188-1190
    • Liu, J.1    Jeppesen, I.2    Nielsen, K.3    Jensen, T.G.4
  • 29
    • 33750997895 scopus 로고    scopus 로고
    • Molecular analysis of chromosomal rearrangements in mammalian cells after phiC31-mediated integration
    • Ehrhardt A, Engler JA, Xu H, Cherry AM, Kay MA. Molecular analysis of chromosomal rearrangements in mammalian cells after phiC31-mediated integration. Hum Gene Ther 2006; 17: 1077-1094.
    • (2006) Hum Gene Ther , vol.17 , pp. 1077-1094
    • Ehrhardt, A.1    Engler, J.A.2    Xu, H.3    Cherry, A.M.4    Kay, M.A.5
  • 32
    • 84865272073 scopus 로고    scopus 로고
    • Homing endonucleases: DNA scissors on a mission
    • Hafez M, Hausner G. Homing endonucleases: DNA scissors on a mission. Genome 2012; 55: 553-569.
    • (2012) Genome , vol.55 , pp. 553-569
    • Hafez, M.1    Hausner, G.2
  • 33
    • 84873566329 scopus 로고    scopus 로고
    • Zinc finger nucleases: Looking toward translation
    • Palpant NJ, Dudzinski D. Zinc finger nucleases: looking toward translation. Gene Therapy 2013; 20: 121-127.
    • (2013) Gene Therapy , vol.20 , pp. 121-127
    • Palpant, N.J.1    Dudzinski, D.2
  • 34
    • 84861990434 scopus 로고    scopus 로고
    • Precision genome engineering with programmable DNA-nicking enzymes
    • Kim E, Kim S, Kim DH, Choi BS, Choi IY, Kim JS et al. Precision genome engineering with programmable DNA-nicking enzymes. Genome Res 2012; 22: 1327-1333.
    • (2012) Genome Res , vol.22 , pp. 1327-1333
    • Kim, E.1    Kim, S.2    Kim, D.H.3    Choi, B.S.4    Choi, I.Y.5    Kim, J.S.6
  • 35
    • 84871519181 scopus 로고    scopus 로고
    • TALENs: A widely applicable technology for targeted genome editing
    • Joung JK, Sander JD. TALENs: a widely applicable technology for targeted genome editing. Nat Rev Mol Cell Biol 2013; 14: 49-55.
    • (2013) Nat Rev Mol Cell Biol , vol.14 , pp. 49-55
    • Joung, J.K.1    Sander, J.D.2
  • 37
    • 80053350622 scopus 로고    scopus 로고
    • Site-specific integration and tailoring of cassette design for sustainable gene transfer
    • Lombardo A, Cesana D, Genovese P, Di Stefano B, Provasi E, Colombo DF et al. Site-specific integration and tailoring of cassette design for sustainable gene transfer. Nat Methods 2011; 8: 861-869.
    • (2011) Nat Methods , vol.8 , pp. 861-869
    • Lombardo, A.1    Cesana, D.2    Genovese, P.3    Di Stefano, B.4    Provasi, E.5    Colombo, D.F.6
  • 38
    • 27244455897 scopus 로고    scopus 로고
    • Deleted in liver cancer 2 (DLC2) suppresses cell transformation by means of inhibition of RhoA activity
    • Leung THY, Ching YP, Yam JWP, Wong CM, Yau TO, Jin DY et al. Deleted in liver cancer 2 (DLC2) suppresses cell transformation by means of inhibition of RhoA activity. Proc Natl Acad Sci USA 2005; 102: 15207-15212.
    • (2005) Proc Natl Acad Sci USA , vol.102 , pp. 15207-15212
    • Thy, L.1    Ching, Y.P.2    Jwp, Y.3    Wong, C.M.4    Yau, T.O.5    Jin, D.Y.6
  • 39
    • 0027237331 scopus 로고
    • Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production
    • Lynch CM, Israel DI, Kaufman RJ, Miller AD. Sequences in the coding region of clotting factor VIII act as dominant inhibitors of RNA accumulation and protein production. Hum Gene Ther 1993; 4: 259-272.
