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Volumn 15, Issue 3, 2007, Pages 445-456

The genetic engineering of hematopoietic stem cells: The rise of lentiviral vectors, the conundrum of the LTR, and the promise of lineage-restricted vectors

Author keywords

[No Author keywords available]

Indexed keywords

BETA GLOBIN; BLOOD CLOTTING FACTOR 9; CARMUSTINE; LENTIVIRUS VECTOR; PHOSPHOGLYCERATE KINASE; PROTEASOME INHIBITOR; RETROVIRUS VECTOR; WISKOTT ALDRICH SYNDROME PROTEIN;

EID: 33847205752     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/sj.mt.6300060     Document Type: Review
Times cited : (77)

References (232)
  • 1
    • 0029887997 scopus 로고    scopus 로고
    • Gene transfer to hematopoietic stem cells: Implications for gene therapy of human disease
    • Dunbar, CE (1996). Gene transfer to hematopoietic stem cells: implications for gene therapy of human disease. Annu Rev Med 47: 11-20.
    • (1996) Annu Rev Med , vol.47 , pp. 11-20
    • Dunbar, C.E.1
  • 2
    • 37649026269 scopus 로고    scopus 로고
    • Adult bone marrow stem cells for cell and gene therapies: Implications for greater use
    • Ballas, CB, Zielske, SP and Gerson, SL (2002). Adult bone marrow stem cells for cell and gene therapies: Implications for greater use. J Cell Biochem Suppl 38: 20-28.
    • (2002) J Cell Biochem Suppl , vol.38 , pp. 20-28
    • Ballas, C.B.1    Zielske, S.P.2    Gerson, S.L.3
  • 3
    • 0242362722 scopus 로고    scopus 로고
    • Gene therapy for inborn and acquired immune deficiency disorders
    • Engel, BC and Kohn, DB (2003). Gene therapy for inborn and acquired immune deficiency disorders. Acta Haematol 110: 60-70.
    • (2003) Acta Haematol , vol.110 , pp. 60-70
    • Engel, B.C.1    Kohn, D.B.2
  • 4
    • 0030999174 scopus 로고    scopus 로고
    • High-level globin gene expression mediated by a recombinant adeno-associated virus genome that contains the 3′-γ globin gene regulatory element and integrates as tandem copies in erythroid cells
    • Hargrove, PW, Vanin, EF, Kurtzman, GJ and Nienhuis, AW (1997). High-level globin gene expression mediated by a recombinant adeno-associated virus genome that contains the 3′-γ globin gene regulatory element and integrates as tandem copies in erythroid cells. Blood 89: 2167-2175.
    • (1997) Blood , vol.89 , pp. 2167-2175
    • Hargrove, P.W.1    Vanin, E.F.2    Kurtzman, G.J.3    Nienhuis, A.W.4
  • 5
    • 22144476522 scopus 로고    scopus 로고
    • Hematopoietic stem cell transduction by recombinant adeno-associated virus vectors: Problems and solutions
    • Srivastava, A (2005). Hematopoietic stem cell transduction by recombinant adeno-associated virus vectors: Problems and solutions. Hum Gene Ther 16: 792-798.
    • (2005) Hum Gene Ther , vol.16 , pp. 792-798
    • Srivastava, A.1
  • 7
    • 24344495491 scopus 로고    scopus 로고
    • Awakening gene therapy with Sleeping Beauty transposons
    • Essner, JJ, McIvor, RS and Hackett, PB (2005). Awakening gene therapy with Sleeping Beauty transposons. Curr Opin Pharmacol 5: 513-519.
    • (2005) Curr Opin Pharmacol , vol.5 , pp. 513-519
    • Essner, J.J.1    McIvor, R.S.2    Hackett, P.B.3
  • 8
    • 18944373328 scopus 로고    scopus 로고
    • Highly efficient endogenous human gene correction using designed zinc-finger nucleases
    • Urnov, FD et al. (2005). Highly efficient endogenous human gene correction using designed zinc-finger nucleases. Nature 435: 646-651.
    • (2005) Nature , vol.435 , pp. 646-651
    • Urnov, F.D.1
  • 9
    • 27344439351 scopus 로고    scopus 로고
    • Complete and persistent phenotypic correction of phenylketonuria in mice by site-specific genome integration of murine phenylalanine hydroxylase cDNA
    • Chen, L and Woo, SL (2005). Complete and persistent phenotypic correction of phenylketonuria in mice by site-specific genome integration of murine phenylalanine hydroxylase cDNA. Proc Natl Acad Sci USA 102: 15581-15586.
    • (2005) Proc Natl Acad Sci USA , vol.102 , pp. 15581-15586
    • Chen, L.1    Woo, S.L.2
  • 10
    • 10744233858 scopus 로고    scopus 로고
    • American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells
    • Kohn, DB et al. (2003). American Society of Gene Therapy (ASGT) ad hoc subcommittee on retroviral-mediated gene transfer to hematopoietic stem cells. Mol Ther 8: 180-187.
    • (2003) Mol Ther , vol.8 , pp. 180-187
    • Kohn, D.B.1
  • 11
    • 0029996147 scopus 로고    scopus 로고
    • In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
    • Naldini, L et al. (1996). In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 272: 263-267.
    • (1996) Science , vol.272 , pp. 263-267
    • Naldini, L.1
  • 12
    • 0031747965 scopus 로고    scopus 로고
    • Human immunodeficiency virus type 1 vectors efficiently transduce human hematopoietic stem cells
    • Sutton, RE, Wu, HT, Rigg, R, Bohnlein, E and Brown, PO (1998). Human immunodeficiency virus type 1 vectors efficiently transduce human hematopoietic stem cells. J Virol 72: 5781-5788.
    • (1998) J Virol , vol.72 , pp. 5781-5788
    • Sutton, R.E.1    Wu, H.T.2    Rigg, R.3    Bohnlein, E.4    Brown, P.O.5
  • 13
    • 0032578385 scopus 로고    scopus 로고
    • 1 human hematopoietic stem cells
    • 1 human hematopoietic stem cells. Proc Natl Acad Sci USA 95: 11939-11944.
    • (1998) Proc Natl Acad Sci USA , vol.95 , pp. 11939-11944
    • Uchida, N.1
  • 15
    • 13044309472 scopus 로고    scopus 로고
    • - human hematopoietic cells by HIV-1-based lentiviral vectors
    • - human hematopoietic cells by HIV-1-based lentiviral vectors. Proc Natl Acad Sci USA 96: 2988-2993.
    • (1999) Proc Natl Acad Sci USA , vol.96 , pp. 2988-2993
    • Case, S.S.1
  • 16
    • 0343628721 scopus 로고    scopus 로고
    • Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin
    • May, C et al. (2000). Therapeutic haemoglobin synthesis in β-thalassaemic mice expressing lentivirus-encoded human β-globin. Nature 406: 82-86.
    • (2000) Nature , vol.406 , pp. 82-86
    • May, C.1
  • 18
    • 0035437131 scopus 로고    scopus 로고
    • Gene transfer into murine hematopoietic stem cells with helper-free foamy virus vectors
    • Vassilopoulos, G, Trobridge, G, Josephson, NC and Russell, DW (2001). Gene transfer into murine hematopoietic stem cells with helper-free foamy virus vectors. Blood 98: 604-609.
    • (2001) Blood , vol.98 , pp. 604-609
    • Vassilopoulos, G.1    Trobridge, G.2    Josephson, N.C.3    Russell, D.W.4
  • 19
    • 31944444349 scopus 로고    scopus 로고
    • Foamy virus vector integration sites in normal human cells
    • Trobridge, GD et al. (2006). Foamy virus vector integration sites in normal human cells. Proc Natl Acad Sci USA 103: 1498-1503.
    • (2006) Proc Natl Acad Sci USA , vol.103 , pp. 1498-1503
    • Trobridge, G.D.1
  • 20
    • 0021355123 scopus 로고
    • Aging and hematopoiesis. II. The ability of bone marrow cells from young and aged mice to cure and maintain cure in W/Wv
    • Boggs, DR, Saxe, DF and Boggs, SS (1984). Aging and hematopoiesis. II. The ability of bone marrow cells from young and aged mice to cure and maintain cure in W/Wv. Transplantation 37: 300-306.
    • (1984) Transplantation , vol.37 , pp. 300-306
    • Boggs, D.R.1    Saxe, D.F.2    Boggs, S.S.3
  • 21
    • 0025205255 scopus 로고
    • Quantitative assay for totipotent reconstituting hematopoietic stem cells by a competitive repopulation strategy
    • Szilvassy, SJ, Humphries, RK, Lansdorp, PM, Eaves, AC and Eaves, CJ (1990). Quantitative assay for totipotent reconstituting hematopoietic stem cells by a competitive repopulation strategy. Proc Natl Acad Sci USA 87: 8736-8740.
    • (1990) Proc Natl Acad Sci USA , vol.87 , pp. 8736-8740
    • Szilvassy, S.J.1    Humphries, R.K.2    Lansdorp, P.M.3    Eaves, A.C.4    Eaves, C.J.5
  • 22
    • 0038148364 scopus 로고    scopus 로고
    • Hematopoietic stem cells engraft in mice with absolute efficiency
    • Benveniste, P, Cantin, C, Hyam, D and Iscove, NN (2003). Hematopoietic stem cells engraft in mice with absolute efficiency. Nat Immunol 4: 708-713.
    • (2003) Nat Immunol , vol.4 , pp. 708-713
    • Benveniste, P.1    Cantin, C.2    Hyam, D.3    Iscove, N.N.4
  • 23
    • 0030914867 scopus 로고    scopus 로고
    • Quiescence, cycling, and turnover in the primitive hematopoietic stem cell compartment
    • Bradford, GB, Williams, B, Rossi, R and Bertoncello, I (1997). Quiescence, cycling, and turnover in the primitive hematopoietic stem cell compartment. Exp Hematol 25: 445-453.
    • (1997) Exp Hematol , vol.25 , pp. 445-453
    • Bradford, G.B.1    Williams, B.2    Rossi, R.3    Bertoncello, I.4
  • 24
    • 0035874515 scopus 로고    scopus 로고
    • The relative quiescence of hematopoietic stem cells in nonhuman primates
    • Mahmud, N et al. (2001). The relative quiescence of hematopoietic stem cells in nonhuman primates. Blood 97: 3061-3068.
    • (2001) Blood , vol.97 , pp. 3061-3068
    • Mahmud, N.1
  • 25
    • 0032980506 scopus 로고    scopus 로고
    • In vivo proliferation and cell cycle kinetics of long-term self-renewing hematopoietic stem cells
    • Cheshier, SH, Morrison, SJ, Liao, X and Weissman, IL (1999). In vivo proliferation and cell cycle kinetics of long-term self-renewing hematopoietic stem cells. Proc Natl Acad Sci USA 96: 3120-3125.
    • (1999) Proc Natl Acad Sci USA , vol.96 , pp. 3120-3125
    • Cheshier, S.H.1    Morrison, S.J.2    Liao, X.3    Weissman, I.L.4
  • 26
    • 0024264749 scopus 로고
    • Polyclonal reconstitution of human marrow after allogeneic bone marrow transplantation
    • Nash, R, Storb, R and Neiman, P (1988). Polyclonal reconstitution of human marrow after allogeneic bone marrow transplantation. Blood 72: 2031-2037.
    • (1988) Blood , vol.72 , pp. 2031-2037
    • Nash, R.1    Storb, R.2    Neiman, P.3
  • 27
    • 0028953448 scopus 로고
    • Behavior of hematopoietic stem cells in a large animal
    • Abkowitz, JL et al. (1995). Behavior of hematopoietic stem cells in a large animal. Proc Natl Acad Sci USA 92: 2031-2035.
    • (1995) Proc Natl Acad Sci USA , vol.92 , pp. 2031-2035
    • Abkowitz, J.L.1
  • 28
    • 0345643319 scopus 로고    scopus 로고
    • Telomere fluorescence measurements in granulocytes and T lymphocyte subsets point to a high turnover of hematopoietic stem cells and memory T cells in early childhood
    • Rufer, N et al. (1999). Telomere fluorescence measurements in granulocytes and T lymphocyte subsets point to a high turnover of hematopoietic stem cells and memory T cells in early childhood. J Exp Med 190: 157-167.
    • (1999) J Exp Med , vol.190 , pp. 157-167
    • Rufer, N.1
  • 29
    • 0002234927 scopus 로고    scopus 로고
    • Many multipotential gene-marked progenitor or stem cell clones contribute to hematopoiesis in nonhuman primates
    • Kim, HJ et al. (2000). Many multipotential gene-marked progenitor or stem cell clones contribute to hematopoiesis in nonhuman primates. Blood 96: 1-8.
    • (2000) Blood , vol.96 , pp. 1-8
    • Kim, H.J.1
  • 30
    • 0028055281 scopus 로고
    • Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus
    • Lewis, PF and Emerman, M (1994). Passage through mitosis is required for oncoretroviruses but not for the human immunodeficiency virus. J Virol 68: 510-516.
    • (1994) J Virol , vol.68 , pp. 510-516
    • Lewis, P.F.1    Emerman, M.2
  • 31
    • 0031594498 scopus 로고    scopus 로고
    • 1b phase of the cell cycle is required for completion of human immunodeficiency virus type 1 reverse transcription in T cells
    • 1b phase of the cell cycle is required for completion of human immunodeficiency virus type 1 reverse transcription in T cells. J Virol 72: 3161-3168.
    • (1998) J Virol , vol.72 , pp. 3161-3168
    • Korin, Y.D.1    Zack, J.A.2
  • 32
    • 0027376309 scopus 로고
    • A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells
    • Bukrinsky, MI et al. (1993). A nuclear localization signal within HIV-1 matrix protein that governs infection of non-dividing cells. Nature 365: 666-669.
    • (1993) Nature , vol.365 , pp. 666-669
    • Bukrinsky, M.I.1
  • 33
    • 0028821383 scopus 로고
    • HIV-1 infection of nondividing cells: C-terminal tyrosine phosphorylation of the viral matrix protein is a key regulator
    • Gallay, P, Swingler, S, Aiken, C and Trono, D (1995). HIV-1 infection of nondividing cells: C-terminal tyrosine phosphorylation of the viral matrix protein is a key regulator. Cell 80: 379-388.
    • (1995) Cell , vol.80 , pp. 379-388
    • Gallay, P.1    Swingler, S.2    Aiken, C.3    Trono, D.4
  • 34
    • 0028239060 scopus 로고
    • The Vpr protein of human immunodeficiency virus type 1 influences nuclear localization of viral nucleic acids in nondividing host cells
    • Heinzinger, NK et al. (1994). The Vpr protein of human immunodeficiency virus type 1 influences nuclear localization of viral nucleic acids in nondividing host cells. Proc Natl Acad Sci USA 91: 7311-7315.
    • (1994) Proc Natl Acad Sci USA , vol.91 , pp. 7311-7315
    • Heinzinger, N.K.1
  • 35
    • 0032557445 scopus 로고    scopus 로고
    • Viral protein R regulates docking of the HIV-1 preintegration complex to the nuclear pore complex
    • Popov, S, Rexach, M, Ratner, L, Blobel, G and Bukrinsky, M (1998). Viral protein R regulates docking of the HIV-1 preintegration complex to the nuclear pore complex. J Biol Chem 273: 13347-13352.
    • (1998) J Biol Chem , vol.273 , pp. 13347-13352
    • Popov, S.1    Rexach, M.2    Ratner, L.3    Blobel, G.4    Bukrinsky, M.5
  • 36
    • 0030987672 scopus 로고    scopus 로고
    • HIV-1 infection of nondividing cells through the recognition of integrase by the importin/karyopherin pathway
    • Gallay, P, Hope, T, Chin, D and Trono, D (1997). HIV-1 infection of nondividing cells through the recognition of integrase by the importin/ karyopherin pathway. Proc Natl Acad Sci USA 94: 9825-9830.
    • (1997) Proc Natl Acad Sci USA , vol.94 , pp. 9825-9830
    • Gallay, P.1    Hope, T.2    Chin, D.3    Trono, D.4
  • 37
    • 0035524975 scopus 로고    scopus 로고
    • Intracellular trafficking of retroviral genomes during the early phase of infection: Viral exploitation of cellular pathways
    • Goff, SP (2001). Intracellular trafficking of retroviral genomes during the early phase of infection: Viral exploitation of cellular pathways. J Gene Med 3: 517-528.
    • (2001) J Gene Med , vol.3 , pp. 517-528
    • Goff, S.P.1
  • 38
    • 0030819379 scopus 로고    scopus 로고
    • Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo
    • Zufferey, R, Nagy, D, Mandel, RJ, Naldini, L and Trono, D (1997). Multiply attenuated lentiviral vector achieves efficient gene delivery in vivo. Nat Biotechnol 15: 871-875.
    • (1997) Nat Biotechnol , vol.15 , pp. 871-875
    • Zufferey, R.1    Nagy, D.2    Mandel, R.J.3    Naldini, L.4    Trono, D.5
  • 39
    • 0034672356 scopus 로고    scopus 로고
    • The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells
    • Sirven, A et al. (2000). The human immunodeficiency virus type-1 central DNA flap is a crucial determinant for lentiviral vector nuclear import and gene transduction of human hematopoietic stem cells. Blood 96: 4103-4110.
    • (2000) Blood , vol.96 , pp. 4103-4110
    • Sirven, A.1
  • 40
    • 0034040751 scopus 로고    scopus 로고
    • Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences
    • Follenzi, A, Ailles, LE, Bakovic, S, Geuna, M and Naldini, L (2000). Gene transfer by lentiviral vectors is limited by nuclear translocation and rescued by HIV-1 pol sequences. Nat Genet 25: 217-222.
    • (2000) Nat Genet , vol.25 , pp. 217-222
    • Follenzi, A.1    Ailles, L.E.2    Bakovic, S.3    Geuna, M.4    Naldini, L.5
  • 42
    • 34248594865 scopus 로고    scopus 로고
    • The cell cycle independence of HIV infections is not determined by known karyophilic viral elements
    • Yamashita, M and Emerman, M (2005). The cell cycle independence of HIV infections is not determined by known karyophilic viral elements. PLoS Pathog 1: E18.
    • (2005) PLoS Pathog , vol.1
    • Yamashita, M.1    Emerman, M.2
  • 44
    • 0034199171 scopus 로고    scopus 로고
    • - bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors
    • - bone marrow and cord blood-derived SCID-repopulating cells with third-generation lentiviral vectors. Mol Ther 1: 566-573.
    • (2000) Mol Ther , vol.1 , pp. 566-573
    • Guenechea, G.1
  • 45
    • 0032956732 scopus 로고    scopus 로고
    • Transduction of human progenitor hematopoietic stem cells by human immunodeficiency virus type 1-based vectors is cell cycle dependent
    • Sutton, RE, Reitsma, MJ, Uchida, N and Brown, PO (1999). Transduction of human progenitor hematopoietic stem cells by human immunodeficiency virus type 1-based vectors is cell cycle dependent. J Virol 73: 3649-3660.
    • (1999) J Virol , vol.73 , pp. 3649-3660
    • Sutton, R.E.1    Reitsma, M.J.2    Uchida, N.3    Brown, P.O.4
  • 46
    • 0032795284 scopus 로고    scopus 로고
    • Nonproductive human immunodeficiency virus type 1 infection in nucleoside-treated G0 lymphocytes
    • Korin, YD and Zack, JA (1999). Nonproductive human immunodeficiency virus type 1 infection in nucleoside-treated G0 lymphocytes. J Virol 73: 6526-6532.
    • (1999) J Virol , vol.73 , pp. 6526-6532
    • Korin, Y.D.1    Zack, J.A.2
  • 47
    • 0025342694 scopus 로고
    • HIV-1 entry into quiescent primary lymphocytes: Molecular analysis reveals a labile, latent viral structure
    • Zack, JA, Arrigo, SJ, Weitsman, SR, Go, AS, Haislip, A and Chen, IS (1990). HIV-1 entry into quiescent primary lymphocytes: Molecular analysis reveals a labile, latent viral structure. Cell 61: 213-222.
    • (1990) Cell , vol.61 , pp. 213-222
    • Zack, J.A.1    Arrigo, S.J.2    Weitsman, S.R.3    Go, A.S.4    Haislip, A.5    Chen, I.S.6
  • 48
    • 0026526144 scopus 로고
    • Incompletely reverse-transcribed human immunodeficiency virus type 1 genomes in quiescent cells can function as intermediates in the retroviral life cycle
    • Zack, JA, Haislip, AM, Krogstad, P and Chen, IS (1992). Incompletely reverse-transcribed human immunodeficiency virus type 1 genomes in quiescent cells can function as intermediates in the retroviral life cycle. J Virol 66: 1717-1725.
    • (1992) J Virol , vol.66 , pp. 1717-1725
    • Zack, J.A.1    Haislip, A.M.2    Krogstad, P.3    Chen, I.S.4
  • 49
    • 0032573177 scopus 로고    scopus 로고
    • Reduction in levels of the cyclin-dependent kinase inhibitor p27(kip-1) coupled with transforming growth factor beta neutralization induces cell-cycle entry and increases retroviral transduction of primitive human hematopoietic cells
    • Dao, MA, Taylor, N and Nolta, JA (1998). Reduction in levels of the cyclin-dependent kinase inhibitor p27(kip-1) coupled with transforming growth factor beta neutralization induces cell-cycle entry and increases retroviral transduction of primitive human hematopoietic cells. Proc Natl Acad Sci USA 95: 13006-13011.
    • (1998) Proc Natl Acad Sci USA , vol.95 , pp. 13006-13011
    • Dao, M.A.1    Taylor, N.2    Nolta, J.A.3
  • 50
    • 0034629129 scopus 로고    scopus 로고
    • Hematopoietic stem cell quiescence maintained by p21cip1/waf1
    • Cheng, T et al. (2000). Hematopoietic stem cell quiescence maintained by p21cip1/waf1. Science 287: 1804-1808.
    • (2000) Science , vol.287 , pp. 1804-1808
    • Cheng, T.1
  • 51
    • 0043240260 scopus 로고    scopus 로고
    • Ex vivo targeting of p21Cip1/Waf1 permits relative expansion of human hematopoietic stem cells
    • Stier, S et al. (2003). Ex vivo targeting of p21Cip1/Waf1 permits relative expansion of human hematopoietic stem cells. Blood 102: 1260-1266.
    • (2003) Blood , vol.102 , pp. 1260-1266
    • Stier, S.1
  • 52
    • 27144543231 scopus 로고    scopus 로고
    • Silencing p21(Waf1/Cip1/Sdi1) expression increases gene transduction efficiency in primitive human hematopoietic cells
    • Zhang, J, Attar, E, Cohen, K, Crumpacker, C and Scadden, D (2005). Silencing p21(Waf1/Cip1/Sdi1) expression increases gene transduction efficiency in primitive human hematopoietic cells. Gene Ther 12: 1444-1452.
    • (2005) Gene Ther , vol.12 , pp. 1444-1452
    • Zhang, J.1    Attar, E.2    Cohen, K.3    Crumpacker, C.4    Scadden, D.5
  • 53
    • 0031870460 scopus 로고    scopus 로고
    • The fluctuating phenotype of the lymphohematopoietic stem cell with cell cycle transit
    • Habibian, HK et al. (1998). The fluctuating phenotype of the lymphohematopoietic stem cell with cell cycle transit. J Exp Med 188: 393-398.
    • (1998) J Exp Med , vol.188 , pp. 393-398
    • Habibian, H.K.1
  • 55
    • 0032535694 scopus 로고    scopus 로고
    • Adhesion to fibronectin maintains regenerative capacity during ex vivo culture and transduction of human hematopoietic stem and progenitor cells
    • Dao, MA, Hashino, K, Kato, I and Nolta, JA (1998). Adhesion to fibronectin maintains regenerative capacity during ex vivo culture and transduction of human hematopoietic stem and progenitor cells. Blood 92: 4612-4621.
    • (1998) Blood , vol.92 , pp. 4612-4621
    • Dao, M.A.1    Hashino, K.2    Kato, I.3    Nolta, J.A.4
  • 57
    • 1542373663 scopus 로고    scopus 로고
    • + cells in the NOD/SCID model
    • + cells in the NOD/SCID model. Blood 103: 2079-2087.
    • (2004) Blood , vol.103 , pp. 2079-2087
    • Ahmed, F.1
  • 58
    • 2542452856 scopus 로고    scopus 로고
    • Effect of chronic cytokine therapy on clonal dynamics in nonhuman primates
    • Kuramoto, K et al. (2004). Effect of chronic cytokine therapy on clonal dynamics in nonhuman primates. Blood 103: 4070-4077.
    • (2004) Blood , vol.103 , pp. 4070-4077
    • Kuramoto, K.1
  • 59
    • 27944484376 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene therapy: Dead or alive?
    • Ferguson, C, Larochelle, A and Dunbar, CE (2005). Hematopoietic stem cell gene therapy: Dead or alive? Trends Biotechnol 23: 589-597.
    • (2005) Trends Biotechnol , vol.23 , pp. 589-597
    • Ferguson, C.1    Larochelle, A.2    Dunbar, C.E.3
  • 60
    • 0027419955 scopus 로고
    • Steel factor influences the distribution and activity of murine hematopoietic stem cells in vivo
    • Fleming, WH, Alpern, EJ, Uchida, N, Ikuta, K and Weissman, IL (1993). Steel factor influences the distribution and activity of murine hematopoietic stem cells in vivo. Proc Natl Acad Sci USA 90: 3760-3764.
    • (1993) Proc Natl Acad Sci USA , vol.90 , pp. 3760-3764
    • Fleming, W.H.1    Alpern, E.J.2    Uchida, N.3    Ikuta, K.4    Weissman, I.L.5
  • 61
    • 0029859412 scopus 로고    scopus 로고
    • Improved retroviral gene transfer into murine and Rhesus peripheral blood or bone marrow repopulating cells primed in vivo with stem cell factor and granulocyte colony-stimulating factor
    • Dunbar, CE et al. (1996). Improved retroviral gene transfer into murine and Rhesus peripheral blood or bone marrow repopulating cells primed in vivo with stem cell factor and granulocyte colony-stimulating factor. Proc Natl Acad Sci USA 93: 11871-11876.
    • (1996) Proc Natl Acad Sci USA , vol.93 , pp. 11871-11876
    • Dunbar, C.E.1
  • 62
    • 0028338452 scopus 로고
    • Effects of granulocyte colony-stimulating factor and stem cell factor, alone and in combination, on the mobilization of peripheral blood cells that engraft lethally irradiated dogs
    • de Revel, T et al. (1994). Effects of granulocyte colony-stimulating factor and stem cell factor, alone and in combination, on the mobilization of peripheral blood cells that engraft lethally irradiated dogs. Blood 83: 3795-3799.
    • (1994) Blood , vol.83 , pp. 3795-3799
    • de Revel, T.1
  • 63
    • 0028833966 scopus 로고
    • Rapid engraftment by peripheral blood progenitor cells mobilized by recombinant human stem cell factor and recombinant human granulocyte colony-stimulating factor in nonhuman primates
    • Andrews, RG, Briddell, RA, Knitter, GH, Rowley, SD, Appelbaum, FR and McNiece, IK (1995). Rapid engraftment by peripheral blood progenitor cells mobilized by recombinant human stem cell factor and recombinant human granulocyte colony-stimulating factor in nonhuman primates. Blood 85: 15-20.
    • (1995) Blood , vol.85 , pp. 15-20
    • Andrews, R.G.1    Briddell, R.A.2    Knitter, G.H.3    Rowley, S.D.4    Appelbaum, F.R.5    McNiece, I.K.6
  • 64
    • 0030761883 scopus 로고    scopus 로고
    • Peripheral blood progenitor cell mobilization using stem cell factor in combination with filgrastim in breast cancer patients
    • Glaspy JA et al. (1997). Peripheral blood progenitor cell mobilization using stem cell factor in combination with filgrastim in breast cancer patients. Blood 90: 2939-2951.
    • (1997) Blood , vol.90 , pp. 2939-2951
    • Glaspy, J.A.1
  • 66
    • 27944455711 scopus 로고    scopus 로고
    • Innovative strategies for PBPC mobilization
    • Fruehauf, S, Seeger, T and Topaly, J (2005). Innovative strategies for PBPC mobilization. Cytotherapy 7: 438-446.
    • (2005) Cytotherapy , vol.7 , pp. 438-446
    • Fruehauf, S.1    Seeger, T.2    Topaly, J.3
  • 67
    • 33746326517 scopus 로고    scopus 로고
    • Structure-function based design of small molecule inhibitors targeting Rho family GTPases
    • Nassar, N, Cancelas, J, Zheng, J, Williams, DA and Zheng, Y (2006). Structure-function based design of small molecule inhibitors targeting Rho family GTPases. Curr Top Med Chem 6: 1109-1116.
    • (2006) Curr Top Med Chem , vol.6 , pp. 1109-1116
    • Nassar, N.1    Cancelas, J.2    Zheng, J.3    Williams, D.A.4    Zheng, Y.5
  • 68
    • 0942266035 scopus 로고    scopus 로고
    • Lentivector-mediated clonal tracking reveals intrinsic heterogeneity in the human hematopoietic stem cell compartment and culture-induced stem cell impairment
    • Mazurier, F, Gan, OI, McKenzie, JL, Doedens, M and Dick JE (2004). Lentivector-mediated clonal tracking reveals intrinsic heterogeneity in the human hematopoietic stem cell compartment and culture-induced stem cell impairment Blood 103: 545-552.
    • (2004) Blood , vol.103 , pp. 545-552
    • Mazurier, F.1    Gan, O.I.2    McKenzie, J.L.3    Doedens, M.4    Dick, J.E.5
  • 69
    • 4344693571 scopus 로고    scopus 로고
    • Cytokines: Value-added products in hematopoietic stem cell gene therapy
    • Zielske, SP and Braun, SE (2004). Cytokines: Value-added products in hematopoietic stem cell gene therapy. Mol Ther 10: 211-219.
    • (2004) Mol Ther , vol.10 , pp. 211-219
    • Zielske, S.P.1    Braun, S.E.2
  • 70
    • 0036852308 scopus 로고    scopus 로고
    • Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells
    • Ailles, L et al. (2002). Molecular evidence of lentiviral vector-mediated gene transfer into human self-renewing, multi-potent, long-term NOD/SCID repopulating hematopoietic cells. Mol Ther 6: 615-626.
    • (2002) Mol Ther , vol.6 , pp. 615-626
    • Ailles, L.1
  • 71
    • 2342478423 scopus 로고    scopus 로고
    • Efficient lentiviral gene transfer to canine repopulating cells using an overnight transduction protocol
    • Hom, PA et al. (2004). Efficient lentiviral gene transfer to canine repopulating cells using an overnight transduction protocol. Blood 103: 3710-3716.
    • (2004) Blood , vol.103 , pp. 3710-3716
    • Hom, P.A.1
  • 72
    • 2542501560 scopus 로고    scopus 로고
    • Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector system
    • Hanawa, H et al. (2004). Efficient gene transfer into rhesus repopulating hematopoietic stem cells using a simian immunodeficiency virus-based lentiviral vector system. Blood 103: 4062-4069.
    • (2004) Blood , vol.103 , pp. 4062-4069
    • Hanawa, H.1
  • 73
    • 20044396722 scopus 로고    scopus 로고
    • Distinct genomic integration of MLV and SIV vectors in Primate hematopoietc stem and progenitor cells
    • Hematti, P et al. (2004). Distinct genomic integration of MLV and SIV vectors in Primate hematopoietc stem and progenitor cells. PLoS Biol 2: e423.
    • (2004) PLoS Biol , vol.2
    • Hematti, P.1
  • 74
    • 19444378586 scopus 로고    scopus 로고
    • Efficiency of transduction of highly purified murine hematopoietic stem cells by lentiviral and oncoretroviral vectors under conditions of minimal in vitro manipulation
    • Mostoslavsky, G, Kotton, DN, Fabian, AJ, Gray, JT, Lee, JS and Mulligan, RC (2005). Efficiency of transduction of highly purified murine hematopoietic stem cells by lentiviral and oncoretroviral vectors under conditions of minimal in vitro manipulation. Mol Ther 11: 932-940.
    • (2005) Mol Ther , vol.11 , pp. 932-940
    • Mostoslavsky, G.1    Kotton, D.N.2    Fabian, A.J.3    Gray, J.T.4    Lee, J.S.5    Mulligan, R.C.6
  • 76
    • 0038787487 scopus 로고    scopus 로고
    • - cells from mobilized peripheral blood after cytokine prestimulation
    • - cells from mobilized peripheral blood after cytokine prestimulation. Stem Cells 21: 472-480.
    • (2003) Stem Cells , vol.21 , pp. 472-480
    • Geronimi, F.1
  • 77
    • 0037366196 scopus 로고    scopus 로고
    • Cytokines, including stem cell factor alone, enhance lentiviral transduction in nondividing human LTCIC and NOD/SCID repopulating cells
    • Zielske, SP and Gerson, SL (2003). Cytokines, including stem cell factor alone, enhance lentiviral transduction in nondividing human LTCIC and NOD/SCID repopulating cells. Mol Ther 7: 325-333.
    • (2003) Mol Ther , vol.7 , pp. 325-333
    • Zielske, S.P.1    Gerson, S.L.2
  • 79
    • 0036811540 scopus 로고    scopus 로고
    • Advances in lentiviral vector design for gene-modification of hematopoietic stem cells
    • Logan, AC, Lutzko, C and Kohn, DB (2002). Advances in lentiviral vector design for gene-modification of hematopoietic stem cells. Curr Opin Biotechnol 13: 429-436.
    • (2002) Curr Opin Biotechnol , vol.13 , pp. 429-436
    • Logan, A.C.1    Lutzko, C.2    Kohn, D.B.3
  • 80
    • 33744496523 scopus 로고    scopus 로고
    • Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction
    • Santoni de Sio, FR, Cascio, P, Zingale, A, Gasparini, M and Naldini, L (2006). Proteasome activity restricts lentiviral gene transfer into hematopoietic stem cells and is down-regulated by cytokines that enhance transduction. Blood 107: 4257-4265.
    • (2006) Blood , vol.107 , pp. 4257-4265
    • Santoni de Sio, F.R.1    Cascio, P.2    Zingale, A.3    Gasparini, M.4    Naldini, L.5
  • 82
    • 0032787134 scopus 로고    scopus 로고
    • - bone marrow long-term culture-initiating cells (LTC-IC), extended LTC-IC, and murine in vivo long-term reconstituting stem cells
    • - bone marrow long-term culture-initiating cells (LTC-IC), extended LTC-IC, and murine in vivo long-term reconstituting stem cells. Blood 94: 4093-4102.
    • (1999) Blood , vol.94 , pp. 4093-4102
    • Ramsfjell, V.1
  • 83
    • 0034633678 scopus 로고    scopus 로고
    • Stimulation of mouse bone marrow cells with kit ligand, FLT3 ligand, and thrombopoietin leads to efficient retrovirus-mediated gene transfer to stem cells, whereas interleukin 3 and interleukin 11 reduce transduction of short- and long-term repopulating cells
    • Wognum, AW, Visser, TP, Peters, K, Bierhuizen, MF and Wagemaker, G (2000). Stimulation of mouse bone marrow cells with kit ligand, FLT3 ligand, and thrombopoietin leads to efficient retrovirus-mediated gene transfer to stem cells, whereas interleukin 3 and interleukin 11 reduce transduction of short- and long-term repopulating cells. Hum Gene Ther 11: 2129-2141.
    • (2000) Hum Gene Ther , vol.11 , pp. 2129-2141
    • Wognum, A.W.1    Visser, T.P.2    Peters, K.3    Bierhuizen, M.F.4    Wagemaker, G.5
  • 85
    • 0034749999 scopus 로고    scopus 로고
    • + stem cell compartment is accompanied by loss of self-renewal capacity
    • + stem cell compartment is accompanied by loss of self-renewal capacity. Immunity 15: 659-669.
    • (2001) Immunity , vol.15 , pp. 659-669
    • Adolfsson, J.1
  • 86
    • 20444503239 scopus 로고    scopus 로고
    • Converging pathways in leukemogenesis and stem cell self-renewal
    • Moore, MA (2005). Converging pathways in leukemogenesis and stem cell self-renewal. Exp Hematol 33: 719-737.
    • (2005) Exp Hematol , vol.33 , pp. 719-737
    • Moore, M.A.1
  • 87
    • 27744583582 scopus 로고    scopus 로고
    • Novel lentiviral vectors displaying "early-acting cytokines" selectively promote survival and transduction of NOD/SCID repopulating human hematopoietic stem cells
    • Verhoeyen, E et al. (2005). Novel lentiviral vectors displaying "early-acting cytokines" selectively promote survival and transduction of NOD/SCID repopulating human hematopoietic stem cells. Blood 106: 3386-3395.
    • (2005) Blood , vol.106 , pp. 3386-3395
    • Verhoeyen, E.1
  • 88
    • 2642670312 scopus 로고    scopus 로고
    • Antiviral activity of the proteasome on incoming human immunodeficiency virus type 1
    • Schwartz, O, Marechal, V, Friguet, B, Arenzana-Seisdedos, F and Heard, JM (1998). Antiviral activity of the proteasome on incoming human immunodeficiency virus type 1. J Virol 72: 3845-3850.
    • (1998) J Virol , vol.72 , pp. 3845-3850
    • Schwartz, O.1    Marechal, V.2    Friguet, B.3    Arenzana-Seisdedos, F.4    Heard, J.M.5
  • 89
    • 0036195928 scopus 로고    scopus 로고
    • Human immunodeficiency virus cDNA metabolism: Notable stability of two-long terminal repeat circles
    • Butler, SL, Johnson, EP and Bushman, FD (2002). Human immunodeficiency virus cDNA metabolism: Notable stability of two-long terminal repeat circles. J Virol 76: 3739-3747.
    • (2002) J Virol , vol.76 , pp. 3739-3747
    • Butler, S.L.1    Johnson, E.P.2    Bushman, F.D.3
  • 90
    • 10944260126 scopus 로고    scopus 로고
    • Retrovirus restriction factors
    • Goff, SP (2004). Retrovirus restriction factors. Mol Cell 16: 849-859.
    • (2004) Mol Cell , vol.16 , pp. 849-859
    • Goff, S.P.1
  • 91
    • 9244260598 scopus 로고    scopus 로고
    • Intrinsic immunity: A front-line defense against viral attack
    • Bieniasz, PD (2004). Intrinsic immunity: A front-line defense against viral attack. Nat Immunol 5: 1109A-1115.
    • (2004) Nat Immunol , vol.5
    • Bieniasz, P.D.1
  • 92
    • 0033524834 scopus 로고    scopus 로고
    • Dependence of human stem cell engraftment and repopulation of NOD/SCID mice on CXCR4
    • Peled, A et al. (1999). Dependence of human stem cell engraftment and repopulation of NOD/SCID mice on CXCR4. Science 283: 845-848.
    • (1999) Science , vol.283 , pp. 845-848
    • Peled, A.1
  • 93
    • 4043117769 scopus 로고    scopus 로고
    • Modulation of hematopoietic stem cell homing and engraftment by CD26
    • Christopherson II, KW, Hangoc, G, Mantel, CR and Broxmeyer, HE (2004). Modulation of hematopoietic stem cell homing and engraftment by CD26. Science 305: 1000-4003.
    • (2004) Science , vol.305 , pp. 1000-4003
    • Christopherson II, K.W.1    Hangoc, G.2    Mantel, C.R.3    Broxmeyer, H.E.4
  • 94
    • 11144356195 scopus 로고    scopus 로고
    • + progenitors increases their proliferation, migration, and NOD/SCID repopulation
    • + progenitors increases their proliferation, migration, and NOD/SCID repopulation. Blood 103: 2942-2949.
    • (2004) Blood , vol.103 , pp. 2942-2949
    • Kahn, J.1
  • 95
    • 33645108527 scopus 로고    scopus 로고
    • Inhibition of CD26 peptidase activity significantly improves engraftment of retrovirally transduced hematopoietic progenitors
    • Tian, C, Bagley, J, Forman, D and Iacomini, J (2006). Inhibition of CD26 peptidase activity significantly improves engraftment of retrovirally transduced hematopoietic progenitors. Gene Ther 13: 652-658.
    • (2006) Gene Ther , vol.13 , pp. 652-658
    • Tian, C.1    Bagley, J.2    Forman, D.3    Iacomini, J.4
  • 98
    • 0032079434 scopus 로고    scopus 로고
    • Growth-supporting activities of fibronectin on hematopoietic stem/progenitor cells in vitro and in vivo: Structural requirement for fibronectin activities of CS1 and cell-binding domains
    • Yokota, T et al. (1998). Growth-supporting activities of fibronectin on hematopoietic stem/progenitor cells in vitro and in vivo: Structural requirement for fibronectin activities of CS1 and cell-binding domains. Blood 91: 3263-3272.
    • (1998) Blood , vol.91 , pp. 3263-3272
    • Yokota, T.1
  • 99
    • 0842287499 scopus 로고    scopus 로고
    • The presence of the carboxy-terminal fragment of fibronectin allows maintenance of non-human primate long-term hematopoietic repopulating cells during extended ex vivo culture and transduction
    • Sellers, SE, Tisdale, JF, Agricola, BA, Donahue, RE and Dunbar, CE (2004). The presence of the carboxy-terminal fragment of fibronectin allows maintenance of non-human primate long-term hematopoietic repopulating cells during extended ex vivo culture and transduction. Exp Hematol 32: 163-170.
    • (2004) Exp Hematol , vol.32 , pp. 163-170
    • Sellers, S.E.1    Tisdale, J.F.2    Agricola, B.A.3    Donahue, R.E.4    Dunbar, C.E.5
  • 100
    • 31844442082 scopus 로고    scopus 로고
    • Busulfan pharmacokinetics, toxicity, and low-dose conditioning for autologous transplantation of genetically modified hematopoietic stem cells in the rhesus macaque model
    • Kang, EM et al. (2006). Busulfan pharmacokinetics, toxicity, and low-dose conditioning for autologous transplantation of genetically modified hematopoietic stem cells in the rhesus macaque model. Exp Hematol 34: 132-139.
    • (2006) Exp Hematol , vol.34 , pp. 132-139
    • Kang, E.M.1
  • 101
    • 33644923202 scopus 로고    scopus 로고
    • Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector
    • Kahl, CA et al. (2006). Effects of busulfan dose escalation on engraftment of infant rhesus monkey hematopoietic stem cells after gene marking by a lentiviral vector. Exp Hematol 34: 369-381.
    • (2006) Exp Hematol , vol.34 , pp. 369-381
    • Kahl, C.A.1
  • 102
    • 0023152463 scopus 로고
    • Retrovirus integration and chromatin structure: Mobney murine leukemia proviral integration sites map near DNAse I-hypersensitive sites
    • Rohdewohld H, Wriker, H, Reik, W, Jaenisch, R and Breindl, M (1987). Retrovirus integration and chromatin structure: Mobney murine leukemia proviral integration sites map near DNAse I-hypersensitive sites. J Virol 61: 336-343.
    • (1987) J Virol , vol.61 , pp. 336-343
    • Rohdewohld, H.1    Wriker, H.2    Reik, W.3    Jaenisch, R.4    Breindl, M.5
  • 103
    • 0037841763 scopus 로고    scopus 로고
    • Transcription start regions in the human genome are favored targets for MLV integration
    • Wu, X, Li, Y, Crise, B and Burgess, SM (2003). Transcription start regions in the human genome are favored targets for MLV integration. Science 300: 1749-1751.
    • (2003) Science , vol.300 , pp. 1749-1751
    • Wu, X.1    Li, Y.2    Crise, B.3    Burgess, S.M.4
  • 104
    • 19344375031 scopus 로고    scopus 로고
    • Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences
    • Mitchell, RS et al. (2004). Retroviral DNA integration: ASLV, HIV, and MLV show distinct target site preferences. PLoS Biol 2: E234.
    • (2004) PLoS Biol , vol.2
    • Mitchell, R.S.1
  • 105
    • 33646875083 scopus 로고    scopus 로고
    • Retrovirus vectors: Toward the plentivirus?
    • Baum, C, Schambach, A, Bohne, J and Galla, M (2006). Retrovirus vectors: toward the plentivirus? Mol Ther 13: 1050-1063.
    • (2006) Mol Ther , vol.13 , pp. 1050-1063
    • Baum, C.1    Schambach, A.2    Bohne, J.3    Galla, M.4
  • 106
    • 0036632178 scopus 로고    scopus 로고
    • RNA 3∓ readthrough of oncoretrovirus and lentivirus: Implications for vector safety and efficacy
    • Zaiss, AK, Son, S and Chang, LJ (2002). RNA 3∓ readthrough of oncoretrovirus and lentivirus: Implications for vector safety and efficacy. J Virol 76: 7209-7219.
    • (2002) J Virol , vol.76 , pp. 7209-7219
    • Zaiss, A.K.1    Son, S.2    Chang, L.J.3
  • 107
    • 0023678897 scopus 로고
    • Retroviral activation of a novel gene encoding a zinc finger protein in IL-3-dependent myeloid leukemia cell lines
    • Morishita, K, Parker, DS, Mucenski, ML, Jenkins, NA, Copeland, NG and Ihle, JN (1988). Retroviral activation of a novel gene encoding a zinc finger protein in IL-3-dependent myeloid leukemia cell lines. Cell 54: 831-840.
    • (1988) Cell , vol.54 , pp. 831-840
    • Morishita, K.1    Parker, D.S.2    Mucenski, M.L.3    Jenkins, N.A.4    Copeland, N.G.5    Ihle, J.N.6
  • 108
    • 33745108790 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration
    • Montini, E et al. (2006). Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol 24: 687-696.
    • (2006) Nat Biotechnol , vol.24 , pp. 687-696
    • Montini, E.1
  • 109
    • 7044228128 scopus 로고    scopus 로고
    • Inserdon of retroviral vectors in NOD/SCID repopulating human peripheral blood progenitor cells occurs preferentially in the vicinity of transcription start regions and in introns
    • Laufs, S, Nagy, KZ, Giordano, FA, Hotz-Wagenblatt, A, Zeller, WJ and Fruehauf, S (2004). Inserdon of retroviral vectors in NOD/SCID repopulating human peripheral blood progenitor cells occurs preferentially in the vicinity of transcription start regions and in introns. Mol Ther 10: 874-881.
    • (2004) Mol Ther , vol.10 , pp. 874-881
    • Laufs, S.1    Nagy, K.Z.2    Giordano, F.A.3    Hotz-Wagenblatt, A.4    Zeller, W.J.5    Fruehauf, S.6
  • 110
    • 0042738935 scopus 로고    scopus 로고
    • Occurrence of leukaemia following gene therapy of X-linked SCID
    • Kohn, DB, Sadelain, M and Glorioso, JC (2003). Occurrence of leukaemia following gene therapy of X-linked SCID. Nat Rev Cancer 3: 477-488.
    • (2003) Nat Rev Cancer , vol.3 , pp. 477-488
    • Kohn, D.B.1    Sadelain, M.2    Glorioso, J.C.3
  • 111
    • 15244346492 scopus 로고    scopus 로고
    • Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells
    • De Palma, M et al. (2005). Promoter trapping reveals significant differences in integration site selection between MLV and HIV vectors in primary hematopoietic cells. Blood 105: 2307-2315.
    • (2005) Blood , vol.105 , pp. 2307-2315
    • De Palma, M.1
  • 112
    • 33646887376 scopus 로고    scopus 로고
    • Genotoxicity of retroviral integration in hematopoietic cells
    • Nienhuis, AW, Dunbar, CE and Sorrentino, BP (2006). Genotoxicity of retroviral integration in hematopoietic cells. Mol Ther 13: 1031-1049.
    • (2006) Mol Ther , vol.13 , pp. 1031-1049
    • Nienhuis, A.W.1    Dunbar, C.E.2    Sorrentino, B.P.3
  • 113
    • 0037134031 scopus 로고    scopus 로고
    • Murine leukemia induced by retroviral gene marking
    • Li, Z et al. (2002). Murine leukemia induced by retroviral gene marking. Science 296: 497.
    • (2002) Science , vol.296 , pp. 497
    • Li, Z.1
  • 114
    • 25144490586 scopus 로고    scopus 로고
    • Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells
    • Calmels, B et al. (2005). Recurrent retroviral vector integration at the Mds1/Evi1 locus in nonhuman primate hematopoietic cells. Blood 106: 2530-2533.
    • (2005) Blood , vol.106 , pp. 2530-2533
    • Calmels, B.1
  • 115
    • 33646594123 scopus 로고    scopus 로고
    • Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque
    • Seggewiss, R et al. (2006). Acute myeloid leukemia is associated with retroviral gene transfer to hematopoietic progenitor cells in a rhesus macaque. Blood 107: 3865-3867.
    • (2006) Blood , vol.107 , pp. 3865-3867
    • Seggewiss, R.1
  • 116
    • 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 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 12: 401-409.
    • (2006) Nat Med , vol.12 , pp. 401-409
    • Ott, M.G.1
  • 117
    • 33344474674 scopus 로고    scopus 로고
    • EVI1 and hematopoietic disorders: History and perspectives
    • Nucifora, G, Laricchia-Robbio, L and Senyuk, V (2006). EVI1 and hematopoietic disorders: History and perspectives. Gene 368: 1-11.
    • (2006) Gene , vol.368 , pp. 1-11
    • Nucifora, G.1    Laricchia-Robbio, L.2    Senyuk, V.3
  • 118
    • 0037443457 scopus 로고    scopus 로고
    • Side effects of retroviral gene transfer into hematopoietic stem cells
    • Baum, C et al. (2003). Side effects of retroviral gene transfer into hematopoietic stem cells. Blood 101: 2099-2114.
    • (2003) Blood , vol.101 , pp. 2099-2114
    • Baum, C.1
  • 119
    • 0036725051 scopus 로고    scopus 로고
    • New genes involved in cancer identified by retroviral tagging
    • Suzuki, T et al. (2002). New genes involved in cancer identified by retroviral tagging. Nat Genet 32: 166-174.
    • (2002) Nat Genet , vol.32 , pp. 166-174
    • Suzuki, T.1
  • 120
    • 27944496521 scopus 로고    scopus 로고
    • Retroviral insertional mutagenesis: Past, present and future
    • Uren, AG, Kool, J, Berns, A and van Lohuizen, M (2005). Retroviral insertional mutagenesis: Past, present and future. Oncogene 24: 7656-7672.
    • (2005) Oncogene , vol.24 , pp. 7656-7672
    • Uren, A.G.1    Kool, J.2    Berns, A.3    van Lohuizen, M.4
  • 121
    • 0142084745 scopus 로고    scopus 로고
    • LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
    • Hacein-Bey-Abina, S et al. (2003). LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 302: 415-419.
    • (2003) Science , vol.302 , pp. 415-419
    • Hacein-Bey-Abina, S.1
  • 122
    • 33750624015 scopus 로고    scopus 로고
    • Cell culture assays reveal the importance of retroviral vector design for insertional genotoxicity
    • Modlich, U et al, (2006). Cell culture assays reveal the importance of retroviral vector design for insertional genotoxicity. Blood 108: 2545-2553.
    • (2006) Blood , vol.108 , pp. 2545-2553
    • Modlich, U.1
  • 123
    • 33847193756 scopus 로고    scopus 로고
    • The Influence of Vector Design on Integration Site Selection by gamma-Retroviral and Lentiviral Vectors
    • American Society of Gene Therapy: Baltimore
    • Recchia, A et al. (2006). The Influence of Vector Design on Integration Site Selection by gamma-Retroviral and Lentiviral Vectors. Mol. Ther. American Society of Gene Therapy: Baltimore, 13.
    • (2006) Mol. Ther. , vol.13
    • Recchia, A.1
  • 124
    • 0034548823 scopus 로고    scopus 로고
    • Lentiviral gene transfer into primary and secondary NOD/SCID repopulating cells
    • Woods, NB et al. (2000). Lentiviral gene transfer into primary and secondary NOD/SCID repopulating cells. Blood 96: 3725-3733.
    • (2000) Blood , vol.96 , pp. 3725-3733
    • Woods, N.B.1
  • 126
    • 0034254827 scopus 로고    scopus 로고
    • Inactivation of a GFP retrovirus occurs at multiple levels in long-term repopulating stem cells and their differentiated progeny
    • Klug, CA, Cheshier, S and Weissman, IL (2000). Inactivation of a GFP retrovirus occurs at multiple levels in long-term repopulating stem cells and their differentiated progeny. Blood 96: 894-901.
    • (2000) Blood , vol.96 , pp. 894-901
    • Klug, C.A.1    Cheshier, S.2    Weissman, I.L.3
  • 127
    • 4344651348 scopus 로고    scopus 로고
    • Self-inactivating lentiviral vectors resist proviral methylation but do not confer position-independent expression in hematopoietic stem cells
    • Mohamedali, A, Moreau-Gaudry, F, Richard, E, Xia, P, Nolta, J and Malik, P (2004). Self-inactivating lentiviral vectors resist proviral methylation but do not confer position-independent expression in hematopoietic stem cells. Mol Ther 10: 249-259.
    • (2004) Mol Ther , vol.10 , pp. 249-259
    • Mohamedali, A.1    Moreau-Gaudry, F.2    Richard, E.3    Xia, P.4    Nolta, J.5    Malik, P.6
  • 128
    • 14844358867 scopus 로고    scopus 로고
    • eGFP reporter genes silence LCRbeta-globin transgene expression via CpG dinucleotides
    • Dalle, B et al. (2005). eGFP reporter genes silence LCRbeta-globin transgene expression via CpG dinucleotides. Mol Ther 11: 591-599.
    • (2005) Mol Ther , vol.11 , pp. 591-599
    • Dalle, B.1
  • 129
    • 0034669932 scopus 로고    scopus 로고
    • High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors
    • Salmon, P, Kindler, V, Ducrey, O, Chapuis, B, Zubler, RH and Trono, D (2000). High-level transgene expression in human hematopoietic progenitors and differentiated blood lineages after transduction with improved lentiviral vectors. Blood 96: 3392-3398.
    • (2000) Blood , vol.96 , pp. 3392-3398
    • Salmon, P.1    Kindler, V.2    Ducrey, O.3    Chapuis, B.4    Zubler, R.H.5    Trono, D.6
  • 130
    • 0034322159 scopus 로고    scopus 로고
    • Lentiviral vectors for enhanced gene expression in human hematopoietic cells
    • Ramezani, A, Hawley, TS and Hawley, RG (2000). Lentiviral vectors for enhanced gene expression in human hematopoietic cells. Mol Ther 2: 458-469.
    • (2000) Mol Ther , vol.2 , pp. 458-469
    • Ramezani, A.1    Hawley, T.S.2    Hawley, R.G.3
  • 131
    • 0036252492 scopus 로고    scopus 로고
    • High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of immunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter
    • Demaison, C et al. (2002). High-level transduction and gene expression in hematopoietic repopulating cells using a human immunodeficiency [correction of immunodeficiency] virus type 1-based lentiviral vector containing an internal spleen focus forming virus promoter. Hum Gene Ther 13: 803-813.
    • (2002) Hum Gene Ther , vol.13 , pp. 803-813
    • Demaison, C.1
  • 132
    • 0034469216 scopus 로고    scopus 로고
    • Lentivirus gene transfer in murine hematopoietic progenitor cells is compromised by a delay in proviral integration and results in transduction mosaicism and heterogeneous gene expression in progeny cells
    • Mikkola, H et al. (2000). Lentivirus gene transfer in murine hematopoietic progenitor cells is compromised by a delay in proviral integration and results in transduction mosaicism and heterogeneous gene expression in progeny cells. J Virol 74: 11911-111918.
    • (2000) J Virol , vol.74 , pp. 11911-111918
    • Mikkola, H.1
  • 133
    • 0034985720 scopus 로고    scopus 로고
    • +-derived hematopoietic cells, including developing T cells and NOD/SCID mouse repopulating cells, following transduction with modified trip lentiviral vectors
    • +-derived hematopoietic cells, including developing T cells and NOD/SCID mouse repopulating cells, following transduction with modified trip lentiviral vectors. Mol Ther 3: 438-448.
    • (2001) Mol Ther , vol.3 , pp. 438-448
    • Sirven, A.1
  • 134
    • 0026534020 scopus 로고
    • Toward gene therapy in haemophilia A: Retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells
    • Hoeben, RC, Einerhand, MP, Briet, E, van Ormondt, H, Valerio, D and van der Eb, AJ (1992). Toward gene therapy in haemophilia A: retrovirus-mediated transfer of a factor VIII gene into murine haematopoietic progenitor cells. Thromb Haemost 67: 341-345.
    • (1992) Thromb Haemost , vol.67 , pp. 341-345
    • Hoeben, R.C.1    Einerhand, M.P.2    Briet, E.3    van Ormondt, H.4    Valerio, D.5    van der Eb, A.J.6
  • 135
    • 0032510756 scopus 로고    scopus 로고
    • Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A
    • Evans, GL and Morgan, RA (1998). Genetic induction of immune tolerance to human clotting factor VIII in a mouse model for hemophilia A. Proc Natl Acad Sci USA 95: 5734-5739.
    • (1998) Proc Natl Acad Sci USA , vol.95 , pp. 5734-5739
    • Evans, G.L.1    Morgan, R.A.2
  • 136
    • 7044284791 scopus 로고    scopus 로고
    • Sustained phenotypic correction of hemophilia a mice following oncoretroviral-mediated expression of a bioengineered human factor VIII gene in long-term hematopoietic repopulating cells
    • Moayeri, M, Ramezani, A, Morgan, RA, Hawley, TS and Hawley, RG (2004). Sustained phenotypic correction of hemophilia a mice following oncoretroviral-mediated expression of a bioengineered human factor VIII gene in long-term hematopoietic repopulating cells. Mol Ther 10: 892-902.
    • (2004) Mol Ther , vol.10 , pp. 892-902
    • Moayeri, M.1    Ramezani, A.2    Morgan, R.A.3    Hawley, T.S.4    Hawley, R.G.5
  • 137
    • 0037853196 scopus 로고    scopus 로고
    • Efficient production of human FVIII in hemophilic mice using lentiviral vectors
    • Kootstra, NA, Matsumura, R and Verma, IM (2003). Efficient production of human FVIII in hemophilic mice using lentiviral vectors. Mol Ther 7: 623-631.
    • (2003) Mol Ther , vol.7 , pp. 623-631
    • Kootstra, N.A.1    Matsumura, R.2    Verma, I.M.3
  • 138
    • 0141962306 scopus 로고    scopus 로고
    • Recombinant factor VIII expression in hematopoietic cells following lentiviral transduction
    • Tiede, A et al. (2003). Recombinant factor VIII expression in hematopoietic cells following lentiviral transduction. Gene Ther 10: 1917-1925.
    • (2003) Gene Ther , vol.10 , pp. 1917-1925
    • Tiede, A.1
  • 139
    • 13644262651 scopus 로고    scopus 로고
    • + cells transduced with simian immunodeficiency virus agmTYO1-based vectors carrying the human coagulation factor VIII gene in NOD/SCID mice
    • + cells transduced with simian immunodeficiency virus agmTYO1-based vectors carrying the human coagulation factor VIII gene in NOD/SCID mice. J Gene Med 6:1049-1060.
    • (2004) J Gene Med , vol.6 , pp. 1049-1060
    • Kikuchi, J.1
  • 140
    • 29944432812 scopus 로고    scopus 로고
    • Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX
    • Bigger, BW et al. (2006). Permanent partial phenotypic correction and tolerance in a mouse model of hemophilia B by stem cell gene delivery of human factor IX. Gene Ther 13: 117-126.
    • (2006) Gene Ther , vol.13 , pp. 117-126
    • Bigger, B.W.1
  • 141
    • 33747076039 scopus 로고    scopus 로고
    • Stem cell-derived erythroid cells mediate long-term systemic protein delivery
    • Chang, AH, Stephen, MT and Sadelain, M (2006). Stem cell-derived erythroid cells mediate long-term systemic protein delivery. Nat Biotechnol 24: 1017-1021.
    • (2006) Nat Biotechnol , vol.24 , pp. 1017-1021
    • Chang, A.H.1    Stephen, M.T.2    Sadelain, M.3
  • 142
    • 27744489220 scopus 로고    scopus 로고
    • Silencing and variegation of gammaretrovirus and lentivirus vectors
    • Ellis, J (2005). Silencing and variegation of gammaretrovirus and lentivirus vectors. Hum Gene Ther 16: 1241-1246.
    • (2005) Hum Gene Ther , vol.16 , pp. 1241-1246
    • Ellis, J.1
  • 143
    • 0017739615 scopus 로고
    • Virus infection of murine teratocarcinoma stem cell lines
    • Teich, NM, Weiss, RA, Martin, GR and Lowy, DR (1977). Virus infection of murine teratocarcinoma stem cell lines. Cell 12: 973-982.
    • (1977) Cell , vol.12 , pp. 973-982
    • Teich, N.M.1    Weiss, R.A.2    Martin, G.R.3    Lowy, D.R.4
  • 144
    • 0018896982 scopus 로고
    • Silent infection of murine embryonal carcinoma cells by Moloney murine leukemia virus
    • Speers, WC, Gautsch, JW and Dixon, FJ (1980). Silent infection of murine embryonal carcinoma cells by Moloney murine leukemia virus. Virology 105: 241-244.
    • (1980) Virology , vol.105 , pp. 241-244
    • Speers, W.C.1    Gautsch, J.W.2    Dixon, F.J.3
  • 145
    • 0033064966 scopus 로고    scopus 로고
    • Amelioration of retroviral vector silencing in locus control region β-globin-transgenic mice and transduced F9 embryonic cells
    • Osborne, CS, Pasceri, P, Singal, R, Sukonnik, T, Ginder, GO and Ellis, J (1999). Amelioration of retroviral vector silencing in locus control region β-globin-transgenic mice and transduced F9 embryonic cells. J Virol 73: 5490-5496.
    • (1999) J Virol , vol.73 , pp. 5490-5496
    • Osborne, C.S.1    Pasceri, P.2    Singal, R.3    Sukonnik, T.4    Ginder, G.O.5    Ellis, J.6
  • 146
    • 0034000919 scopus 로고    scopus 로고
    • The cHS4 insulator increases the probability of retroviral expression at random chromosomal integration sites
    • Rivella, S, Callegari, JA, May, C, Tan, CW and Sadelain, M (2000). The cHS4 insulator increases the probability of retroviral expression at random chromosomal íntegration sites. J Virol 74: 4679-4687.
    • (2000) J Virol , vol.74 , pp. 4679-4687
    • Rivella, S.1    Callegari, J.A.2    May, C.3    Tan, C.W.4    Sadelain, M.5
  • 147
    • 0037383575 scopus 로고    scopus 로고
    • Genetic manipulation of primate embryonic and hematopoietic stem cells with simian lentivirus vectors
    • Hanazono, Y, Asano, T, Ueda, Y and Ozawa, K (2003). Genetic manipulation of primate embryonic and hematopoietic stem cells with simian lentivirus vectors. Trends Cardiovasc Med 13: 106-110.
    • (2003) Trends Cardiovasc Med , vol.13 , pp. 106-110
    • Hanazono, Y.1    Asano, T.2    Ueda, Y.3    Ozawa, K.4
  • 148
    • 0346422481 scopus 로고    scopus 로고
    • Mutation of CpGs in the murine stem cell virus retroviral vector long terminal repeat represses silencing in embryonic stem cells
    • Swindle, CS, Kim, HG and Klug, CA (2004). Mutation of CpGs in the murine stem cell virus retroviral vector long terminal repeat represses silencing in embryonic stem cells. J Biol Chem 279: 34-41.
    • (2004) J Biol Chem , vol.279 , pp. 34-41
    • Swindle, C.S.1    Kim, H.G.2    Klug, C.A.3
  • 149
    • 3042732990 scopus 로고    scopus 로고
    • Retrovirus silencing, variegation, extinction, and memory are controlled by a dynamic interplay of multiple epigenetic modifications
    • Yao, S, Sukonnik, T, Kean, T, Bharadwaj, RR, Pasceri, P and Ellis, J (2004). Retrovirus silencing, variegation, extinction, and memory are controlled by a dynamic interplay of multiple epigenetic modifications. Mol Ther 10: 27-36.
    • (2004) Mol Ther , vol.10 , pp. 27-36
    • Yao, S.1    Sukonnik, T.2    Kean, T.3    Bharadwaj, R.R.4    Pasceri, P.5    Ellis, J.6
  • 150
    • 0020503308 scopus 로고
    • Independent mechanisms involved in suppression of the Moloney leukemia virus genome during differentiation of murine teratocarcinoma cells
    • Niwa, O, Yokota, Y, Ishida, H and Sugahara, T (1983). Independent mechanisms involved in suppression of the Moloney leukemia virus genome during differentiation of murine teratocarcinoma cells. Cell 32: 1105-1113.
    • (1983) Cell , vol.32 , pp. 1105-1113
    • Niwa, O.1    Yokota, Y.2    Ishida, H.3    Sugahara, T.4
  • 151
    • 0031975190 scopus 로고    scopus 로고
    • Host cis-mediated extinction of a retrovirus permissive for expression in embryonal stem cells during differentiation
    • Laker, C et al. (1998). Host cis-mediated extinction of a retrovirus permissive for expression in embryonal stem cells during differentiation. J Virol 72: 339-348.
    • (1998) J Virol , vol.72 , pp. 339-348
    • Laker, C.1
  • 152
    • 0037133212 scopus 로고    scopus 로고
    • Transgenesis by lentiviral vectors: Lack of gene silencing in mammalian embryonic stem cells and preimplantation embryos
    • Pfeifer, A, Ikawa, M, Dayn, Y and Verma, IM (2002). Transgenesis by lentiviral vectors: Lack of gene silencing in mammalian embryonic stem cells and preimplantation embryos. Proc Natl Acad Sci USA 99: 2140-2145.
    • (2002) Proc Natl Acad Sci USA , vol.99 , pp. 2140-2145
    • Pfeifer, A.1    Ikawa, M.2    Dayn, Y.3    Verma, I.M.4
  • 153
    • 0036468677 scopus 로고    scopus 로고
    • Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectors
    • Lois, C, Hong, EJ, Pease, S, Brown, EJ and Baltimore, D (2002). Germline transmission and tissue-specific expression of transgenes delivered by lentiviral vectors. Science 295: 868-872.
    • (2002) Science , vol.295 , pp. 868-872
    • Lois, C.1    Hong, E.J.2    Pease, S.3    Brown, E.J.4    Baltimore, D.5
  • 154
    • 0142150950 scopus 로고    scopus 로고
    • Generation of transgenic mice using lentiviral vectors: A novel preclinical assessment of lentiviral vectors for gene therapy
    • Ikawa, M, Tanaka, N, Kao, WW and Verma, IM (2003). Generation of transgenic mice using lentiviral vectors: A novel preclinical assessment of lentiviral vectors for gene therapy. Mol Ther 8: 666-673.
    • (2003) Mol Ther , vol.8 , pp. 666-673
    • Ikawa, M.1    Tanaka, N.2    Kao, W.W.3    Verma, I.M.4
  • 155
    • 0034670033 scopus 로고    scopus 로고
    • Lentivirus-based vectors transduce mouse hematopoietic stem cells with similar efficiency to moloney murine leukemia virus-based vectors
    • Barrette, S, Douglas, JL, Seidel, NE and Bodine, DM (2000). Lentivirus-based vectors transduce mouse hematopoietic stem cells with similar efficiency to moloney murine leukemia virus-based vectors. Blood 96: 3385-3391.
    • (2000) Blood , vol.96 , pp. 3385-3391
    • Barrette, S.1    Douglas, J.L.2    Seidel, N.E.3    Bodine, D.M.4
  • 156
    • 0034329445 scopus 로고    scopus 로고
    • Retrovirus vector silencing is de novo methylase independent and marked by a repressive histone code
    • Pannell, D et al. (2000). Retrovirus vector silencing is de novo methylase independent and marked by a repressive histone code. EMBO J 19: 5884-5894.
    • (2000) EMBO J , vol.19 , pp. 5884-5894
    • Pannell, D.1
  • 157
    • 0036242306 scopus 로고    scopus 로고
    • Development of gene therapy for hematopoietic stem cells using lentiviral vectors
    • Woods, NB, Ooka, A and Karlsson, S (2002). Development of gene therapy for hematopoietic stem cells using lentiviral vectors. Leukemia 16: 563-569.
    • (2002) Leukemia , vol.16 , pp. 563-569
    • Woods, N.B.1    Ooka, A.2    Karlsson, S.3
  • 158
    • 0033755914 scopus 로고    scopus 로고
    • Lentivirus vector gene expression during ES cell-derived hematopoietic development in vitro
    • Hamaguchi, I et al. (2000). Lentivirus vector gene expression during ES cell-derived hematopoietic development in vitro. J Virol 74: 10778-10784.
    • (2000) J Virol , vol.74 , pp. 10778-10784
    • Hamaguchi, I.1
  • 159
    • 30344464717 scopus 로고    scopus 로고
    • Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O(6)-methylguanine-DNA methyltransferase in hematopoietic cells
    • Schambach, A et al. (2006). Equal potency of gammaretroviral and lentiviral SIN vectors for expression of O(6)-methylguanine-DNA methyltransferase in hematopoietic cells. Mol Ther 13: 391-400.
    • (2006) Mol Ther , vol.13 , pp. 391-400
    • Schambach, A.1
  • 160
    • 0031743608 scopus 로고    scopus 로고
    • Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery
    • Zufferey, R et al. (1998). Self-inactivating lentivirus vector for safe and efficient in vivo gene delivery. J Virol 72: 9873-9880.
    • (1998) J Virol , vol.72 , pp. 9873-9880
    • Zufferey, R.1
  • 161
    • 0031710033 scopus 로고    scopus 로고
    • A third-generation lentivirus vector with a conditional packaging system
    • Dull, T et al. (1998). A third-generation lentivirus vector with a conditional packaging system. J Virol 72: 8463-8471.
    • (1998) J Virol , vol.72 , pp. 8463-8471
    • Dull, T.1
  • 162
    • 0029164582 scopus 로고
    • Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells
    • Riviere, I, Brose, K and Mulligan, RC (1995). Effects of retroviral vector design on expression of human adenosine deaminase in murine bone marrow transplant recipients engrafted with genetically modified cells. Proc Natl Acad Sci USA 92: 6733-6737.
    • (1995) Proc Natl Acad Sci USA , vol.92 , pp. 6733-6737
    • Riviere, I.1    Brose, K.2    Mulligan, R.C.3
  • 164
    • 0028216348 scopus 로고
    • Hepatitis B virus RNA element that facilitates accumulation of surface gene transcripts in the cytoplasm
    • Huang, ZM and Yen, TS (1994). Hepatitis B virus RNA element that facilitates accumulation of surface gene transcripts in the cytoplasm. J Virol 68: 3193-3199.
    • (1994) J Virol , vol.68 , pp. 3193-3199
    • Huang, Z.M.1    Yen, T.S.2
  • 165
    • 0031950572 scopus 로고    scopus 로고
    • Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element
    • Donello, JE, Loeb, JE and Hope, TJ (1998). Woodchuck hepatitis virus contains a tripartite posttranscriptional regulatory element. J Virol 72: 5085-5092.
    • (1998) J Virol , vol.72 , pp. 5085-5092
    • Donello, J.E.1    Loeb, J.E.2    Hope, T.J.3
  • 166
    • 0036044209 scopus 로고    scopus 로고
    • Enhancement of green fluorescent protein expression in adeno-associated virus with the Woodchuck hepatitis virus post-transcriptional regulatory element
    • Loeb, JE, Weitzman, MD and Hope, TJ (2002). Enhancement of green fluorescent protein expression in adeno-associated virus with the Woodchuck hepatitis virus post-transcriptional regulatory element Methods Mol Biol 183: 331-340.
    • (2002) Methods Mol Biol , vol.183 , pp. 331-340
    • Loeb, J.E.1    Weitzman, M.D.2    Hope, T.J.3
  • 167
    • 0032978712 scopus 로고    scopus 로고
    • Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors
    • Zufferey, R, Donello, JE, Trono, D and Hope, TJ (1999). Woodchuck hepatitis virus posttranscriptional regulatory element enhances expression of transgenes delivered by retroviral vectors. J Virol 73: 2886-2892.
    • (1999) J Virol , vol.73 , pp. 2886-2892
    • Zufferey, R.1    Donello, J.E.2    Trono, D.3    Hope, T.J.4
  • 168
    • 0034322479 scopus 로고    scopus 로고
    • Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors
    • Schambach, A, Wodrich, H, Hildinger, M, Bohne, J, Krausslich, HG and Baum, C (2000). Context dependence of different modules for posttranscriptional enhancement of gene expression from retroviral vectors. Mol Ther 2: 435-445.
    • (2000) Mol Ther , vol.2 , pp. 435-445
    • Schambach, A.1    Wodrich, H.2    Hildinger, M.3    Bohne, J.4    Krausslich, H.G.5    Baum, C.6
  • 169
    • 11844288284 scopus 로고    scopus 로고
    • Potential oncogene activity of the Woodchuck hepatitis post-transcriptional regulatory element (WPRE)
    • Kingsman, SM, Mitrophanous, K and Olsen, JC (2005). Potential oncogene activity of the Woodchuck hepatitis post-transcriptional regulatory element (WPRE). Gene Ther 12: 3-4.
    • (2005) Gene Ther , vol.12 , pp. 3-4
    • Kingsman, S.M.1    Mitrophanous, K.2    Olsen, J.C.3
  • 170
    • 33645103149 scopus 로고    scopus 로고
    • Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression
    • Schambach, A et al. (2006). Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression. Gene Ther 13: 641-645.
    • (2006) Gene Ther , vol.13 , pp. 641-645
    • Schambach, A.1
  • 171
    • 0031949085 scopus 로고    scopus 로고
    • Genetic treatment of severe hemoglobinopathies: The combat against transgene variegation and transgene silencing
    • Rivella, S and Sadelain, M (1998). Genetic treatment of severe hemoglobinopathies: The combat against transgene variegation and transgene silencing. Semin Hematol 35: 112-125.
    • (1998) Semin Hematol , vol.35 , pp. 112-125
    • Rivella, S.1    Sadelain, M.2
  • 172
    • 0032999477 scopus 로고    scopus 로고
    • Stem cell gene therapy for the beta-chain hemoglobinopathies. Problems and progress
    • Discussion 107-108
    • Emery, DW and Stamatoyannopoulos, G (1999). Stem cell gene therapy for the beta-chain hemoglobinopathies. Problems and progress. Ann N Y Acad Sci 872: 94-107; Discussion 107-108.
    • (1999) Ann N Y Acad Sci , vol.872 , pp. 94-107
    • Emery, D.W.1    Stamatoyannopoulos, G.2
  • 173
    • 22744455256 scopus 로고    scopus 로고
    • Gene therapy progress and prospects: Development of improved lentiviral and retroviral vectors - Design, biosafety, and production
    • Sinn, PL, Sauter, SL and McCray, PB Jr (2005). Gene therapy progress and prospects: Development of improved lentiviral and retroviral vectors - design, biosafety, and production. Gene Ther 12: 1089-1098.
    • (2005) Gene Ther , vol.12 , pp. 1089-1098
    • Sinn, P.L.1    Sauter, S.L.2    McCray Jr., P.B.3
  • 174
    • 0036467996 scopus 로고    scopus 로고
    • Insulators: Many functions, many mechanisms
    • West, AG, Gaszner, M and Felsenfeld, G (2002). Insulators: Many functions, many mechanisms. Genes Dev 16: 271-288.
    • (2002) Genes Dev , vol.16 , pp. 271-288
    • West, A.G.1    Gaszner, M.2    Felsenfeld, G.3
  • 175
    • 0037076316 scopus 로고    scopus 로고
    • Position-effect protection and enhancer blocking by the chicken beta-globin insulator are separable activities
    • Recillas-Targa, F et al. (2002). Position-effect protection and enhancer blocking by the chicken beta-globin insulator are separable activities. Proc Natl Acad Sci USA 99: 6883-6888.
    • (2002) Proc Natl Acad Sci USA , vol.99 , pp. 6883-6888
    • Recillas-Targa, F.1
  • 176
    • 8644240108 scopus 로고    scopus 로고
    • Recruitment of histone modifications by USF proteins at a vertebrate barrier element
    • West, AG, Huang, S, Gaszner, M, Litt, MD and Wsenfeld, G (2004). Recruitment of histone modifications by USF proteins at a vertebrate barrier element. Mol Cell 16: 453-463.
    • (2004) Mol Cell , vol.16 , pp. 453-463
    • West, A.G.1    Huang, S.2    Gaszner, M.3    Litt, M.D.4    Wsenfeld, G.5
  • 177
    • 0034255145 scopus 로고    scopus 로고
    • A chromatin insulator protects retrovirus vectors from chromosomal position effects
    • Emery, DW, Yannaki, E, Tubb, J and Stamatoyannopoulos, G (2000). A chromatin insulator protects retrovirus vectors from chromosomal position effects. Proc Natl Acad Sci USA 97: 9150-9155.
    • (2000) Proc Natl Acad Sci USA , vol.97 , pp. 9150-9155
    • Emery, D.W.1    Yannaki, E.2    Tubb, J.3    Stamatoyannopoulos, G.4
  • 178
    • 0036099418 scopus 로고    scopus 로고
    • Topological constraints governing the use of the chicken HS4 chromatin insulator in oncoretrovirus vectors
    • Yannaki, E, Tubb, J, Aker, M, Stamatoyannopoulos, G and Emery, DW (2002). Topological constraints governing the use of the chicken HS4 chromatin insulator in oncoretrovirus vectors. Mol Ther 5: 589-598.
    • (2002) Mol Ther , vol.5 , pp. 589-598
    • Yannaki, E.1    Tubb, J.2    Aker, M.3    Stamatoyannopoulos, G.4    Emery, D.W.5
  • 179
    • 0038792207 scopus 로고    scopus 로고
    • Performance- and safety-enhanced lentiviral vectors containing the human interferon-β scaffold attachment region and the chicken β-globin insulator
    • Ramezani, A, Hawley, TS and Hawley, RG (2003). Performance- and safety-enhanced lentiviral vectors containing the human interferon-β scaffold attachment region and the chicken β-globin insulator. Blood 101: 4717-4724.
    • (2003) Blood , vol.101 , pp. 4717-4724
    • Ramezani, A.1    Hawley, T.S.2    Hawley, R.G.3
  • 180
    • 0026597313 scopus 로고
    • Biological significance of unwinding capability of nuclear matrix-associating DNAs
    • Bode, J et al. (1992). Biological significance of unwinding capability of nuclear matrix-associating DNAs. Science 255: 195-197.
    • (1992) Science , vol.255 , pp. 195-197
    • Bode, J.1
  • 181
    • 0031899366 scopus 로고    scopus 로고
    • Human matrix attachment regions insulate transgene expression from chromosomal position effects in Drosophila melanogaster
    • Namciu, SJ, Blochlinger, KB and Fournier, RE (1998). Human matrix attachment regions insulate transgene expression from chromosomal position effects in Drosophila melanogaster. Mol Cell Biol 18: 2382-2391.
    • (1998) Mol Cell Biol , vol.18 , pp. 2382-2391
    • Namciu, S.J.1    Blochlinger, K.B.2    Fournier, R.E.3
  • 182
    • 0034103927 scopus 로고    scopus 로고
    • Transcriptional augmentation: Modulation of gene expression by scaffold/matrix-attached regions (S/MAR elements)
    • Bode, J, Benham, C, Knapp, A and Mielke, C (2000). Transcriptional augmentation: Modulation of gene expression by scaffold/matrix-attached regions (S/MAR elements). Crit Rev Eukaryot Gene Exp 10: 73-90.
    • (2000) Crit Rev Eukaryot Gene Exp , vol.10 , pp. 73-90
    • Bode, J.1    Benham, C.2    Knapp, A.3    Mielke, C.4
  • 183
    • 0031958593 scopus 로고    scopus 로고
    • Scaffold attachment region-mediated enhancement of retroviral vector expression in primary T cells
    • Agarwal, M, Austin, TW, Morel, F, Chen, I, Bohnlein, E and Plavec, I (1998). Scaffold attachment region-mediated enhancement of retroviral vector expression in primary T cells. J Virol 72: 3720-3728.
    • (1998) J Virol , vol.72 , pp. 3720-3728
    • Agarwal, M.1    Austin, T.W.2    Morel, F.3    Chen, I.4    Bohnlein, E.5    Plavec, I.6
  • 185
    • 27144474499 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene therapy with drug resistance genes: An update
    • Budak-Alpdogan, T, Banerjee, D and Bertino, JR (2005). Hematopoietic stem cell gene therapy with drug resistance genes: An update. Cancer Gene Ther 12: 849-863.
    • (2005) Cancer Gene Ther , vol.12 , pp. 849-863
    • Budak-Alpdogan, T.1    Banerjee, D.2    Bertino, J.R.3
  • 186
    • 33344476395 scopus 로고    scopus 로고
    • Survival of the fittest: In vivo selection and stem cell gene therapy
    • Neff, T, Beard, BC and Kiem, HP (2006). Survival of the fittest: In vivo selection and stem cell gene therapy. Blood 107: 1751-1760.
    • (2006) Blood , vol.107 , pp. 1751-1760
    • Neff, T.1    Beard, B.C.2    Kiem, H.P.3
  • 188
    • 0034990304 scopus 로고    scopus 로고
    • Protection and in vivo selection of hematopoietic stem cells using temozolomide, O6-benzylguanine, and an alkyltransferase-expressing retroviral vector
    • Sawai, N, Zhou, S, Vanin, EF, Houghton, P, Brent TP and Sorrentino, BP (2001). Protection and in vivo selection of hematopoietic stem cells using temozolomide, O6-benzylguanine, and an alkyltransferase-expressing retroviral vector. Mol Ther 3: 78-87.
    • (2001) Mol Ther , vol.3 , pp. 78-87
    • Sawai, N.1    Zhou, S.2    Vanin, E.F.3    Houghton, P.4    Brent, T.P.5    Sorrentino, B.P.6
  • 189
    • 0036708229 scopus 로고    scopus 로고
    • Hematoprotection and enrichment of transduced cells in vivo after gene transfer of MGMT(P140K) into hematopoietic stem cells
    • Jansen, M et al. (2002). Hematoprotection and enrichment of transduced cells in vivo after gene transfer of MGMT(P140K) into hematopoietic stem cells. Cancer Gene Ther 9: 737-746.
    • (2002) Cancer Gene Ther , vol.9 , pp. 737-746
    • Jansen, M.1
  • 190
    • 0037772187 scopus 로고    scopus 로고
    • Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells
    • Persons, DA et al. (2003). Successful treatment of murine beta-thalassemia using in vivo selection of genetically modified, drug-resistant hematopoietic stem cells. Blood 102: 506-513.
    • (2003) Blood , vol.102 , pp. 506-513
    • Persons, D.A.1
  • 191
    • 0347086142 scopus 로고    scopus 로고
    • Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model
    • Neff, T et al. (2003). Methylguanine methyltransferase-mediated in vivo selection and chemoprotection of allogeneic stem cells in a large-animal model. J Clin Invest 112: 1581-1588.
    • (2003) J Clin Invest , vol.112 , pp. 1581-1588
    • Neff, T.1
  • 192
    • 0347086143 scopus 로고    scopus 로고
    • In vivo selection of MGMT(P140K) lentivirus-transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning
    • Zielske, SP, Reese, JS, Lingas, KT, Donze, JR and Gerson, SL (2003). In vivo selection of MGMT(P140K) lentivirus-transduced human NOD/SCID repopulating cells without pretransplant irradiation conditioning. J Clin Invest 112: 1561-1570.
    • (2003) J Clin Invest , vol.112 , pp. 1561-1570
    • Zielske, S.P.1    Reese, J.S.2    Lingas, K.T.3    Donze, J.R.4    Gerson, S.L.5
  • 193
    • 0041440074 scopus 로고    scopus 로고
    • Repopulating defect of mismatch repair-deficient hematopoietic stem cells
    • Reese, JS, Liu, L and Gerson, SL (2003). Repopulating defect of mismatch repair-deficient hematopoietic stem cells. Blood 102: 1626-1633.
    • (2003) Blood , vol.102 , pp. 1626-1633
    • Reese, J.S.1    Liu, L.2    Gerson, S.L.3
  • 194
    • 0033215140 scopus 로고    scopus 로고
    • Locus control regions: Coming of age at a decade plus
    • Li, Q, Harju, S and Peterson, KR (1999). Locus control regions: Coming of age at a decade plus. Trends Genet 15: 403-408.
    • (1999) Trends Genet , vol.15 , pp. 403-408
    • Li, Q.1    Harju, S.2    Peterson, K.R.3
  • 196
    • 0036532118 scopus 로고    scopus 로고
    • Chlps of the β-globin locus: Unraveling gene regulation within an active domain
    • Bulger, M, Sawado, T, Schubeler, D and Groudine, M (2002). Chlps of the β-globin locus: Unraveling gene regulation within an active domain. Curr Opin Genet Dev 12: 170-177.
    • (2002) Curr Opin Genet Dev , vol.12 , pp. 170-177
    • Bulger, M.1    Sawado, T.2    Schubeler, D.3    Groudine, M.4
  • 197
    • 0031593199 scopus 로고    scopus 로고
    • Locus control regions of mammalian β-globin gene clusters: Combining phylogenetic analyses and experimental results to gain functional insights
    • Hardison, R, Slightom, JL, Gumucio, DL, Goodman, M, Stojanovic, N and Miller, W (1997). Locus control regions of mammalian β-globin gene clusters: Combining phylogenetic analyses and experimental results to gain functional insights. Gene 205: 73-94.
    • (1997) Gene , vol.205 , pp. 73-94
    • Hardison, R.1    Slightom, J.L.2    Gumucio, D.L.3    Goodman, M.4    Stojanovic, N.5    Miller, W.6
  • 198
    • 0023663887 scopus 로고
    • Position-independent, high-level expression of the human β-globin gene in transgenic mice
    • Grosveld, F, van Assendelft, GB, Greaves, DR and Kollias, G (1987). Position-independent, high-level expression of the human β-globin gene in transgenic mice. Cell 51: 975-985.
    • (1987) Cell , vol.51 , pp. 975-985
    • Grosveld, F.1    van Assendelft, G.B.2    Greaves, D.R.3    Kollias, G.4
  • 199
    • 0024536789 scopus 로고
    • The β-globin dominant control region activates homologous and heterologous promoters in a tissue-specific manner
    • Blom van Assendelft, G, Hanscombe, O, Grosveld, F and Greaves, DR (1989). The β-globin dominant control region activates homologous and heterologous promoters in a tissue-specific manner. Cell 56: 969-977.
    • (1989) Cell , vol.56 , pp. 969-977
    • Blom van Assendelft, G.1    Hanscombe, O.2    Grosveld, F.3    Greaves, D.R.4
  • 200
    • 0037085774 scopus 로고    scopus 로고
    • Successful treatment of murine β-thalassemia intermedia by transfer of the human β-globin gene
    • May, C, Rivella, S, Chadbum, A and Sadelain, M (2002). Successful treatment of murine β-thalassemia intermedia by transfer of the human β-globin gene. Blood 99: 1902-1908.
    • (2002) Blood , vol.99 , pp. 1902-1908
    • May, C.1    Rivella, S.2    Chadbum, A.3    Sadelain, M.4
  • 201
    • 0035861452 scopus 로고    scopus 로고
    • Correction of sickle cell disease in transgenic mouse models by gene therapy
    • Pawliuk, R et al. (2001). Correction of sickle cell disease in transgenic mouse models by gene therapy. Science 294: 2368-2371.
    • (2001) Science , vol.294 , pp. 2368-2371
    • Pawliuk, R.1
  • 202
    • 0037195091 scopus 로고    scopus 로고
    • Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells
    • Imren, S et al. (2002). Permanent and panerythroid correction of murine β thalassemia by multiple lentiviral integration in hematopoietic stem cells. Proc Natl Acad Sci USA 99: 14380-14385.
    • (2002) Proc Natl Acad Sci USA , vol.99 , pp. 14380-14385
    • Imren, S.1
  • 203
    • 0037443467 scopus 로고    scopus 로고
    • The degree of phenotypic correction of murine β-thalassemia intermedia following lentiviral-mediated transfer of a human γ-globin gene is influenced by chromosomal position effects and vector copy number
    • Persons, DA, Hargrove, PW, Allay, ER, Hanawa, H and Nienhuis, AW (2003). The degree of phenotypic correction of murine β-thalassemia intermedia following lentiviral-mediated transfer of a human γ-globin gene is influenced by chromosomal position effects and vector copy number. Blood 101: 2175-2183.
    • (2003) Blood , vol.101 , pp. 2175-2183
    • Persons, D.A.1    Hargrove, P.W.2    Allay, E.R.3    Hanawa, H.4    Nienhuis, A.W.5
  • 204
    • 0037606048 scopus 로고    scopus 로고
    • A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human β-globin gene transfer
    • Rivella, S, May, C, Chadbum, A, Riviere, I and Sadelain, M (2003). A novel murine model of Cooley anemia and its rescue by lentiviral-mediated human β-globin gene transfer. Blood 101: 2932-2939.
    • (2003) Blood , vol.101 , pp. 2932-2939
    • Rivella, S.1    May, C.2    Chadbum, A.3    Riviere, I.4    Sadelain, M.5
  • 205
    • 0344305491 scopus 로고    scopus 로고
    • Correction of a mouse model of sickle cell disease: Lentiviral/antisickling β-globin gene transduction of unmobilized, purified hematopoietic stem cells
    • Levasseur, DN, Ryan, TM, Pawlik, KM and Townes, TM (2003). Correction of a mouse model of sickle cell disease: Lentiviral/antisickling β-globin gene transduction of unmobilized, purified hematopoietic stem cells. Blood 102: 4312-4319.
    • (2003) Blood , vol.102 , pp. 4312-4319
    • Levasseur, D.N.1    Ryan, T.M.2    Pawlik, K.M.3    Townes, T.M.4
  • 206
    • 4944234406 scopus 로고    scopus 로고
    • Extended β-globin locus control region elements promote consistent therapeutic expression of a γ-globin lentiviral vector in murine β-thalassemia
    • Hanawa, H, Hargrove, PW, Kepes, S, Srivastava, Dr, Nienhuis, AW and Persons, DA (2004). Extended β-globin locus control region elements promote consistent therapeutic expression of a γ-globin lentiviral vector in murine β-thalassemia. Blood 104: 2281-2290.
    • (2004) Blood , vol.104 , pp. 2281-2290
    • Hanawa, H.1    Hargrove, P.W.2    Kepes, S.3    Srivastava, D.K.4    Nienhuis, A.W.5    Persons, D.A.6
  • 207
    • 9444281434 scopus 로고    scopus 로고
    • Successful correction of the human β-thalassemia major phenotype using a lentiviral vector
    • Puthenveetil, G et al. (2004). Successful correction of the human β-thalassemia major phenotype using a lentiviral vector. Blood 104: 3445-3453.
    • (2004) Blood , vol.104 , pp. 3445-3453
    • Puthenveetil, G.1
  • 208
    • 0035037078 scopus 로고    scopus 로고
    • Locus control region of the human CD2 gene in a lentivirus vector confers position-independent transgene expression
    • Kowolik, CM, Hu, J and Yee, JK (2001). Locus control region of the human CD2 gene in a lentivirus vector confers position-independent transgene expression. J Virol 75: 4641-4648.
    • (2001) J Virol , vol.75 , pp. 4641-4648
    • Kowolik, C.M.1    Hu, J.2    Yee, J.K.3
  • 210
    • 0037438638 scopus 로고    scopus 로고
    • Gene therapy of apolipoprotein E-deficient mice using a novel macrophage-specific retroviral vector
    • Gough, PJ and Raines, EW (2003). Gene therapy of apolipoprotein E-deficient mice using a novel macrophage-specific retroviral vector. Blood 101: 485-491.
    • (2003) Blood , vol.101 , pp. 485-491
    • Gough, P.J.1    Raines, E.W.2
  • 211
    • 0037079725 scopus 로고    scopus 로고
    • Targeting transgene expression to antigen-presenting cells derived from lentivirus-transduced engrafting human hematopoietic stem/progenitor cells
    • Cui, Y, Golob, J, Kelleher, E, Ye, Z, Pardoll, D and Cheng, L (2002). Targeting transgene expression to antigen-presenting cells derived from lentivirus-transduced engrafting human hematopoietic stem/progenitor cells. Blood 99: 399-408.
    • (2002) Blood , vol.99 , pp. 399-408
    • Cui, Y.1    Golob, J.2    Kelleher, E.3    Ye, Z.4    Pardoll, D.5    Cheng, L.6
  • 212
    • 4043179784 scopus 로고    scopus 로고
    • Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency
    • Tahara, M et al (2004). Trans-splicing repair of CD40 ligand deficiency results in naturally regulated correction of a mouse model of hyper-IgM X-linked immunodeficiency. Nat Med 10: 835-841.
    • (2004) Nat Med , vol.10 , pp. 835-841
    • Tahara, M.1
  • 213
    • 0033198944 scopus 로고    scopus 로고
    • RNA-triggered gene silencing
    • Fire, A (1999). RNA-triggered gene silencing. Trends Genet 15: 358-363.
    • (1999) Trends Genet , vol.15 , pp. 358-363
    • Fire, A.1
  • 214
    • 0035942736 scopus 로고    scopus 로고
    • Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
    • Elbashir, SM, Harborth, J, Lendeckel, W, Yalcin, A, Weber, K and Tuschl, T (2001). Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 411: 494-498.
    • (2001) Nature , vol.411 , pp. 494-498
    • Elbashir, S.M.1    Harborth, J.2    Lendeckel, W.3    Yalcin, A.4    Weber, K.5    Tuschl, T.6
  • 216
    • 16644398798 scopus 로고    scopus 로고
    • Therapeutic applications of RNA interference: Recent advances in siRNA design
    • Scherer, L and Rossi, JJ (2004). Therapeutic applications of RNA interference: Recent advances in siRNA design. Adv Genet 52: 1-21.
    • (2004) Adv Genet , vol.52 , pp. 1-21
    • Scherer, L.1    Rossi, J.J.2
  • 217
    • 19844375161 scopus 로고    scopus 로고
    • Gene therapy progress and prospects: Recent progress in transgene and RNAi expression cassettes
    • III, CR and Chiou, HC (2005). Gene therapy progress and prospects: recent progress in transgene and RNAi expression cassettes. Gene Ther 12: 795-802.
    • (2005) Gene Ther , vol.12 , pp. 795-802
    • III, C.R.1    Chiou, H.C.2
  • 219
    • 4544277478 scopus 로고    scopus 로고
    • Determinants of interferon-stimulated gene induction by RNAi vectors
    • Pebernard, S and Iggo, RD (2004). Determinants of interferon-stimulated gene induction by RNAi vectors. Differentiation 72: 103-111.
    • (2004) Differentiation , vol.72 , pp. 103-111
    • Pebernard, S.1    Iggo, R.D.2
  • 220
    • 21844476451 scopus 로고    scopus 로고
    • Analysis of double-stranded RNA-induced apoptosis pathways using interferon-response noninducible small interfering RNA expression vector library
    • Matsumoto, S, Miyagishi, M, Akashi, H, Nagai, R and Taira, K (2005). Analysis of double-stranded RNA-induced apoptosis pathways using interferon-response noninducible small interfering RNA expression vector library. J Biol Chem 280: 25687-25696.
    • (2005) J Biol Chem , vol.280 , pp. 25687-25696
    • Matsumoto, S.1    Miyagishi, M.2    Akashi, H.3    Nagai, R.4    Taira, K.5
  • 221
    • 30544436003 scopus 로고    scopus 로고
    • A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference
    • Samakoglu, S et al. (2006). A genetic strategy to treat sickle cell anemia by coregulating globin transgene expression and RNA interference. Nat Biotechnol 24: 89-94.
    • (2006) Nat Biotechnol , vol.24 , pp. 89-94
    • Samakoglu, S.1
  • 222
    • 33751251058 scopus 로고    scopus 로고
    • Gene transfer in humans using a conditionally replicating lentiviral vector
    • Levine, BL et al. (2006). Gene transfer in humans using a conditionally replicating lentiviral vector. Proc Natl Acad Sci USA 103: 17372-17377.
    • (2006) Proc Natl Acad Sci USA , vol.103 , pp. 17372-17377
    • Levine, B.L.1
  • 223
    • 0037014337 scopus 로고    scopus 로고
    • No soluble common cytokine receptor gamma chain (gamma(c)) in activated human lymphocyte cultures - Comparison with soluble IL-2Rα
    • Lundin, K, Tuukkanen, AM, Jansson, C, Nordstrom, T and Lindqvist, C (2002). No soluble common cytokine receptor gamma chain (gamma(c)) in activated human lymphocyte cultures - comparison with soluble IL-2Rα. Immunol Lett 82: 235-240.
    • (2002) Immunol Lett , vol.82 , pp. 235-240
    • Lundin, K.1    Tuukkanen, A.M.2    Jansson, C.3    Nordstrom, T.4    Lindqvist, C.5
  • 224
    • 15844426339 scopus 로고    scopus 로고
    • Gamma-c gene transfer into SCID X1 patients' B-cell lines restores normal high-affinity interleukin-2 receptor expression and function
    • Hacein-Bey, H et al. (1996). gamma-c gene transfer into SCID X1 patients' B-cell lines restores normal high-affinity interleukin-2 receptor expression and function. Blood 87: 3108-3116.
    • (1996) Blood , vol.87 , pp. 3108-3116
    • Hacein-Bey, H.1
  • 225
    • 0034724857 scopus 로고    scopus 로고
    • Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease
    • Cavazzana-Calvo, M et al. (2000). Gene therapy of human severe combined immunodeficiency (SCID)-X1 disease. Science 288: 669-672.
    • (2000) Science , vol.288 , pp. 669-672
    • Cavazzana-Calvo, M.1
  • 226
    • 0019403767 scopus 로고
    • Biochemical and functional abnormalities in lymphocytes from an adenosine deaminase-deficient patient during enzyme replacement therapy
    • Hutton, JJ et al. (1981). Biochemical and functional abnormalities in lymphocytes from an adenosine deaminase-deficient patient during enzyme replacement therapy. J Clin Invest 68: 413-421.
    • (1981) J Clin Invest , vol.68 , pp. 413-421
    • Hutton, J.J.1
  • 227
    • 0024460181 scopus 로고
    • Improved retroviral vectors for gene transfer and expression
    • 980-982, 984-986
    • Miller, AD and Rosman, GJ (1989). Improved retroviral vectors for gene transfer and expression. Biotechniques 7: 980-982, 984-986, 989-990.
    • (1989) Biotechniques , vol.7 , pp. 989-990
    • Miller, A.D.1    Rosman, G.J.2
  • 228
    • 18444376150 scopus 로고    scopus 로고
    • Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement
    • Aiuti, A et al. (2002). Immune reconstitution in ADA-SCID after PBL gene therapy and discontinuation of enzyme replacement. Nat Med 8: 423-425.
    • (2002) Nat Med , vol.8 , pp. 423-425
    • Aiuti, A.1
  • 230
    • 33645458063 scopus 로고    scopus 로고
    • Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation
    • Dupre, L et al. (2006). Efficacy of gene therapy for Wiskott-Aldrich syndrome using a WAS promoter/cDNA-containing lentiviral vector and nonlethal irradiation. Hum Gene Ther 17: 303-313.
    • (2006) Hum Gene Ther , vol.17 , pp. 303-313
    • Dupre, L.1
  • 231
    • 28444459497 scopus 로고    scopus 로고
    • Correction of murine hemophilia a by hematopoietic stem cell gene therapy
    • Moayeri, M, Hawley, TS and Hawley, RG (2005). Correction of murine hemophilia a by hematopoietic stem cell gene therapy. Mol Ther 12: 1034-1042.
    • (2005) Mol Ther , vol.12 , pp. 1034-1042
    • Moayeri, M.1    Hawley, T.S.2    Hawley, R.G.3
  • 232
    • 33846196263 scopus 로고    scopus 로고
    • Therapeutic options for patients with severe β-thalassemia: The need for globin gene therapy
    • in press
    • Sadelain, M et al. Therapeutic options for patients with severe β-thalassemia: The need for globin gene therapy. Hum Gene Ther, in press.
    • Hum Gene Ther
    • Sadelain, M.1


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