메뉴 건너뛰기




Volumn 9, Issue 8, 2014, Pages

Delivery of full-length factor VIII using a piggyBac transposon vector to correct a mouse model of hemophilia A

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; COMPLEMENTARY DNA; TRANSPOSON;

EID: 84920853211     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0104957     Document Type: Article
Times cited : (44)

References (43)
  • 2
    • 1642459412 scopus 로고    scopus 로고
    • Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A
    • DOI 10.1182/blood-2003-05-1426
    • Brown BD, Shi CX, Powell S, Hurlbut D, Graham FL, et al. (2004) Helper-dependent adenoviral vectors mediate therapeutic factor VIII expression for several months with minimal accompanying toxicity in a canine model of severe hemophilia A. Blood. pp. 804-810. (Pubitemid 38129536)
    • (2004) Blood , vol.103 , Issue.3 , pp. 804-810
    • Brown, B.D.1    Shi, C.X.2    Powell, S.3    Hurlbut, D.4    Graham, F.L.5    Lillicrap, D.6
  • 3
    • 79952122629 scopus 로고    scopus 로고
    • Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII
    • Hu C, Cela RG, Suzuki M, Lee B, Lipshutz GS (2011) Neonatal helper-dependent adenoviral vector gene therapy mediates correction of hemophilia A and tolerance to human factor VIII. Proc Natl Acad Sci USA. pp. 2082-2087.
    • (2011) Proc Natl Acad Sci USA , pp. 2082-2087
    • Hu, C.1    Cela, R.G.2    Suzuki, M.3    Lee, B.4    Lipshutz, G.S.5
  • 6
    • 46249125272 scopus 로고    scopus 로고
    • Complete correction of hemophilia a with adeno-associated viral vectors containing a full-size expression cassette
    • DOI 10.1089/hum.2007.0182
    • Lu H, Chen L, Wang J, Huack B, Sarkar R, et al. (2008) Complete correction of hemophilia A with adeno-associated viral vectors containing a full-size expression cassette. Hum Gene Ther. pp. 648-654. (Pubitemid 351913523)
    • (2008) Human Gene Therapy , vol.19 , Issue.6 , pp. 648-654
    • Lu, H.1    Chen, L.2    Wang, J.3    Huack, B.4    Sarkar, R.5    Zhou, S.6    Xu, R.7    Ding, Q.8    Wang, X.9    Wang, H.10    Xiao, W.11
  • 8
    • 0037853196 scopus 로고    scopus 로고
    • Efficient production of human FVIII in hemophilic mice using lentiviral vectors
    • DOI 10.1016/S1525-0016(03)00073-X
    • Kootstra NA, Matsumura R, Verma IM (2003) Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol Ther. pp. 623-631. (Pubitemid 36626242)
    • (2003) Molecular Therapy , vol.7 , Issue.5 I , pp. 623-631
    • Kootstra, N.A.1    Matsumura, R.2    Verma, I.M.3
  • 9
    • 23944484509 scopus 로고    scopus 로고
    • Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer
    • DOI 10.1182/blood-2004-11-4358
    • Kang Y, Xie L, Tran DT, Stein CS, Hickey M, et al. (2005) Persistent expression of factor VIII in vivo following nonprimate lentiviral gene transfer. Blood. pp. 1552-1558. (Pubitemid 41208565)
    • (2005) Blood , vol.106 , Issue.5 , pp. 1552-1558
    • Kang, Y.1    Xie, L.2    Tran, D.T.3    Stein, C.S.4    Hickey, M.5    Davidson, B.L.6    McCray Jr., P.B.7
  • 10
    • 79953322192 scopus 로고    scopus 로고
    • A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A
    • Matsui H, Hegadorn C, Ozelo M, Burnett E, Tuttle A, et al. (2011) A microRNA-regulated and GP64-pseudotyped lentiviral vector mediates stable expression of FVIII in a murine model of Hemophilia A. Mol Ther. pp. 723-730.
