메뉴 건너뛰기




Volumn 19, Issue 6, 2009, Pages 221-223

RNA-interference-based gene therapy approaches to HIV type-1 treatment: Tackling the hurdles from bench to bedside

Author keywords

[No Author keywords available]

Indexed keywords

LENTIVIRUS VECTOR; MESSENGER RNA; SHORT HAIRPIN RNA; SMALL INTERFERING RNA;

EID: 77949667231     PISSN: 09563202     EISSN: None     Source Type: Journal    
DOI: 10.1177/095632020901900602     Document Type: Review
Times cited : (12)

References (153)
  • 1
    • 0020596551 scopus 로고
    • Isolation of a T-lymphotropic retrovirus from a patient at risk for acquired immune deficiency syndrome (AIDS)
    • Barre-Sinoussi F, Chermann JC, Rey P, et al. Isolation of a T-lymphotropic retrovirus from a patient at risk for acquired immune deficiency syndrome (AIDS). Science 1983; 220:868-871.
    • (1983) Science , vol.220 , pp. 868-871
    • Barre-Sinoussi, F.1    Chermann, J.C.2    Rey, P.3
  • 2
    • 0021261510 scopus 로고
    • Frequent detection and isolation of cytopathic retroviruses (HTLV-III) from patients with AIDS and at risk for AIDS
    • Gallo RC, Salahuddin SZ, Popovic M, et al. Frequent detection and isolation of cytopathic retroviruses (HTLV-III) from patients with AIDS and at risk for AIDS. Science 1984; 224:500-503.
    • (1984) Science , vol.224 , pp. 500-503
    • Gallo, R.C.1    Salahuddin, S.Z.2    Popovic, M.3
  • 3
    • 58749104364 scopus 로고    scopus 로고
    • The promises and pitfalls of RNA-interference- based therapeutics
    • Castanotto D, Rossi JJ. The promises and pitfalls of RNA-interference- based therapeutics. Nature 2009; 457:426-433.
    • (2009) Nature , vol.457 , pp. 426-433
    • Castanotto, D.1    Rossi, J.J.2
  • 4
    • 0242379146 scopus 로고    scopus 로고
    • Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis
    • Ding H, Schwarz DS, Keene A, et al. Selective silencing by RNAi of a dominant allele that causes amyotrophic lateral sclerosis. Aging Cell 2003; 2:209-217.
    • (2003) Aging Cell , vol.2 , pp. 209-217
    • Ding, H.1    Schwarz, D.S.2    Keene, A.3
  • 5
    • 0038023197 scopus 로고    scopus 로고
    • Inhibition of hepatitis B virus in mice by RNA interference
    • McCaffrey AP, Nakai H, Pandey K, et al. Inhibition of hepatitis B virus in mice by RNA interference. Nat Biotechnol 2003; 21:639-644.
    • (2003) Nat Biotechnol , vol.21 , pp. 639-644
    • McCaffrey, A.P.1    Nakai, H.2    Pandey, K.3
  • 6
    • 33745625836 scopus 로고    scopus 로고
    • Therapeutic potential of RNA interference against cancer
    • Takeshita F, Ochiya T. Therapeutic potential of RNA interference against cancer. Cancer Sci 2006; 97:689-696.
    • (2006) Cancer Sci , vol.97 , pp. 689-696
    • Takeshita, F.1    Ochiya, T.2
  • 7
    • 1242316299 scopus 로고    scopus 로고
    • Molecular medicine for the brain: Silencing of disease genes with RNA interference
    • Davidson BL, Paulson HL. Molecular medicine for the brain: silencing of disease genes with RNA interference. Lancet Neurol 2004; 3:145-149.
    • (2004) Lancet Neurol , vol.3 , pp. 145-149
    • Davidson, B.L.1    Paulson, H.L.2
  • 8
    • 0037452625 scopus 로고    scopus 로고
    • Interference of hepatitis C virus RNA replication by short interfering RNAs
    • Kapadia SB, Brideau-Andersen A, Chisari FV. Interference of hepatitis C virus RNA replication by short interfering RNAs. Proc Natl Acad Sci U S A 2003; 100:2014-2018.
    • (2003) Proc Natl Acad Sci U S A , vol.100 , pp. 2014-2018
    • Kapadia, S.B.1    Brideau-Andersen, A.2    Chisari, F.V.3
  • 10
    • 58149458865 scopus 로고    scopus 로고
    • Evaluation of safety and efficacy of RNAi against HIV-1 in the human immune system (Rag-2(-/-)(c)(-/-)) mouse model
    • ter Brake O, Legrand N, von Eije KJ, et al. Evaluation of safety and efficacy of RNAi against HIV-1 in the human immune system (Rag-2(-/-)(c)(-/-)) mouse model. Gene Ther 2009; 16:148-153.
    • (2009) Gene Ther , vol.16 , pp. 148-153
    • ter Brake, O.1    Legrand, N.2    von Eije, K.J.3
  • 11
    • 34848910418 scopus 로고    scopus 로고
    • Return to the RNAi world: Rethinking gene expression and evolution (Nobel Lecture)
    • Mello CC. Return to the RNAi world: rethinking gene expression and evolution (Nobel Lecture). Angew Chem Int Ed Engl 2007; 46:6985-6994.
    • (2007) Angew Chem Int Ed Engl , vol.46 , pp. 6985-6994
    • Mello, C.C.1
  • 12
    • 0035966319 scopus 로고    scopus 로고
    • Ambros V. microRNAs: tiny regulators with great potential. Cell 2001; 107:823-826.
    • Ambros V. microRNAs: tiny regulators with great potential. Cell 2001; 107:823-826.
  • 13
    • 0038613483 scopus 로고    scopus 로고
    • Baehrecke EH. miRNAs: micro managers of programmed cell death. Curr Biol 2003; 13:R473-R475.
    • Baehrecke EH. miRNAs: micro managers of programmed cell death. Curr Biol 2003; 13:R473-R475.
  • 14
    • 0038299558 scopus 로고    scopus 로고
    • Role of microRNAs in plant and animal development
    • Carrington JC, Ambros V. Role of microRNAs in plant and animal development. Science 2003; 301:336-338.
    • (2003) Science , vol.301 , pp. 336-338
    • Carrington, J.C.1    Ambros, V.2
  • 15
    • 0345818258 scopus 로고    scopus 로고
    • McManus MT. microRNAs and cancer. Semin Cancer Biol 2003; 13:253-258.
    • McManus MT. microRNAs and cancer. Semin Cancer Biol 2003; 13:253-258.
  • 16
    • 11144223157 scopus 로고    scopus 로고
    • Conserved and divergent paths that regulate self-renewal in mouse and human embryonic stem cells
    • Rao M. Conserved and divergent paths that regulate self-renewal in mouse and human embryonic stem cells. Dev Biol 2004; 275:269-286.
    • (2004) Dev Biol , vol.275 , pp. 269-286
    • Rao, M.1
  • 17
    • 29144505309 scopus 로고    scopus 로고
    • The widespread impact of mammalian microRNAs on mRNA repression and evolution
    • Farh KK, Grimson A, Jan C, et al. The widespread impact of mammalian microRNAs on mRNA repression and evolution. Science 2005; 310:1817-1821.
    • (2005) Science , vol.310 , pp. 1817-1821
    • Farh, K.K.1    Grimson, A.2    Jan, C.3
  • 18
    • 0347444723 scopus 로고    scopus 로고
    • Bartel DP. microRNAs: genomics, biogenesis, mechanism, and function. Cell 2004; 116:281-297.
    • Bartel DP. microRNAs: genomics, biogenesis, mechanism, and function. Cell 2004; 116:281-297.
  • 19
    • 4644309196 scopus 로고    scopus 로고
    • The functions of animal microRNAs
    • Ambros V. The functions of animal microRNAs. Nature 2004; 431:350-355.
    • (2004) Nature , vol.431 , pp. 350-355
    • Ambros, V.1
  • 20
    • 0037009364 scopus 로고    scopus 로고
    • microRNA maturation: Stepwise processing and subcellular localization
    • Lee Y, Jeon K, Lee JT, Kim S, Kim VN. microRNA maturation: stepwise processing and subcellular localization. EMBO J 2002; 21:4663-4670.
    • (2002) EMBO J , vol.21 , pp. 4663-4670
    • Lee, Y.1    Jeon, K.2    Lee, J.T.3    Kim, S.4    Kim, V.N.5
  • 22
    • 9144225636 scopus 로고    scopus 로고
    • The microprocessor complex mediates the genesis of microRNAs
    • Gregory RI, Yan KP, Amuthan G, et al. The microprocessor complex mediates the genesis of microRNAs. Nature 2004; 432:235-240.
    • (2004) Nature , vol.432 , pp. 235-240
    • Gregory, R.I.1    Yan, K.P.2    Amuthan, G.3
  • 23
    • 10644234841 scopus 로고    scopus 로고
    • The Drosha-DGCR8 complex in primary microRNA processing
    • Han J, Lee Y, Yeom KH, Kim YK, Jin H, Kim VN. The Drosha-DGCR8 complex in primary microRNA processing. Genes Dev 2004; 18:3016-3027.
    • (2004) Genes Dev , vol.18 , pp. 3016-3027
    • Han, J.1    Lee, Y.2    Yeom, K.H.3    Kim, Y.K.4    Jin, H.5    Kim, V.N.6
  • 24
    • 10344248903 scopus 로고    scopus 로고
    • The human DiGeorge syndrome critical region gene 8 and its D. melanogaster homolog are required for miRNA biogenesis
    • Landthaler M, Yalcin A, Tuschl T. The human DiGeorge syndrome critical region gene 8 and its D. melanogaster homolog are required for miRNA biogenesis. Curr Biol 2004; 14:2162-2167.
    • (2004) Curr Biol , vol.14 , pp. 2162-2167
    • Landthaler, M.1    Yalcin, A.2    Tuschl, T.3
  • 25
    • 0141843656 scopus 로고    scopus 로고
    • The nuclear RNase III Drosha initiates microRNA processing
    • Lee Y, Ahn C, Han J, et al. The nuclear RNase III Drosha initiates microRNA processing. Nature 2003; 425:415-419.
    • (2003) Nature , vol.425 , pp. 415-419
    • Lee, Y.1    Ahn, C.2    Han, J.3
  • 26
    • 34447097693 scopus 로고    scopus 로고
    • Intronic microRNA precursors that bypass Drosha processing
    • Ruby JG, Jan CH, Bartel DP. Intronic microRNA precursors that bypass Drosha processing. Nature 2007; 448:83-86.
    • (2007) Nature , vol.448 , pp. 83-86
    • Ruby, J.G.1    Jan, C.H.2    Bartel, D.P.3
  • 27
    • 35549005351 scopus 로고    scopus 로고
    • And now introducing mammalian mirtrons
    • Chan SP, Slack FJ. And now introducing mammalian mirtrons. Dev Cell 2007; 13:605-607.
    • (2007) Dev Cell , vol.13 , pp. 605-607
    • Chan, S.P.1    Slack, F.J.2
  • 28
    • 1642499415 scopus 로고    scopus 로고
    • Exportin 5 is a RanGTP-dependent dsRNA-binding protein that mediates nuclear export of pre-miRNAs
    • Bohnsack MT, Czaplinski K, Gorlich D. Exportin 5 is a RanGTP-dependent dsRNA-binding protein that mediates nuclear export of pre-miRNAs. RNA 2004; 10:185-191.
    • (2004) RNA , vol.10 , pp. 185-191
    • Bohnsack, M.T.1    Czaplinski, K.2    Gorlich, D.3
  • 30
    • 0347361541 scopus 로고    scopus 로고
    • Exportin-5 mediates the nuclear export of pre-microRNAs and short hairpin RNAs
    • Yi R, Qin Y, Macara IG, Cullen BR. Exportin-5 mediates the nuclear export of pre-microRNAs and short hairpin RNAs. Genes Dev 2003; 17:3011-3016.
    • (2003) Genes Dev , vol.17 , pp. 3011-3016
    • Yi, R.1    Qin, Y.2    Macara, I.G.3    Cullen, B.R.4
  • 31
    • 3142613181 scopus 로고    scopus 로고
    • Single processing center models for human Dicer and bacterial RNase III
    • Zhang H, Kolb FA, Jaskiewicz L, Westhof E, Filipowicz W. Single processing center models for human Dicer and bacterial RNase III. Cell 2004; 118:57-68.
    • (2004) Cell , vol.118 , pp. 57-68
    • Zhang, H.1    Kolb, F.A.2    Jaskiewicz, L.3    Westhof, E.4    Filipowicz, W.5
  • 32
    • 23644433363 scopus 로고    scopus 로고
    • TRBP recruits the Dicer complex to Ago2 for microRNA processing and gene silencing
    • Chendrimada TP, Gregory RI, Kumaraswamy E, et al. TRBP recruits the Dicer complex to Ago2 for microRNA processing and gene silencing. Nature 2005; 436:740-744.
    • (2005) Nature , vol.436 , pp. 740-744
    • Chendrimada, T.P.1    Gregory, R.I.2    Kumaraswamy, E.3
  • 33
    • 27744537851 scopus 로고    scopus 로고
    • Human RISC couples microRNA biogenesis and posttranscriptional gene silencing
    • Gregory RI, Chendrimada TP, Cooch N, Shiekhattar R. Human RISC couples microRNA biogenesis and posttranscriptional gene silencing. Cell 2005; 123:631-640.
    • (2005) Cell , vol.123 , pp. 631-640
    • Gregory, R.I.1    Chendrimada, T.P.2    Cooch, N.3    Shiekhattar, R.4
  • 34
    • 29144431656 scopus 로고    scopus 로고
    • A human, ATP-independent, RISC assembly machine fueled by pre-miRNA
    • Maniataki E, Mourelatos Z. A human, ATP-independent, RISC assembly machine fueled by pre-miRNA. Genes Dev 2005; 19:2979-2990.
    • (2005) Genes Dev , vol.19 , pp. 2979-2990
    • Maniataki, E.1    Mourelatos, Z.2
  • 36
    • 14644393684 scopus 로고    scopus 로고
    • Perspective: Machines for RNAi
    • Tomari Y, Zamore PD. Perspective: machines for RNAi. Genes Dev 2005; 19:517-529.
    • (2005) Genes Dev , vol.19 , pp. 517-529
    • Tomari, Y.1    Zamore, P.D.2
  • 38
    • 0037418839 scopus 로고    scopus 로고
    • bantam Encodes a developmentally regulated microRNA that controls cell proliferation and regulates the proapoptotic gene hid in Drosophila
    • Brennecke J, Hipfner DR, Stark A, Russell RB, Cohen SM. bantam Encodes a developmentally regulated microRNA that controls cell proliferation and regulates the proapoptotic gene hid in Drosophila. Cell 2003; 113:25-36.
    • (2003) Cell , vol.113 , pp. 25-36
    • Brennecke, J.1    Hipfner, D.R.2    Stark, A.3    Russell, R.B.4    Cohen, S.M.5
  • 39
    • 1642374097 scopus 로고    scopus 로고
    • Specificity of microRNA target selection in translational repression
    • Doench JG, Sharp PA. Specificity of microRNA target selection in translational repression. Genes Dev 2004; 18:504-511.
    • (2004) Genes Dev , vol.18 , pp. 504-511
    • Doench, J.G.1    Sharp, P.A.2
  • 40
    • 2442672918 scopus 로고    scopus 로고
    • A combined computational-experimental approach predicts human microRNA targets
    • Kiriakidou M, Nelson PT, Kouranov A, et al. A combined computational-experimental approach predicts human microRNA targets. Genes Dev 2004; 18:1165-1178.
    • (2004) Genes Dev , vol.18 , pp. 1165-1178
    • Kiriakidou, M.1    Nelson, P.T.2    Kouranov, A.3
  • 41
    • 0036544755 scopus 로고    scopus 로고
    • Micro RNAs are complementary to 3′ UTR sequence motifs that mediate negative post-transcriptional regulation
    • Lai EC. Micro RNAs are complementary to 3′ UTR sequence motifs that mediate negative post-transcriptional regulation. Nat Genet 2002; 30:363-364.
    • (2002) Nat Genet , vol.30 , pp. 363-364
    • Lai, E.C.1
  • 44
    • 2142654329 scopus 로고    scopus 로고
    • microRNA-directed cleavage of HOXB8 mRNA
    • Yekta S, Shih IH, Bartel DP. microRNA-directed cleavage of HOXB8 mRNA. Science 2004; 304:594-596.
    • (2004) Science , vol.304 , pp. 594-596
    • Yekta, S.1    Shih, I.H.2    Bartel, D.P.3
  • 45
    • 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, Tuschl T. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature 2001; 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
  • 46
    • 0037134020 scopus 로고    scopus 로고
    • A system for stable expression of short interfering RNAs in mammalian cells
    • Brummelkamp TR, Bernards R, Agami R. A system for stable expression of short interfering RNAs in mammalian cells. Science 2002; 296:550-553.
    • (2002) Science , vol.296 , pp. 550-553
    • Brummelkamp, T.R.1    Bernards, R.2    Agami, R.3
  • 47
    • 0037089589 scopus 로고    scopus 로고
    • Short hairpin RNAs (shRNAs) induce sequence-specific silencing in mammalian cells
    • Paddison PJ, Caudy AA, Bernstein E, Hannon GJ, Conklin DS. Short hairpin RNAs (shRNAs) induce sequence-specific silencing in mammalian cells. Genes Dev 2002; 16:948-958.
    • (2002) Genes Dev , vol.16 , pp. 948-958
    • Paddison, P.J.1    Caudy, A.A.2    Bernstein, E.3    Hannon, G.J.4    Conklin, D.S.5
  • 48
    • 0036294527 scopus 로고    scopus 로고
    • Both natural and designed micro RNAs can inhibit the expression of cognate mRNAs when expressed in human cells
    • Zeng Y, Wagner EJ, Cullen BR. Both natural and designed micro RNAs can inhibit the expression of cognate mRNAs when expressed in human cells. Mol Cell 2002; 9:1327-1333.
    • (2002) Mol Cell , vol.9 , pp. 1327-1333
    • Zeng, Y.1    Wagner, E.J.2    Cullen, B.R.3
  • 49
    • 33745299723 scopus 로고    scopus 로고
    • Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
    • Grimm D, Streetz KL, Jopling CL, et al. Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 2006; 441:537-541.
    • (2006) Nature , vol.441 , pp. 537-541
    • Grimm, D.1    Streetz, K.L.2    Jopling, C.L.3
  • 50
    • 44449121785 scopus 로고    scopus 로고
    • Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi
    • McBride JL, Boudreau RL, Harper SQ, et al. Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi. Proc Natl Acad Sci U S A 2008; 105:5868-5873.
    • (2008) Proc Natl Acad Sci U S A , vol.105 , pp. 5868-5873
    • McBride, J.L.1    Boudreau, R.L.2    Harper, S.Q.3
  • 51
    • 50649121877 scopus 로고    scopus 로고
    • Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs
    • Boudreau RL, Monteys AM, Davidson BL. Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs. RNA 2008; 14:1834-1844.
    • (2008) RNA , vol.14 , pp. 1834-1844
    • Boudreau, R.L.1    Monteys, A.M.2    Davidson, B.L.3
  • 52
    • 34548548614 scopus 로고    scopus 로고
    • Combinatorial delivery of small interfering RNAs reduces RNAi efficacy by selective incorporation into RISC
    • Castanotto D, Sakurai K, Lingeman R, et al. Combinatorial delivery of small interfering RNAs reduces RNAi efficacy by selective incorporation into RISC. Nucleic Acids Res 2007; 35:5154-5164.
    • (2007) Nucleic Acids Res , vol.35 , pp. 5154-5164
    • Castanotto, D.1    Sakurai, K.2    Lingeman, R.3
  • 53
    • 36148972467 scopus 로고    scopus 로고
    • Reduced levels of Ago2 expression result in increased siRNA competition in mammalian cells
    • Vickers TA, Lima WF, Nichols JG, Crooke ST. Reduced levels of Ago2 expression result in increased siRNA competition in mammalian cells. Nucleic Acids Res 2007; 35:6598-6610.
    • (2007) Nucleic Acids Res , vol.35 , pp. 6598-6610
    • Vickers, T.A.1    Lima, W.F.2    Nichols, J.G.3    Crooke, S.T.4
  • 55
    • 0037685280 scopus 로고    scopus 로고
    • Expression profiling reveals off-target gene regulation by RNAi
    • Jackson AL, Bartz SR, Schelter J, et al. Expression profiling reveals off-target gene regulation by RNAi. Nat Biotechnol 2003; 21:635-637.
    • (2003) Nat Biotechnol , vol.21 , pp. 635-637
    • Jackson, A.L.1    Bartz, S.R.2    Schelter, J.3
  • 56
    • 33745608078 scopus 로고    scopus 로고
    • Off-target effects by siRNA can induce toxic phenotype
    • Fedorov Y, Anderson EM, Birmingham A, et al. Off-target effects by siRNA can induce toxic phenotype. RNA 2006; 12:1188-1196.
    • (2006) RNA , vol.12 , pp. 1188-1196
    • Fedorov, Y.1    Anderson, E.M.2    Birmingham, A.3
  • 57
    • 33745612464 scopus 로고    scopus 로고
    • Widespread siRNA "off-target" transcript silencing mediated by seed region sequence complementarity
    • Jackson AL, Burchard J, Schelter J, et al. Widespread siRNA "off-target" transcript silencing mediated by seed region sequence complementarity. RNA 2006; 12:1179-1187.
    • (2006) RNA , vol.12 , pp. 1179-1187
    • Jackson, A.L.1    Burchard, J.2    Schelter, J.3
  • 58
    • 33646188259 scopus 로고    scopus 로고
    • 3′ UTR seed matches, but not overall identity, are associated with RNAi off-targets
    • Birmingham A, Anderson EM, Reynolds A, et al. 3′ UTR seed matches, but not overall identity, are associated with RNAi off-targets. Nat Methods 2006; 3:199-204.
    • (2006) Nat Methods , vol.3 , pp. 199-204
    • Birmingham, A.1    Anderson, E.M.2    Reynolds, A.3
  • 59
    • 0038419800 scopus 로고    scopus 로고
    • Induction of an interferon response by RNAi vectors in mammalian cells
    • Bridge AJ, Pebernard S, Ducraux A, Nicoulaz AL, Iggo R. Induction of an interferon response by RNAi vectors in mammalian cells. Nat Genet 2003; 34:263-264.
    • (2003) Nat Genet , vol.34 , pp. 263-264
    • Bridge, A.J.1    Pebernard, S.2    Ducraux, A.3    Nicoulaz, A.L.4    Iggo, R.5
  • 61
    • 27944459341 scopus 로고    scopus 로고
    • Activation of the mammalian immune system by siRNAs
    • Marques JT, Williams BR. Activation of the mammalian immune system by siRNAs. Nat Biotechnol 2005; 23:1399-1405.
    • (2005) Nat Biotechnol , vol.23 , pp. 1399-1405
    • Marques, J.T.1    Williams, B.R.2
  • 62
    • 49549117077 scopus 로고    scopus 로고
    • T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice
    • Kumar P, Ban HS, Kim SS, et al. T cell-specific siRNA delivery suppresses HIV-1 infection in humanized mice. Cell 2008; 134:577-586.
    • (2008) Cell , vol.134 , pp. 577-586
    • Kumar, P.1    Ban, H.S.2    Kim, S.S.3
  • 63
    • 0029996147 scopus 로고    scopus 로고
    • In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector
    • Naldini L, Blomer U, Gallay P, et al. In vivo gene delivery and stable transduction of nondividing cells by a lentiviral vector. Science 1996; 272:263-267.
    • (1996) Science , vol.272 , pp. 263-267
    • Naldini, L.1    Blomer, U.2    Gallay, P.3
  • 64
    • 0032005193 scopus 로고    scopus 로고
    • Neither human immunodeficiency virus-1 (HIV-1) nor HIV-2 infects most-primitive human hematopoietic stem cells as assessed in long-term bone marrow cultures
    • Weichold FF, Zella D, Barabitskaja O, et al. Neither human immunodeficiency virus-1 (HIV-1) nor HIV-2 infects most-primitive human hematopoietic stem cells as assessed in long-term bone marrow cultures. Blood 1998; 91:907-915.
    • (1998) Blood , vol.91 , pp. 907-915
    • Weichold, F.F.1    Zella, D.2    Barabitskaja, O.3
  • 65
    • 33846809447 scopus 로고    scopus 로고
    • Primitive hematopoietic cells resist HIV-1 infection via p21
    • Zhang J, Scadden DT, Crumpacker CS. Primitive hematopoietic cells resist HIV-1 infection via p21. J Clin Invest 2007; 117:473-481.
    • (2007) J Clin Invest , vol.117 , pp. 473-481
    • Zhang, J.1    Scadden, D.T.2    Crumpacker, C.S.3
  • 66
    • 12144291445 scopus 로고    scopus 로고
    • Anti-human immunodeficiency virus hematopoietic progenitor cell-delivered ribozyme in a phase I study: Myeloid and lymphoid reconstitution in human immunodeficiency virus type-1-infected patients
    • Amado RG, Mitsuyasu RT, Rosenblatt JD, et al. Anti-human immunodeficiency virus hematopoietic progenitor cell-delivered ribozyme in a phase I study: myeloid and lymphoid reconstitution in human immunodeficiency virus type-1-infected patients. Hum Gene Ther 2004; 15:251-262.
    • (2004) Hum Gene Ther , vol.15 , pp. 251-262
    • Amado, R.G.1    Mitsuyasu, R.T.2    Rosenblatt, J.D.3
  • 69
    • 0035661288 scopus 로고    scopus 로고
    • Lentivirus in the clinic
    • Dropulic B. Lentivirus in the clinic. Mol Ther 2001; 4:511-512.
    • (2001) Mol Ther , vol.4 , pp. 511-512
    • Dropulic, B.1
  • 70
    • 0142124143 scopus 로고    scopus 로고
    • Human immunodeficiency virus type 1 escape from RNA interference
    • Boden D, Pusch O, Lee F, Tucker L, Ramratnam B. Human immunodeficiency virus type 1 escape from RNA interference. J Virol 2003; 77:11531-11535.
    • (2003) J Virol , vol.77 , pp. 11531-11535
    • Boden, D.1    Pusch, O.2    Lee, F.3    Tucker, L.4    Ramratnam, B.5
  • 71
    • 1242319384 scopus 로고    scopus 로고
    • Human immunodeficiency virus type 1 escapes from RNA interference-mediated inhibition
    • Das AT, Brummelkamp TR, Westerhout EM, et al. Human immunodeficiency virus type 1 escapes from RNA interference-mediated inhibition. J Virol 2004; 78:2601-2605.
    • (2004) J Virol , vol.78 , pp. 2601-2605
    • Das, A.T.1    Brummelkamp, T.R.2    Westerhout, E.M.3
  • 72
    • 33746210111 scopus 로고    scopus 로고
    • Effective suppression of human immunodeficiency virus type 1 through a combination of short- or long-hairpin RNAs targeting essential sequences for retroviral integration
    • Nishitsuji H, Kohara M, Kannagi M, Masuda T. Effective suppression of human immunodeficiency virus type 1 through a combination of short- or long-hairpin RNAs targeting essential sequences for retroviral integration. J Virol 2006; 80:7658-7666.
    • (2006) J Virol , vol.80 , pp. 7658-7666
    • Nishitsuji, H.1    Kohara, M.2    Kannagi, M.3    Masuda, T.4
  • 73
    • 30344474936 scopus 로고    scopus 로고
    • Sequence homology required by human immunodeficiency virus type 1 to escape from short interfering RNAs
    • Sabariegos R, Gimenez-Barcons M, Tapia N, Clotet B, Martinez MA. Sequence homology required by human immunodeficiency virus type 1 to escape from short interfering RNAs. J Virol 2006; 80:571-577.
    • (2006) J Virol , vol.80 , pp. 571-577
    • Sabariegos, R.1    Gimenez-Barcons, M.2    Tapia, N.3    Clotet, B.4    Martinez, M.A.5
  • 74
    • 33751001588 scopus 로고    scopus 로고
    • Silencing of HIV-1 with RNA interference: A multiple shRNA approach
    • ter Brake O, Konstantinova P, Ceylan M, Berkhout B. Silencing of HIV-1 with RNA interference: a multiple shRNA approach. Mol Ther 2006; 14:883-892.
    • (2006) Mol Ther , vol.14 , pp. 883-892
    • ter Brake, O.1    Konstantinova, P.2    Ceylan, M.3    Berkhout, B.4
  • 75
    • 32444432596 scopus 로고    scopus 로고
    • Novel Pol II fusion promoter directs human immunodeficiency virus type 1-inducible coexpression of a short hairpin RNA and protein
    • Unwalla HJ, Li HT, Bahner I, Li MJ, Kohn D, Rossi JJ. Novel Pol II fusion promoter directs human immunodeficiency virus type 1-inducible coexpression of a short hairpin RNA and protein. J Virol 2006; 80:1863-1873.
    • (2006) J Virol , vol.80 , pp. 1863-1873
    • Unwalla, H.J.1    Li, H.T.2    Bahner, I.3    Li, M.J.4    Kohn, D.5    Rossi, J.J.6
  • 76
    • 13844320402 scopus 로고    scopus 로고
    • HIV-1 can escape from RNA interference by evolving an alternative structure in its RNA genome
    • Westerhout EM, Ooms M, Vink M, Das AT, Berkhout B. HIV-1 can escape from RNA interference by evolving an alternative structure in its RNA genome. Nucleic Acids Res 2005; 33:796-804.
    • (2005) Nucleic Acids Res , vol.33 , pp. 796-804
    • Westerhout, E.M.1    Ooms, M.2    Vink, M.3    Das, A.T.4    Berkhout, B.5
  • 77
    • 36849042601 scopus 로고    scopus 로고
    • RNA interference against viruses: Strike and counterstrike
    • Haasnoot J, Westerhout EM, Berkhout B. RNA interference against viruses: strike and counterstrike. Nat Biotechnol 2007; 25:1435-1443.
    • (2007) Nat Biotechnol , vol.25 , pp. 1435-1443
    • Haasnoot, J.1    Westerhout, E.M.2    Berkhout, B.3
  • 78
    • 40149092147 scopus 로고    scopus 로고
    • Human immunodeficiency virus type 1 escape is restricted when conserved genome sequences are targeted by RNA interference
    • von Eije KJ, ter Brake O, Berkhout B. Human immunodeficiency virus type 1 escape is restricted when conserved genome sequences are targeted by RNA interference. J Virol 2008; 82:2895-2903.
    • (2008) J Virol , vol.82 , pp. 2895-2903
    • von Eije, K.J.1    ter Brake, O.2    Berkhout, B.3
  • 80
    • 27744605569 scopus 로고    scopus 로고
    • Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy
    • Li MJ, Kim J, Li S, et al. Long-term inhibition of HIV-1 infection in primary hematopoietic cells by lentiviral vector delivery of a triple combination of anti-HIV shRNA, anti-CCR5 ribozyme, and a nucleolar-localizing TAR decoy. Mol Ther 2005; 12:900-909.
    • (2005) Mol Ther , vol.12 , pp. 900-909
    • Li, M.J.1    Kim, J.2    Li, S.3
  • 81
    • 33646736068 scopus 로고    scopus 로고
    • A novel approach for inhibition of HIV-1 by RNA interference: Counteracting viral escape with a second generation of siRNAs
    • ter Brake O, Berkhout B. A novel approach for inhibition of HIV-1 by RNA interference: counteracting viral escape with a second generation of siRNAs. Journal of RNAi and Gene Silencing 2005; 1:56-65.
    • (2005) Journal of RNAi and Gene Silencing , vol.1 , pp. 56-65
    • ter Brake, O.1    Berkhout, B.2
  • 82
    • 34548574859 scopus 로고    scopus 로고
    • Lentiviral vectors that carry anti-HIV shRNAs: Problems and solutions
    • ter Brake O, Berkhout B. Lentiviral vectors that carry anti-HIV shRNAs: problems and solutions. J Gene Med 2007; 9:743-750.
    • (2007) J Gene Med , vol.9 , pp. 743-750
    • ter Brake, O.1    Berkhout, B.2
  • 83
    • 44449120988 scopus 로고    scopus 로고
    • Production and titration of lentiviral vectors
    • Chapter 4:Unit 4.21
    • Salmon P, Trono D. Production and titration of lentiviral vectors. Curr Protoc Neurosci 2006; Chapter 4:Unit 4.21.
    • (2006) Curr Protoc Neurosci
    • Salmon, P.1    Trono, D.2
  • 85
    • 39549117983 scopus 로고    scopus 로고
    • Delivery vehicles for small interfering RNA in vivo
    • de Fougerolles AR. Delivery vehicles for small interfering RNA in vivo. Hum Gene Ther 2008; 19:125-132.
    • (2008) Hum Gene Ther , vol.19 , pp. 125-132
    • de Fougerolles, A.R.1
  • 86
    • 34547843654 scopus 로고    scopus 로고
    • A systematic analysis of the effect of target RNA structure on RNA interference
    • Westerhout EM, Berkhout B. A systematic analysis of the effect of target RNA structure on RNA interference. Nucleic Acids Res 2007; 35:4322-4330.
    • (2007) Nucleic Acids Res , vol.35 , pp. 4322-4330
    • Westerhout, E.M.1    Berkhout, B.2
  • 87
    • 0037173610 scopus 로고    scopus 로고
    • Modulation of HIV-1 replication by RNA interference
    • Jacque JM, Triques K, Stevenson M. Modulation of HIV-1 replication by RNA interference. Nature 2002; 418:435-438.
    • (2002) Nature , vol.418 , pp. 435-438
    • Jacque, J.M.1    Triques, K.2    Stevenson, M.3
  • 88
    • 22744451729 scopus 로고    scopus 로고
    • Lentiviral siRNAs targeting multiple highly conserved RNA sequences of human immunodeficiency virus type 1
    • Chang LJ, Liu X, He J. Lentiviral siRNAs targeting multiple highly conserved RNA sequences of human immunodeficiency virus type 1. Gene Ther 2005; 12:1133-1144.
    • (2005) Gene Ther , vol.12 , pp. 1133-1144
    • Chang, L.J.1    Liu, X.2    He, J.3
  • 89
    • 77949767696 scopus 로고    scopus 로고
    • Towards an RNAi-based gene therapy
    • Berkhout B, ter Brake O. Towards an RNAi-based gene therapy. BIOforum Europe 2008; 4:35-37.
    • (2008) BIOforum Europe , vol.4 , pp. 35-37
    • Berkhout, B.1    ter Brake, O.2
  • 90
    • 0036066508 scopus 로고    scopus 로고
    • siRNA-directed inhibition of HIV-1 infection
    • Novina CD, Murray MF, Dykxhoorn DM, et al. siRNA-directed inhibition of HIV-1 infection. Nat Med 2002; 8:681-686.
    • (2002) Nat Med , vol.8 , pp. 681-686
    • Novina, C.D.1    Murray, M.F.2    Dykxhoorn, D.M.3
  • 91
    • 0037112667 scopus 로고    scopus 로고
    • Prevention of HIV-1 infection in human peripheral blood mononuclear cells by specific RNA interference
    • Park WS, Miyano-Kurosaki N, Hayafune M, et al. Prevention of HIV-1 infection in human peripheral blood mononuclear cells by specific RNA interference. Nucleic Acids Res 2002; 30:4830-4835.
    • (2002) Nucleic Acids Res , vol.30 , pp. 4830-4835
    • Park, W.S.1    Miyano-Kurosaki, N.2    Hayafune, M.3
  • 92
    • 0036891843 scopus 로고    scopus 로고
    • RNA interference directed against viral and cellular targets inhibits human immunodeficiency virus type 1 replication
    • Surabhi RM, Gaynor RB. RNA interference directed against viral and cellular targets inhibits human immunodeficiency virus type 1 replication. J Virol 2002; 76:12963-12973.
    • (2002) J Virol , vol.76 , pp. 12963-12973
    • Surabhi, R.M.1    Gaynor, R.B.2
  • 93
    • 0036721314 scopus 로고    scopus 로고
    • Potent and specific inhibition of human immunodeficiency virus type 1 replication by RNA interference
    • Coburn GA, Cullen BR. Potent and specific inhibition of human immunodeficiency virus type 1 replication by RNA interference. J Virol 2002; 76:9225-9231.
    • (2002) J Virol , vol.76 , pp. 9225-9231
    • Coburn, G.A.1    Cullen, B.R.2
  • 94
    • 0036256430 scopus 로고    scopus 로고
    • Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells
    • Lee NS, Dohjima T, Bauer G, et al. Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat Biotechnol 2002; 20:500-505.
    • (2002) Nat Biotechnol , vol.20 , pp. 500-505
    • Lee, N.S.1    Dohjima, T.2    Bauer, G.3
  • 95
    • 65949091600 scopus 로고    scopus 로고
    • Stringent testing identifies highly potent and escape-proof anti-HIV short hairpin RNAs
    • von Eije KJ, ter Brake O, Berkhout B. Stringent testing identifies highly potent and escape-proof anti-HIV short hairpin RNAs. J Gene Med 2009; 11:459-467.
    • (2009) J Gene Med , vol.11 , pp. 459-467
    • von Eije, K.J.1    ter Brake, O.2    Berkhout, B.3
  • 96
    • 0037032933 scopus 로고    scopus 로고
    • Suppression of chemokine receptor expression by RNA interference allows for inhibition of HIV-1 replication
    • Martinez MA, Gutierrez A, Armand-Ugon M, et al. Suppression of chemokine receptor expression by RNA interference allows for inhibition of HIV-1 replication. AIDS 2002; 16:2385-2390.
    • (2002) AIDS , vol.16 , pp. 2385-2390
    • Martinez, M.A.1    Gutierrez, A.2    Armand-Ugon, M.3
  • 97
    • 49849102544 scopus 로고    scopus 로고
    • Knockdown of cellular RNA helicase DDX3 by short hairpin RNAs suppresses HIV-1 viral replication without inducing apoptosis
    • Ishaq M, Hu J, Wu X, et al. Knockdown of cellular RNA helicase DDX3 by short hairpin RNAs suppresses HIV-1 viral replication without inducing apoptosis. Mol Biotechnol 2008; 39:231-238.
    • (2008) Mol Biotechnol , vol.39 , pp. 231-238
    • Ishaq, M.1    Hu, J.2    Wu, X.3
  • 98
    • 32444439020 scopus 로고    scopus 로고
    • Transient and stable knockdown of the integrase cofactor LEDGF/ p75 reveals its role in the replication cycle of human immunodeficiency virus
    • Vandekerckhove L, Christ F, Van Maele B, et al. Transient and stable knockdown of the integrase cofactor LEDGF/ p75 reveals its role in the replication cycle of human immunodeficiency virus. J Virol 2006; 80:1886-1896.
    • (2006) J Virol , vol.80 , pp. 1886-1896
    • Vandekerckhove, L.1    Christ, F.2    Van Maele, B.3
  • 99
    • 34548779028 scopus 로고    scopus 로고
    • Stable reduction of CCR5 by RNAi through hematopoietic stem cell transplant in non-human primates
    • An DS, Donahue RE, Kamata M, et al. Stable reduction of CCR5 by RNAi through hematopoietic stem cell transplant in non-human primates. Proc Natl Acad Sci U S A 2007; 104:13110-13115.
    • (2007) Proc Natl Acad Sci U S A , vol.104 , pp. 13110-13115
    • An, D.S.1    Donahue, R.E.2    Kamata, M.3
  • 100
    • 33745485344 scopus 로고    scopus 로고
    • CCR5: No longer a "good for nothing" gene - chemokine control of West Nile virus infection
    • Lim JK, Glass WG, McDermott DH, Murphy PM. CCR5: no longer a "good for nothing" gene - chemokine control of West Nile virus infection. Trends Immunol 2006; 27:308-312.
    • (2006) Trends Immunol , vol.27 , pp. 308-312
    • Lim, J.K.1    Glass, W.G.2    McDermott, D.H.3    Murphy, P.M.4
  • 101
    • 1642564331 scopus 로고    scopus 로고
    • Bispecific short hairpin siRNA constructs targeted to CD4, CXCR4, and CCR5 confer HIV-1 resistance
    • Anderson J, Banerjea A, Akkina R. Bispecific short hairpin siRNA constructs targeted to CD4, CXCR4, and CCR5 confer HIV-1 resistance. Oligonucleotides 2003; 13:303-312.
    • (2003) Oligonucleotides , vol.13 , pp. 303-312
    • Anderson, J.1    Banerjea, A.2    Akkina, R.3
  • 102
    • 33748438866 scopus 로고    scopus 로고
    • Optimization and functional effects of stable short hairpin RNA expression in primary human lymphocytes via lentiviral vectors
    • An DS, Qin FX, Auyeung VC, et al. Optimization and functional effects of stable short hairpin RNA expression in primary human lymphocytes via lentiviral vectors. Mol Ther 2006; 14:494-504.
    • (2006) Mol Ther , vol.14 , pp. 494-504
    • An, D.S.1    Qin, F.X.2    Auyeung, V.C.3
  • 103
    • 0034932187 scopus 로고    scopus 로고
    • Mechanism of human stem cell migration and repopulation of NOD/SCID and B2mnull NOD/SCID mice. The role of SDF-1/CXCR4 interactions
    • Lapidot T. Mechanism of human stem cell migration and repopulation of NOD/SCID and B2mnull NOD/SCID mice. The role of SDF-1/CXCR4 interactions. Ann N Y Acad Sci 2001; 938:83-95.
    • (2001) Ann N Y Acad Sci , vol.938 , pp. 83-95
    • Lapidot, T.1
  • 104
    • 39349097864 scopus 로고    scopus 로고
    • Identification of host proteins required for HIV infection through a functional genomic screen
    • Brass AL, Dykxhoorn DM, Benita Y, et al. Identification of host proteins required for HIV infection through a functional genomic screen. Science 2008; 319:921-926.
    • (2008) Science , vol.319 , pp. 921-926
    • Brass, A.L.1    Dykxhoorn, D.M.2    Benita, Y.3
  • 105
    • 55249088255 scopus 로고    scopus 로고
    • Genome-scale RNAi screen for host factors required for HIV replication
    • Zhou H, Xu M, Huang Q, et al. Genome-scale RNAi screen for host factors required for HIV replication. Cell Host Microbe 2008; 4:495-504.
    • (2008) Cell Host Microbe , vol.4 , pp. 495-504
    • Zhou, H.1    Xu, M.2    Huang, Q.3
  • 106
    • 52949130695 scopus 로고    scopus 로고
    • Global analysis of host-pathogen interactions that regulate early-stage HIV-1 replication
    • Konig R, Zhou Y, Elleder D, et al. Global analysis of host-pathogen interactions that regulate early-stage HIV-1 replication. Cell 2008; 135:49-60.
    • (2008) Cell , vol.135 , pp. 49-60
    • Konig, R.1    Zhou, Y.2    Elleder, D.3
  • 107
    • 0027198459 scopus 로고
    • Inhibition of human immunodeficiency virus type 1 replication by regulated expression of a polymeric Tat activation response RNA decoy as a strategy for gene therapy in AIDS
    • Lisziewicz J, Sun D, Smythe J, et al. Inhibition of human immunodeficiency virus type 1 replication by regulated expression of a polymeric Tat activation response RNA decoy as a strategy for gene therapy in AIDS. Proc Natl Acad Sci U S A 1993; 90:8000-8004.
    • (1993) Proc Natl Acad Sci U S A , vol.90 , pp. 8000-8004
    • Lisziewicz, J.1    Sun, D.2    Smythe, J.3
  • 108
    • 0025363891 scopus 로고
    • Ribozymes as potential anti-HIV-1 therapeutic agents
    • Sarver N, Cantin EM, Chang PS, et al. Ribozymes as potential anti-HIV-1 therapeutic agents. Science 1990; 247:1222-1225.
    • (1990) Science , vol.247 , pp. 1222-1225
    • Sarver, N.1    Cantin, E.M.2    Chang, P.S.3
  • 110
    • 34247278567 scopus 로고    scopus 로고
    • Combinatorial RNAi: A winning strategy for the race against evolving targets?
    • Grimm D, Kay MA. Combinatorial RNAi: a winning strategy for the race against evolving targets? Mol Ther 2007; 15:878-888.
    • (2007) Mol Ther , vol.15 , pp. 878-888
    • Grimm, D.1    Kay, M.A.2
  • 111
    • 0027055182 scopus 로고
    • Dual-target inhibition of HIV-1 in vitro by means of an adeno-associated virus antisense vector
    • Chatterjee S, Johnson PR, Wong KK, Jr. Dual-target inhibition of HIV-1 in vitro by means of an adeno-associated virus antisense vector. Science 1992; 258:1485-1488.
    • (1992) Science , vol.258 , pp. 1485-1488
    • Chatterjee, S.1    Johnson, P.R.2    Wong Jr., K.K.3
  • 112
    • 33751251058 scopus 로고    scopus 로고
    • Gene transfer in humans using a conditionally replicating lentiviral vector
    • Levine BL, Humeau LM, Boyer J, et al. Gene transfer in humans using a conditionally replicating lentiviral vector. Proc Natl Acad Sci U S A 2006; 103:17372-17377.
    • (2006) Proc Natl Acad Sci U S A , vol.103 , pp. 17372-17377
    • Levine, B.L.1    Humeau, L.M.2    Boyer, J.3
  • 113
    • 0033168686 scopus 로고    scopus 로고
    • +) cells from the bone marrow of human immunodeficiency virus-1-infected children
    • +) cells from the bone marrow of human immunodeficiency virus-1-infected children. Blood 1999; 94:368-371.
    • (1999) Blood , vol.94 , pp. 368-371
    • Kohn, D.B.1    Bauer, G.2    Rice, C.R.3
  • 114
    • 0029655644 scopus 로고    scopus 로고
    • RNA aptamers selected to bind human immunodeficiency virus type 1 Rev in vitro are Rev responsive in vivo
    • Symensma TL, Giver L, Zapp M, Takle GB, Ellington AD. RNA aptamers selected to bind human immunodeficiency virus type 1 Rev in vitro are Rev responsive in vivo. J Virol 1996; 70:179-187.
    • (1996) J Virol , vol.70 , pp. 179-187
    • Symensma, T.L.1    Giver, L.2    Zapp, M.3    Takle, G.B.4    Ellington, A.D.5
  • 115
    • 31044433795 scopus 로고    scopus 로고
    • The antisense strand of small interfering RNAs directs histone methylation and transcriptional gene silencing in human cells
    • Weinberg MS, Villeneuve LM, Ehsani A, et al. The antisense strand of small interfering RNAs directs histone methylation and transcriptional gene silencing in human cells. RNA 2006; 12:256-262.
    • (2006) RNA , vol.12 , pp. 256-262
    • Weinberg, M.S.1    Villeneuve, L.M.2    Ehsani, A.3
  • 116
    • 42449160202 scopus 로고    scopus 로고
    • Requirements for gene silencing mediated by U1 snRNA binding to a target sequence
    • Abad X, Vera M, Jung SP, et al. Requirements for gene silencing mediated by U1 snRNA binding to a target sequence. Nucleic Acids Res 2008; 36:2338-2352.
    • (2008) Nucleic Acids Res , vol.36 , pp. 2338-2352
    • Abad, X.1    Vera, M.2    Jung, S.P.3
  • 117
    • 34848814237 scopus 로고    scopus 로고
    • