메뉴 건너뛰기




Volumn 12, Issue 10, 2010, Pages 810-817

AAV plasmid DNA simplifies liver-directed in vivo gene therapy: Comparison of expression levels after plasmid DNA-, adeno-associated virus- and adenovirus-mediated liver transfection

Author keywords

AAV plasmid; in vivo gene therapy; Naked DNA gene transfer; Nonviral gene transfer

Indexed keywords

ADENOVIRUS VECTOR; MAJOR HISTOCOMPATIBILITY ANTIGEN CLASS 1; PARVOVIRUS VECTOR; PLASMID DNA; RT1 A ANTIGEN; UNCLASSIFIED DRUG;

EID: 77958559564     PISSN: 1099498X     EISSN: 15212254     Source Type: Journal    
DOI: 10.1002/jgm.1498     Document Type: Article
Times cited : (4)

References (66)
  • 1
    • 0037093518 scopus 로고    scopus 로고
    • Induction of donor-specific tolerance by adenovirus-mediated CD40Ig gene therapy in rat liver transplantation
    • Nomura M, Yamashita K, Murakami M, et al. Induction of donor-specific tolerance by adenovirus-mediated CD40Ig gene therapy in rat liver transplantation. Transplantation 2002; 73: 1403-1410.
    • (2002) Transplantation , vol.73 , pp. 1403-1410
    • Nomura, M.1    Yamashita, K.2    Murakami, M.3
  • 2
    • 0034962640 scopus 로고    scopus 로고
    • Adenovirus-mediated CD40 ligand gene therapy in a rat model of orthotopic hepatocellular carcinoma
    • Schmitz V, Barajas M, Wang L, et al. Adenovirus-mediated CD40 ligand gene therapy in a rat model of orthotopic hepatocellular carcinoma. Hepatology 2001; 34: 72-81.
    • (2001) Hepatology , vol.34 , pp. 72-81
    • Schmitz, V.1    Barajas, M.2    Wang, L.3
  • 3
    • 0037442265 scopus 로고    scopus 로고
    • Long-term liver allograft survival induced by combined treatment with rAAV-hCTLA4Ig gene transfer and low-dose FK506
    • Yang Z, Wu X, Tsui TY, Hou Y, Luk JM, Fan ST. Long-term liver allograft survival induced by combined treatment with rAAV-hCTLA4Ig gene transfer and low-dose FK506. Transplantation 2003; 75: 303-308.
    • (2003) Transplantation , vol.75 , pp. 303-308
    • Yang, Z.1    Wu, X.2    Tsui, T.Y.3    Hou, Y.4    Luk, J.M.5    Fan, S.T.6
  • 4
    • 0344950999 scopus 로고    scopus 로고
    • Recombinant adeno-associated virus vector: is it ideal for gene delivery in liver transplantation?
    • Yang ZF, Wu XB, Tsui TY, Hou YD, Luk JM, Fan ST. Recombinant adeno-associated virus vector: is it ideal for gene delivery in liver transplantation? Liver Transpl 2003; 9: 411-420.
    • (2003) Liver Transpl , vol.9 , pp. 411-420
    • Yang, Z.F.1    Wu, X.B.2    Tsui, T.Y.3    Hou, Y.D.4    Luk, J.M.5    Fan, S.T.6
  • 5
    • 77952571753 scopus 로고    scopus 로고
    • Two decades of clinical gene therapy-success is finally mounting
    • Herzog RW, Cao O, Srivastava A. Two decades of clinical gene therapy-success is finally mounting. Discov Med 2010; 9: 105-111.
    • (2010) Discov Med , vol.9 , pp. 105-111
    • Herzog, R.W.1    Cao, O.2    Srivastava, A.3
  • 7
    • 0038745599 scopus 로고    scopus 로고
    • Progress and problems with the use of viral vectors for gene therapy
    • Thomas CE, Ehrhardt A, Kay MA. Progress and problems with the use of viral vectors for gene therapy. Nat Rev Genet 2003; 4: 346-358.