    • (1993) Hum Gene Ther , vol.4 , pp. 259-272
    • Lynch, C.M.1    Israel, D.I.2    Kaufman, R.J.3    Miller, A.D.4
  • 40
    • 0028917478 scopus 로고
    • Expression of the blood-clotting factor-VIII cDNA is repressed by a transcriptional silencer located in its coding region
    • Hoeben RC, Fallaux FJ, Cramer SJ, van den Wollenberg DJ, van Ormondt H, Briët E et al. Expression of the blood-clotting factor-VIII cDNA is repressed by a transcriptional silencer located in its coding region. Blood 1995; 85: 2447-2454.
    • (1995) Blood , vol.85 , pp. 2447-2454
    • Hoeben, R.C.1    Fallaux, F.J.2    Cramer, S.J.3    Van Den Wollenberg, D.J.4    Van Ormondt, H.5    Briët, E.6
  • 41
    • 0034728357 scopus 로고    scopus 로고
    • ATP-dependent dissociation of non-disulfide-linked aggregates of coagulation factor VIII is a rate-limiting step for secretion
    • Tagliavacca L, Wang Q, Kaufman RJ. ATP-dependent dissociation of non-disulfide-linked aggregates of coagulation factor VIII is a rate-limiting step for secretion. Biochemistry 2000; 39: 1973-1981.
    • (2000) Biochemistry , vol.39 , pp. 1973-1981
    • Tagliavacca, L.1    Wang, Q.2    Kaufman, R.J.3
  • 42
    • 1342282981 scopus 로고    scopus 로고
    • Identification of porcine coagulation factor VIII domains responsible for high level expression via enhanced secretion
    • Doering CB, Healey JF, Parker ET, Barrow RT, Lollar P. Identification of porcine coagulation factor VIII domains responsible for high level expression via enhanced secretion. J Biol Chem 2004; 279: 6546-6552.
    • (2004) J Biol Chem , vol.279 , pp. 6546-6552
    • Doering, C.B.1    Healey, J.F.2    Parker, E.T.3    Barrow, R.T.4    Lollar, P.5
  • 43
    • 82355163626 scopus 로고    scopus 로고
    • Umbilical cord lining stem cells as a novel and promising source for ocular surface regeneration
    • Reza HM, Ng BY, Gimeno FL, Phan TT, Ang LPK. Umbilical cord lining stem cells as a novel and promising source for ocular surface regeneration. Stem Cell Rev 2011; 7: 935-947.
    • (2011) Stem Cell Rev , vol.7 , pp. 935-947
    • Reza, H.M.1    Ng, B.Y.2    Gimeno, F.L.3    Phan, T.T.4    Ang, L.P.K.5
  • 44
    • 84863056432 scopus 로고    scopus 로고
    • Characterization of human umbilical cord lining derived epithelial cells and transplantation potential
    • Zhou Y, Gan SU, Lin G, Lim YT, Masilamani J, Mustafa FB et al. Characterization of human umbilical cord lining derived epithelial cells and transplantation potential. Cell Transplant 2011; 20: 1827-1841.
    • (2011) Cell Transplant , vol.20 , pp. 1827-1841
    • Zhou, Y.1    Gan, S.U.2    Lin, G.3    Lim, Y.T.4    Masilamani, J.5    Mustafa, F.B.6
  • 45
    • 0035945238 scopus 로고    scopus 로고
    • Directed evolution of a recombinase for improved genomic integration at a native human sequence
    • Sclimenti CR, Thyagarajan B, Calos MP. Directed evolution of a recombinase for improved genomic integration at a native human sequence. Nucleic Acids Res 2001; 29: 5044-5051.
    • (2001) Nucleic Acids Res , vol.29 , pp. 5044-5051
    • Sclimenti, C.R.1    Thyagarajan, B.2    Calos, M.P.3
  • 46
    • 0035710746 scopus 로고    scopus 로고
    • Analysis of relative gene expression data using real-time quantitative PCR and the 2(-delta delta C(T)) method
    • Livak KJ, Schmittgen TD. Analysis of relative gene expression data using real-time quantitative PCR and the 2(-delta delta C(T)) method. Methods 2001; 25: 402-408.
    • (2001) Methods , vol.25 , pp. 402-408
    • Livak, K.J.1    Schmittgen, T.D.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.