    • (2011) Mol Ther , pp. 723-730
    • Matsui, H.1    Hegadorn, C.2    Ozelo, M.3    Burnett, E.4    Tuttle, A.5
  • 11
    • 35348867348 scopus 로고    scopus 로고
    • Ex vivo gene therapy for hemophilia A that enhances safe delivery and sustained in vivo factor VIII expression from lentivirally engineered endothelial progenitors
    • DOI 10.1634/stemcells.2006-0699
    • Matsui H, Shibata M, Brown B, Labelle A, Hegadorn C, et al. (2007) Ex vivo gene therapy for hemophilia A that enhances safe delivery and sustained in vivo factor VIII expression from lentivirally engineered endothelial progenitors. Stem Cells. pp. 2660-2669. (Pubitemid 47582759)
    • (2007) Stem Cells , vol.25 , Issue.10 , pp. 2660-2669
    • Matsui, H.1    Shibata, M.2    Brown, B.3    Labelle, A.4    Hegadorn, C.5    Andrews, C.6    Hebbel, R.P.7    Galipeau, J.8    Hough, C.9    Lillicrap, D.10
  • 13
    • 65449140491 scopus 로고    scopus 로고
    • Gene therapy for haemophilia...Yes, but...With non-viral vectors?
    • Liras A, Olmedillas S (2009) Gene therapy for haemophilia...yes, but...with non-viral vectors? Haemophilia. pp. 811-816.
    • (2009) Haemophilia , pp. 811-816
    • Liras, A.1    Olmedillas, S.2
  • 14
    • 0034986065 scopus 로고    scopus 로고
    • Acute direct adenoviral vector cytotoxicity and chronic, but not acute, inflammatory responses correlate with decreased vector-mediated transgene expression in the brain
    • DOI 10.1006/mthe.2000.0224
    • Thomas CE, Birkett D, Anozie I, Castro MG, Lowenstein PR (2001) Acute direct adenoviral vector cytotoxicity and chronic, but not acute, inflammatory responses correlate with decreased vector-mediated transgene expression in the brain. Mol Ther 3: 36-46. (Pubitemid 32509803)
    • (2001) Molecular Therapy , vol.3 , Issue.1 , pp. 36-46
    • Thomas, C.E.1    Birkett, D.2    Anozie, I.3    Castro, M.G.4    Lowenstein, P.R.5
  • 15
    • 70449570719 scopus 로고    scopus 로고
    • Functional roles of the factor VIII B domain
    • Pipe SW (2009) Functional roles of the factor VIII B domain. Haemophilia. pp. 1187-1196.
    • (2009) Haemophilia , pp. 1187-1196
    • Pipe, S.W.1
  • 16
    • 80053554900 scopus 로고    scopus 로고
    • Long-term expression of human coagulation factor VIII in a tolerant mouse model using the QC31 integrase system
    • Chavez CL, Keravala A, Chu JN, Farruggio AP, Cuéllar VE, et al. (2012) Long-term expression of human coagulation factor VIII in a tolerant mouse model using the QC31 integrase system. Hum Gene Ther. pp. 390-398.
    • (2012) Hum Gene Ther , pp. 390-398
    • Chavez, C.L.1    Keravala, A.2    Chu, J.N.3    Farruggio, A.P.4    Cuéllar, V.E.5
  • 17
    • 79960424171 scopus 로고    scopus 로고
    • In vivo genome editing restores haemostasis in a mouse model of haemophilia
    • Li H, Haurigot V, Doyon Y, Li T, Wong SY, et al. (2011) In vivo genome editing restores haemostasis in a mouse model of haemophilia. Nature. pp. 217-221.
    • (2011) Nature , pp. 217-221
    • Li, H.1    Haurigot, V.2    Doyon, Y.3    Li, T.4    Wong, S.Y.5
  • 20
    • 23744479962 scopus 로고    scopus 로고
    • Efficient transposition of the piggyBac (PB) transposon in mammalian cells and mice
    • DOI 10.1016/j.cell.2005.07.013, PII S0092867405007075
    • Ding S, Wu X, Li G, Han M, Zhuang Y, et al. (2005) Efficient transposition of the piggyBac (PB) transposon in mammalian cells and mice. Cell. pp. 473-483. (Pubitemid 41127147)
    • (2005) Cell , vol.122 , Issue.3 , pp. 473-483
    • Ding, S.1    Wu, X.2    Li, G.3    Han, M.4    Zhuang, Y.5    Xu, T.6
  • 21
    • 33845981514 scopus 로고    scopus 로고
    • PiggyBac transposon-mediated gene transfer in human cells
    • DOI 10.1038/sj.mt.6300028, PII 6300028
    • Wilson MH, Coates CJ, George AL (2007) PiggyBac transposon-mediated gene transfer in human cells. Mol Ther.pp. 139-145. (Pubitemid 46043659)
    • (2007) Molecular Therapy , vol.15 , Issue.1 , pp. 139-145
    • Wilson, M.H.1    Coates, C.J.2    George Jr., A.L.3
  • 22
    • 77957579849 scopus 로고    scopus 로고
    • Gene transfer efficiency and genome-wide integration profiling of Sleeping Beauty, Tol2, and piggyBac transposons in human primary T cells
    • Huang X, Guo H, Tammana S, Jung Y-C, Mellgren E, et al. (2010) Gene transfer efficiency and genome-wide integration profiling of Sleeping Beauty, Tol2, and piggyBac transposons in human primary T cells. Mol Ther. pp. 1803-1813.
    • (2010) Mol Ther , pp. 1803-1813
    • Huang, X.1    Guo, H.2    Tammana, S.3    Jung, Y.-C.4    Mellgren, E.5
  • 24
  • 25
    • 65449121435 scopus 로고    scopus 로고
    • Isolation of human iPS cells using EOS lentiviral vectors to select for pluripotency
    • Hotta A, Cheung AYL, Farra N, Vijayaragavan K, Séguin CA, et al. (2009) Isolation of human iPS cells using EOS lentiviral vectors to select for pluripotency. Nat Methods. pp. 370-376.
    • (2009) Nat Methods , pp. 370-376
    • Hotta, A.1    Cheung, A.Y.L.2    Farra, N.3    Vijayaragavan, K.4    Séguin, C.A.5
  • 27
    • 0025884056 scopus 로고
    • Efficient selection for high-expression transfectants with a novel eukaryotic vector
    • Niwa H, Yamamura K, Miyazaki J (1991) Efficient selection for high-expression transfectants with a novel eukaryotic vector. Gene 108: 193-199.
    • (1991) Gene , vol.108 , pp. 193-199
    • Niwa, H.1    Yamamura, K.2    Miyazaki, J.3
  • 28
    • 0029133142 scopus 로고
    • Novel forms of B-domain-deleted recombinant factor VIII molecules. Construction and biochemical characterization
    • Lind P, Larsson K, Spira J, Sydow-Backman M, Almstedt A, et al. (1995) Novel forms of B-domain-deleted recombinant factor VIII molecules. Construction and biochemical characterization. Eur J Biochem 232: 19-27.
    • (1995) Eur J Biochem , vol.232 , pp. 19-27
    • Lind, P.1    Larsson, K.2    Spira, J.3    Sydow-Backman, M.4    Almstedt, A.5
  • 29
    • 0035169521 scopus 로고    scopus 로고
    • Antigenicity of putative phospholipid membrane-binding residues in factor VIII
    • DOI 10.1182/blood.V97.1.169
    • Barrow RT, Healey JF, Jacquemin MG, Saint-Remy JM, Lollar P (2001) Antigenicity of putative phospholipid membrane-binding residues in factor VIII. Blood 97: 169-174. (Pubitemid 32061257)
    • (2001) Blood , vol.97 , Issue.1 , pp. 169-174
    • Barrow, R.T.1    Healey, J.F.2    Jacquemin, M.G.3    Saint-Remy, J.-M.R.4    Lollar, P.5
  • 30
    • 36248966518 scopus 로고    scopus 로고
    • Induction of Pluripotent Stem Cells from Adult Human Fibroblasts by Defined Factors
    • DOI 10.1016/j.cell.2007.11.019, PII S0092867407014717
    • Takahashi K, Tanabe K, Ohnuki M, Narita M, Ichisaka T, et al. (2007) Induction of pluripotent stem cells from adult human fibroblasts by defined factors. Cell 131: 861-872. (Pubitemid 350138099)
    • (2007) Cell , vol.131 , Issue.5 , pp. 861-872
    • Takahashi, K.1    Tanabe, K.2    Ohnuki, M.3    Narita, M.4    Ichisaka, T.5    Tomoda, K.6    Yamanaka, S.7
  • 31
    • 0032805251 scopus 로고    scopus 로고
    • Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA
    • DOI 10.1038/sj.gt.3300947
    • Liu F, Song Y, Liu D (1999) Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther. pp. 1258-1266. (Pubitemid 29359019)
    • (1999) Gene Therapy , vol.6 , Issue.7 , pp. 1258-1266
    • Liu, F.1    Song, Y.K.2    Liu, D.3
  • 34
    • 63049090064 scopus 로고    scopus 로고
    • A fresh look at iPS cells
    • Yamanaka S (2009) A fresh look at iPS cells. Cell 137: 13-17.
    • (2009) Cell , vol.137 , pp. 13-17
    • Yamanaka, S.1
  • 35
    • 77958579746 scopus 로고    scopus 로고
    • Gene therapy strategies for hemophilia: Benefits versus risks
    • Petrus I, Chuah M, Vandendriessche T (2010) Gene therapy strategies for hemophilia: benefits versus risks. J Gene Med. pp. 797-809.
    • (2010) J Gene Med , pp. 797-809
    • Petrus, I.1    Chuah, M.2    Vandendriessche, T.3
  • 37
    • 33645734405 scopus 로고    scopus 로고
    • Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1
    • Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, et al. (2006) Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. pp. 401-409.
    • (2006) Nat Med , pp. 401-409
    • Ott, M.G.1    Schmidt, M.2    Schwarzwaelder, K.3    Stein, S.4    Siler, U.5
  • 39
    • 84897570147 scopus 로고    scopus 로고
    • Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo
    • Cesana D, Ranzani M, Volpin M, Bartholomae C, Duros C, et al. (2014) Uncovering and dissecting the genotoxicity of self-inactivating lentiviral vectors in vivo. Mol Ther.
    • (2014) Mol Ther
    • Cesana, D.1    Ranzani, M.2    Volpin, M.3    Bartholomae, C.4    Duros, C.5
  • 40
    • 61649121501 scopus 로고    scopus 로고
    • Image-guided, lobe-specific hydrodynamic gene delivery to swine liver
    • Kamimura K, Suda T, Xu W, Zhang G, Liu D (2009) Image-guided, lobe-specific hydrodynamic gene delivery to swine liver. Mol Ther. pp. 491-499.
    • (2009) Mol Ther , pp. 491-499
    • Kamimura, K.1    Suda, T.2    Xu, W.3    Zhang, G.4    Liu, D.5
  • 41
    • 44349119297 scopus 로고    scopus 로고
    • Computer-assisted hydrodynamic gene delivery
    • DOI 10.1038/mt.2008.66, PII MT200866
    • Suda T, Suda K, Liu D (2008) Computer-assisted hydrodynamic gene delivery. Mol Ther. pp. 1098-1104. (Pubitemid 351737075)
    • (2008) Molecular Therapy , vol.16 , Issue.6 , pp. 1098-1104
    • Suda, T.1    Suda, K.2    Liu, D.3
  • 42
    • 79955998860 scopus 로고    scopus 로고
    • Efficacy and safety of Sleeping Beauty transposon-mediated gene transfer in preclinical animal studies
    • 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.
    • (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
  • 43
    • 81355149640 scopus 로고    scopus 로고
    • Nanotechnology for therapy and imaging of liver diseases
    • Reddy LH, Couvreur P (2011) Nanotechnology for therapy and imaging of liver diseases. J Hepatol 55: 1461-1466.
    • (2011) J Hepatol , vol.55 , pp. 1461-1466
    • Reddy, L.H.1    Couvreur, P.2


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.