    • (2003) Nat Rev Genet , vol.4 , pp. 346-358
    • Thomas, C.E.1    Ehrhardt, A.2    Kay, M.A.3
  • 8
    • 34447535345 scopus 로고    scopus 로고
    • Engineering targeted viral vectors for gene therapy
    • Waehler R, Russell SJ, Curiel DT. Engineering targeted viral vectors for gene therapy. Nat Rev Genet 2007; 8: 573-587.
    • (2007) Nat Rev Genet , vol.8 , pp. 573-587
    • Waehler, R.1    Russell, S.J.2    Curiel, D.T.3
  • 9
    • 33748290173 scopus 로고    scopus 로고
    • Gene therapy progress and prospects: non-viral gene therapy by systemic delivery
    • Li SD, Huang L. Gene therapy progress and prospects: non-viral gene therapy by systemic delivery. Gene Ther 2006; 13: 1313-1319.
    • (2006) Gene Ther , vol.13 , pp. 1313-1319
    • Li, S.D.1    Huang, L.2
  • 10
    • 1942535827 scopus 로고    scopus 로고
    • Gene therapy for organ grafts using rapid injection of naked DNA: application to the rat liver
    • Inoue S, Hakamata Y, Kaneko M, Kobayashi E. Gene therapy for organ grafts using rapid injection of naked DNA: application to the rat liver. Transplantation 2004; 77: 997-1003.
    • (2004) Transplantation , vol.77 , pp. 997-1003
    • Inoue, S.1    Hakamata, Y.2    Kaneko, M.3    Kobayashi, E.4
  • 11
    • 0035993571 scopus 로고    scopus 로고
    • Expression of high levels of soluble allogeneic major histocompatability complex class I antigen by adenoviral gene transfer strongly inhibits allospecific cytotoxic T lymphocytes in vivo
    • Graeb C, Justl M, Scherer MN, et al. Expression of high levels of soluble allogeneic major histocompatability complex class I antigen by adenoviral gene transfer strongly inhibits allospecific cytotoxic T lymphocytes in vivo. Transplant Proc 2002; 34: 1398-1399.
    • (2002) Transplant Proc , vol.34 , pp. 1398-1399
    • Graeb, C.1    Justl, M.2    Scherer, M.N.3
  • 12
    • 33846022383 scopus 로고    scopus 로고
    • Gene therapy progress and prospects: hydrodynamic gene delivery
    • Herweijer H, Wolff JA. Gene therapy progress and prospects: hydrodynamic gene delivery. Gene Ther 2007; 14: 99-107.
    • (2007) Gene Ther , vol.14 , pp. 99-107
    • Herweijer, H.1    Wolff, J.A.2
  • 13
    • 4344700911 scopus 로고    scopus 로고
    • Long-term transgene expression from plasmid DNA gene therapy vectors is negatively affected by CpG dinucleotides
    • Hodges BL, Taylor KM, Joseph MF, Bourgeois SA, Scheule RK. Long-term transgene expression from plasmid DNA gene therapy vectors is negatively affected by CpG dinucleotides. Mol Ther 2004; 10: 269-278.
    • (2004) Mol Ther , vol.10 , pp. 269-278
    • Hodges, B.L.1    Taylor, K.M.2    Joseph, M.F.3    Bourgeois, S.A.4    Scheule, R.K.5
  • 14
    • 33746733616 scopus 로고    scopus 로고
    • Gene therapy for haemophilia
    • Ponder KP. Gene therapy for haemophilia. Curr Opin Hematol 2006; 13: 301-307.
    • (2006) Curr Opin Hematol , vol.13 , pp. 301-307
    • Ponder, K.P.1
  • 15
    • 33746238237 scopus 로고    scopus 로고
    • Mechanism of plasmid delivery by hydrodynamic tail vein injection. II. Morphological studies
    • Budker VG, Subbotin VM, Budker T, Sebestyen MG, Zhang G, Wolff JA. Mechanism of plasmid delivery by hydrodynamic tail vein injection. II. Morphological studies. J Gene Med 2006; 8: 874-888.
    • (2006) J Gene Med , vol.8 , pp. 874-888
    • Budker, V.G.1    Subbotin, V.M.2    Budker, T.3    Sebestyen, M.G.4    Zhang, G.5    Wolff, J.A.6
  • 16
    • 33746265976 scopus 로고    scopus 로고
    • Mechanism of plasmid delivery by hydrodynamic tail vein injection. I. Hepatocyte uptake of various molecules
    • Sebestyen MG, Budker VG, Budker T, et al. Mechanism of plasmid delivery by hydrodynamic tail vein injection. I. Hepatocyte uptake of various molecules. J Gene Med 2006; 8: 852-873.
    • (2006) J Gene Med , vol.8 , pp. 852-873
    • Sebestyen, M.G.1    Budker, V.G.2    Budker, T.3
  • 17
    • 33645937646 scopus 로고    scopus 로고
    • The mechanism of naked DNA uptake and expression
    • Wolff JA, Budker V. The mechanism of naked DNA uptake and expression. Adv Genet 2005; 54: 3-20.
    • (2005) Adv Genet , vol.54 , pp. 3-20
    • Wolff, J.A.1    Budker, V.2
  • 19
    • 33745089314 scopus 로고    scopus 로고
    • Adeno-associated virus as a gene therapy vector: vector development, production and clinical applications
    • Grieger JC, Samulski RJ. Adeno-associated virus as a gene therapy vector: vector development, production and clinical applications. Adv Biochem Eng Biotechnol 2005; 99: 119-145.
    • (2005) Adv Biochem Eng Biotechnol , vol.99 , pp. 119-145
    • Grieger, J.C.1    Samulski, R.J.2
  • 20
    • 1542369977 scopus 로고    scopus 로고
    • From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy
    • Grimm D, Kay MA. From virus evolution to vector revolution: use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy. Curr Gene Ther 2003; 3: 281-304.
    • (2003) Curr Gene Ther , vol.3 , pp. 281-304
    • Grimm, D.1    Kay, M.A.2
  • 21
    • 0037676229 scopus 로고    scopus 로고
    • AAV-mediated gene transfer for haemophilia
    • High K. AAV-mediated gene transfer for haemophilia. Genet Med 2002; 4: 56S-61S.
    • (2002) Genet Med , vol.4
    • High, K.1
  • 22
    • 21244476011 scopus 로고    scopus 로고
    • AAV-mediated gene transfer for treatment of haemophilia
    • Wang L, Herzog RW. AAV-mediated gene transfer for treatment of haemophilia. Curr Gene Ther 2005; 5: 349-360.
    • (2005) Curr Gene Ther , vol.5 , pp. 349-360
    • Wang, L.1    Herzog, R.W.2
  • 23
    • 43349089087 scopus 로고    scopus 로고
    • Prolongation of heart allograft survival after long-term expression of soluble MHC class I antigens and vIL-10 in the liver by AAV-plasmid-mediated gene transfer
    • Doenecke A, Frank E, Scherer MN, Schlitt HJ, Geissler EK. Prolongation of heart allograft survival after long-term expression of soluble MHC class I antigens and vIL-10 in the liver by AAV-plasmid-mediated gene transfer. Langenbecks Arch Surg 2008; 393: 343-348.
    • (2008) Langenbecks Arch Surg , vol.393 , pp. 343-348
    • Doenecke, A.1    Frank, E.2    Scherer, M.N.3    Schlitt, H.J.4    Geissler, E.K.5
  • 25
    • 0030855528 scopus 로고    scopus 로고
    • Sensitivity and reproducibility in adenoviral infectious titer determination
    • Nyberg-Hoffman C, Shabram P, Li W, Giroux D, Aguilar-Cordova E. Sensitivity and reproducibility in adenoviral infectious titer determination. Nat Med 1997; 3: 808-811.
    • (1997) Nat Med , vol.3 , pp. 808-811
    • Nyberg-Hoffman, C.1    Shabram, P.2    Li, W.3    Giroux, D.4    Aguilar-Cordova, E.5
  • 26
    • 0026660060 scopus 로고
    • Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector
    • Walsh CE, Liu JM, Xiao X, Young NS, Nienhuis AW, Samulski RJ. Regulated high level expression of a human gamma-globin gene introduced into erythroid cells by an adeno-associated virus vector. Proc Natl Acad Sci USA 1992; 89: 7257-7261.
    • (1992) Proc Natl Acad Sci USA , vol.89 , pp. 7257-7261
    • Walsh, C.E.1    Liu, J.M.2    Xiao, X.3    Young, N.S.4    Nienhuis, A.W.5    Samulski, R.J.6
  • 28
    • 0035163792 scopus 로고    scopus 로고
    • Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column
    • Auricchio A, Hildinger M, O'Connor E, Gao GP, Wilson JM. Isolation of highly infectious and pure adeno-associated virus type 2 vectors with a single-step gravity-flow column. Hum Gene Ther 2001; 12: 71-76.
    • (2001) Hum Gene Ther , vol.12 , pp. 71-76
    • Auricchio, A.1    Hildinger, M.2    O'Connor, E.3    Gao, G.P.4    Wilson, J.M.5
  • 29
    • 2642642141 scopus 로고    scopus 로고
    • Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus
    • Xiao X, Li J, Samulski RJ. Production of high-titer recombinant adeno-associated virus vectors in the absence of helper adenovirus. J Virol 1998; 72: 2224-2232.
    • (1998) J Virol , vol.72 , pp. 2224-2232
    • Xiao, X.1    Li, J.2    Samulski, R.J.3
  • 30
    • 0032870158 scopus 로고    scopus 로고
    • Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2
    • Girod A, Ried M, Wobus C, et al. Genetic capsid modifications allow efficient re-targeting of adeno-associated virus type 2. Nat Med 1999; 5: 1052-1056.
    • (1999) Nat Med , vol.5 , pp. 1052-1056
    • Girod, A.1    Ried, M.2    Wobus, C.3
  • 31
    • 0033541640 scopus 로고    scopus 로고
    • Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses
    • Clark KR, Liu X, McGrath JP, Johnson PR. Highly purified recombinant adeno-associated virus vectors are biologically active and free of detectable helper and wild-type viruses. Hum Gene Ther 1999; 10: 1031-1039.
    • (1999) Hum Gene Ther , vol.10 , pp. 1031-1039
    • Clark, K.R.1    Liu, X.2    McGrath, J.P.3    Johnson, P.R.4
  • 32
    • 0014468579 scopus 로고
    • Improved technique of heart transplantation in rats
    • Ono K, Lindsey ES. Improved technique of heart transplantation in rats. J Thorac Cardiovasc Surg 1969; 57: 225-229.
    • (1969) J Thorac Cardiovasc Surg , vol.57 , pp. 225-229
    • Ono, K.1    Lindsey, E.S.2
  • 33
    • 0036776216 scopus 로고    scopus 로고
    • Use of an adenoviral vector to express soluble donor-major histocompatibility complex molecules capable of suppressing the immune response in rat transplant recipients
    • Graeb C, Justl M, Scherer MN, et al. Use of an adenoviral vector to express soluble donor-major histocompatibility complex molecules capable of suppressing the immune response in rat transplant recipients. Hum Immunol 2002; 63: 844-852.
    • (2002) Hum Immunol , vol.63 , pp. 844-852
    • Graeb, C.1    Justl, M.2    Scherer, M.N.3
  • 34
    • 4243248938 scopus 로고    scopus 로고
    • Soluble donor MHC class I gene transfer to thymus promotes allograft survival in a high-responder heart transplant model
    • Geissler EK, Scherer MN, Graeb C. Soluble donor MHC class I gene transfer to thymus promotes allograft survival in a high-responder heart transplant model. Transpl Int 2000; 13(Suppl 1): S452-S455.
    • (2000) Transpl Int , vol.13 , Issue.SUPPL. 1
    • Geissler, E.K.1    Scherer, M.N.2    Graeb, C.3
  • 35
    • 0032805251 scopus 로고    scopus 로고
    • Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA
    • Liu F, Song Y, Liu D. Hydrodynamics-based transfection in animals by systemic administration of plasmid DNA. Gene Ther 1999; 6: 1258-1266.
    • (1999) Gene Ther , vol.6 , pp. 1258-1266
    • Liu, F.1    Song, Y.2    Liu, D.3
  • 36
    • 0031018829 scopus 로고    scopus 로고
    • Isolated-organ perfusion for local gene delivery: efficient adenovirus-mediated gene transfer into the liver
    • de Roos WK, Fallaux FJ, Marinelli AW, et al. Isolated-organ perfusion for local gene delivery: efficient adenovirus-mediated gene transfer into the liver. Gene Ther 1997; 4: 55-62.
    • (1997) Gene Ther , vol.4 , pp. 55-62
    • de Roos, W.K.1    Fallaux, F.J.2    Marinelli, A.W.3
  • 37
    • 13044304200 scopus 로고    scopus 로고
    • Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons
    • Morral N, O'Neal W, Rice K, et al. Administration of helper-dependent adenoviral vectors and sequential delivery of different vector serotype for long-term liver-directed gene transfer in baboons. Proc Natl Acad Sci USA 1999; 96: 12816-12821.
    • (1999) Proc Natl Acad Sci USA , vol.96 , pp. 12816-12821
    • Morral, N.1    O'Neal, W.2    Rice, K.3
  • 38
    • 0030997346 scopus 로고    scopus 로고
    • Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors
    • Snyder RO, Miao CH, Patijn GA, et al. Persistent and therapeutic concentrations of human factor IX in mice after hepatic gene transfer of recombinant AAV vectors. Nat Genet 1997; 16: 270-276.
    • (1997) Nat Genet , vol.16 , pp. 270-276
    • Snyder, R.O.1    Miao, C.H.2    Patijn, G.A.3
  • 40
    • 19444365150 scopus 로고    scopus 로고
    • Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models
    • Davidoff AM, Gray JT, Ng CY, et al. Comparison of the ability of adeno-associated viral vectors pseudotyped with serotype 2, 5, and 8 capsid proteins to mediate efficient transduction of the liver in murine and nonhuman primate models. Mol Ther 2005; 11: 875-888.
    • (2005) Mol Ther , vol.11 , pp. 875-888
    • Davidoff, A.M.1    Gray, J.T.2    Ng, C.Y.3
  • 41
    • 20844460836 scopus 로고    scopus 로고
    • Increased hepatic transduction with reduced systemic dissemination and proinflammatory cytokines following hydrodynamic injection of helper-dependent adenoviral vectors
    • Brunetti-Pierri N, Palmer DJ, Mane V, et al. Increased hepatic transduction with reduced systemic dissemination and proinflammatory cytokines following hydrodynamic injection of helper-dependent adenoviral vectors. Mol Ther 2005; 12: 99-106.
    • (2005) Mol Ther , vol.12 , pp. 99-106
    • Brunetti-Pierri, N.1    Palmer, D.J.2    Mane, V.3
  • 42
    • 40549130686 scopus 로고    scopus 로고
    • Hydrodynamics-based delivery of plasmid DNA encoding CTLA4-Ig prolonged cardiac allograft survival in rats
    • Takekubo M, Tsuchida M, Haga M, et al. Hydrodynamics-based delivery of plasmid DNA encoding CTLA4-Ig prolonged cardiac allograft survival in rats. J Gene Med 2007; 10: 290-297.
    • (2007) J Gene Med , vol.10 , pp. 290-297
    • Takekubo, M.1    Tsuchida, M.2    Haga, M.3
  • 43
    • 0033893570 scopus 로고    scopus 로고
    • Long-term expression of human alpha1-antitrypsin gene in mouse liver achieved by intravenous administration of plasmid DNA using a hydrodynamics-based procedure
    • Zhang G, Song YK, Liu D. Long-term expression of human alpha1-antitrypsin gene in mouse liver achieved by intravenous administration of plasmid DNA using a hydrodynamics-based procedure. Gene Ther 2000; 7: 1344-1349.
    • (2000) Gene Ther , vol.7 , pp. 1344-1349
    • Zhang, G.1    Song, Y.K.2    Liu, D.3
  • 44
    • 31444439735 scopus 로고    scopus 로고
    • Intranuclear disposition of exogenous DNA in vivo: silencing, methylation and fragmentation
    • Ochiai H, Harashima H, Kamiya H. Intranuclear disposition of exogenous DNA in vivo: silencing, methylation and fragmentation. FEBS Lett 2006; 580: 918-922.
    • (2006) FEBS Lett , vol.580 , pp. 918-922
    • Ochiai, H.1    Harashima, H.2    Kamiya, H.3
  • 45
    • 0041911490 scopus 로고    scopus 로고
    • Long-term therapeutic levels of human alpha-1 antitrypsin in plasma after hydrodynamic injection of nonviral DNA
    • Alino SF, Crespo A, Dasi F. Long-term therapeutic levels of human alpha-1 antitrypsin in plasma after hydrodynamic injection of nonviral DNA. Gene Ther 2003; 10: 1672-1679.
    • (2003) Gene Ther , vol.10 , pp. 1672-1679
    • Alino, S.F.1    Crespo, A.2    Dasi, F.3
  • 46
    • 0141539412 scopus 로고    scopus 로고
    • High-level factor VIII gene expression in vivo achieved by nonviral liver-specific gene therapy vectors
    • Miao CH, Ye X, Thompson AR. High-level factor VIII gene expression in vivo achieved by nonviral liver-specific gene therapy vectors. Hum Gene Ther 2003; 14: 1297-1305.
    • (2003) Hum Gene Ther , vol.14 , pp. 1297-1305
    • Miao, C.H.1    Ye, X.2    Thompson, A.R.3
  • 47
    • 0041315884 scopus 로고    scopus 로고
    • Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo
    • Chen ZY, He CY, Ehrhardt A, Kay MA. Minicircle DNA vectors devoid of bacterial DNA result in persistent and high-level transgene expression in vivo. Mol Ther 2003; 8: 495-500.
    • (2003) Mol Ther , vol.8 , pp. 495-500
    • Chen, Z.Y.1    He, C.Y.2    Ehrhardt, A.3    Kay, M.A.4
  • 48
    • 2642519089 scopus 로고    scopus 로고
    • Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo
    • Chen ZY, He CY, Meuse L, Kay MA. Silencing of episomal transgene expression by plasmid bacterial DNA elements in vivo. Gene Ther 2004; 11: 856-864.
    • (2004) Gene Ther , vol.11 , pp. 856-864
    • Chen, Z.Y.1    He, C.Y.2    Meuse, L.3    Kay, M.A.4
  • 49
    • 25144492340 scopus 로고    scopus 로고
    • Sleeping beauty transposon-mediated gene therapy for prolonged expression
    • Hackett PB, Ekker SC, Largaespada DA, McIvor RS. Sleeping beauty transposon-mediated gene therapy for prolonged expression. Adv Genet 2005; 54: 189-232.
    • (2005) Adv Genet , vol.54 , pp. 189-232
    • Hackett, P.B.1    Ekker, S.C.2    Largaespada, D.A.3    McIvor, R.S.4
  • 50
    • 1842689863 scopus 로고    scopus 로고
    • Transposon-independent increase of transcription by the Sleeping Beauty transposase
    • Masuda K, Yamamoto S, Endoh M, Kaneda Y. Transposon-independent increase of transcription by the Sleeping Beauty transposase. Biochem Biophys Res Commun 2004; 317: 796-800.
    • (2004) Biochem Biophys Res Commun , vol.317 , pp. 796-800
    • Masuda, K.1    Yamamoto, S.2    Endoh, M.3    Kaneda, Y.4
  • 51
    • 0142213094 scopus 로고    scopus 로고
    • Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo
    • Mikkelsen JG, Yant SR, Meuse L, Huang Z, Xu H, Kay MA. Helper-Independent Sleeping Beauty transposon-transposase vectors for efficient nonviral gene delivery and persistent gene expression in vivo. Mol Ther 2003; 8: 654-665.
    • (2003) Mol Ther , vol.8 , pp. 654-665
    • Mikkelsen, J.G.1    Yant, S.R.2    Meuse, L.3    Huang, Z.4    Xu, H.5    Kay, M.A.6
  • 52
    • 0033555686 scopus 로고    scopus 로고
    • A vector based on the SV40 origin of replication and chromosomal S/MARs replicates episomally in CHO cells
    • Piechaczek C, Fetzer C, Baiker A, Bode J, Lipps HJ. A vector based on the SV40 origin of replication and chromosomal S/MARs replicates episomally in CHO cells. Nucleic Acids Res 1999; 27: 426-428.
    • (1999) Nucleic Acids Res , vol.27 , pp. 426-428
    • Piechaczek, C.1    Fetzer, C.2    Baiker, A.3    Bode, J.4    Lipps, H.J.5
  • 53
    • 3843139313 scopus 로고    scopus 로고
    • Nuclear scaffold/matrix attached region modules linked to a transcription unit are sufficient for replication and maintenance of a mammalian episome
    • Jenke AC, Stehle IM, Herrmann F, et al. Nuclear scaffold/matrix attached region modules linked to a transcription unit are sufficient for replication and maintenance of a mammalian episome. Proc Natl Acad Sci USA 2004; 101: 11322-11327.
    • (2004) Proc Natl Acad Sci USA , vol.101 , pp. 11322-11327
    • Jenke, A.C.1    Stehle, I.M.2    Herrmann, F.3
  • 54
    • 0034960344 scopus 로고    scopus 로고
    • Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo
    • Nakai H, Yant SR, Storm TA, Fuess S, Meuse L, Kay MA. Extrachromosomal recombinant adeno-associated virus vector genomes are primarily responsible for stable liver transduction in vivo. J Virol 2001; 75: 6969-6976.
    • (2001) J Virol , vol.75 , pp. 6969-6976
    • Nakai, H.1    Yant, S.R.2    Storm, T.A.3    Fuess, S.4    Meuse, L.5    Kay, M.A.6
  • 55
    • 27744533048 scopus 로고    scopus 로고
    • Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector
    • Conlon TJ, Cossette T, Erger K, et al. Efficient hepatic delivery and expression from a recombinant adeno-associated virus 8 pseudotyped alpha1-antitrypsin vector. Mol Ther 2005; 12: 867-875.
    • (2005) Mol Ther , vol.12 , pp. 867-875
    • Conlon, T.J.1    Cossette, T.2    Erger, K.3
  • 56
    • 0037221408 scopus 로고    scopus 로고
    • Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice
    • Nakai H, Montini E, Fuess S, et al. Helper-independent and AAV-ITR-independent chromosomal integration of double-stranded linear DNA vectors in mice. Mol Ther 2003; 7: 101-111.
    • (2003) Mol Ther , vol.7 , pp. 101-111
    • Nakai, H.1    Montini, E.2    Fuess, S.3
  • 57
    • 34548761857 scopus 로고    scopus 로고
    • Existence of transient functional double-stranded DNA intermediates during recombinant AAV transduction
    • Wang J, Xie J, Lu H, et al. Existence of transient functional double-stranded DNA intermediates during recombinant AAV transduction. Proc Natl Acad Sci USA 2007; 104: 13104-13109.
    • (2007) Proc Natl Acad Sci USA , vol.104 , pp. 13104-13109
    • Wang, J.1    Xie, J.2    Lu, H.3
  • 58
    • 40249118457 scopus 로고    scopus 로고
    • Hydrodynamic gene delivery to the pig liver via an isolated segment of the inferior vena cava
    • Fabre JW, Grehan A, Whitehorne M, et al. Hydrodynamic gene delivery to the pig liver via an isolated segment of the inferior vena cava. Gene Ther 2008; 15: 452-462.
    • (2008) Gene Ther , vol.15 , pp. 452-462
    • Fabre, J.W.1    Grehan, A.2    Whitehorne, M.3
  • 59
    • 33845209883 scopus 로고    scopus 로고
    • Naked plasmid DNA transfer to the porcine liver using rapid injection with large volume
    • Yoshino H, Hashizume K, Kobayashi E. Naked plasmid DNA transfer to the porcine liver using rapid injection with large volume. Gene Ther 2006; 13: 1696-1702.
    • (2006) Gene Ther , vol.13 , pp. 1696-1702
    • Yoshino, H.1    Hashizume, K.2    Kobayashi, E.3
  • 60
    • 63649138339 scopus 로고    scopus 로고
    • Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates
    • Brunetti-Pierri N, Stapleton GE, Law M, et al. Efficient, long-term hepatic gene transfer using clinically relevant HDAd doses by balloon occlusion catheter delivery in nonhuman primates. Mol Ther 2009; 17: 327-333.
    • (2009) Mol Ther , vol.17 , pp. 327-333
    • Brunetti-Pierri, N.1    Stapleton, G.E.2    Law, M.3
  • 61
    • 33644820684 scopus 로고    scopus 로고
    • Successful transduction of liver in haemophilia by AAV-Factor IX and limitations imposed by the host immune response
    • Manno CS, Pierce GF, Arruda VR, et al. Successful transduction of liver in haemophilia by AAV-Factor IX and limitations imposed by the host immune response. Nat Med 2006; 12: 342-347.
    • (2006) Nat Med , vol.12 , pp. 342-347
    • Manno, C.S.1    Pierce, G.F.2    Arruda, V.R.3
  • 62
    • 77958608600 scopus 로고    scopus 로고
    • AAV gene vector manufacturing: what is feasible now and in the near future? Workshop. 24 June 2006; Méridien Beach Plaza Hotel, Monte Carlo,
    • Danos O. AAV gene vector manufacturing: what is feasible now and in the near future? Workshop. 24 June 2006; Méridien Beach Plaza Hotel, Monte Carlo, 2006.
    • (2006)
    • Danos, O.1
  • 63
    • 33747123681 scopus 로고    scopus 로고
    • Successful parallel development and integration of a plasmid-based biologic, container/closure system and electrokinetic delivery device
    • Hebel H, Attra H, Khan A, Draghia-Akli R. Successful parallel development and integration of a plasmid-based biologic, container/closure system and electrokinetic delivery device. Vaccine 2006; 24: 4607-4614.
    • (2006) Vaccine , vol.24 , pp. 4607-4614
    • Hebel, H.1    Attra, H.2    Khan, A.3    Draghia-Akli, R.4
  • 64
    • 77958533281 scopus 로고    scopus 로고
    • The Vector Directory: An Analysis of International GMP Gene Therapy Vector Capacity and Capability
    • London: Department of Health: .
    • Watson R. The Vector Directory: An Analysis of International GMP Gene Therapy Vector Capacity and Capability. London: Department of Health: 2007.
    • (2007)
    • Watson, R.1
  • 65
    • 4344715026 scopus 로고    scopus 로고
    • A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs
    • Hagstrom JE, Hegge J, Zhang G, et al. A facile nonviral method for delivering genes and siRNAs to skeletal muscle of mammalian limbs. Mol Ther 2004; 10: 386-398.
    • (2004) Mol Ther , vol.10 , pp. 386-398
    • Hagstrom, J.E.1    Hegge, J.2    Zhang, G.3
  • 66
    • 4344700281 scopus 로고    scopus 로고
    • Opening the floodgates: clinically applicable hydrodynamic delivery of plasmid DNA to skeletal muscle
    • Wells DJ. Opening the floodgates: clinically applicable hydrodynamic delivery of plasmid DNA to skeletal muscle. Mol Ther 2004; 10: 207-208.
    • (2004) Mol Ther , vol.10 , pp. 207-208
    • Wells, D.J.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.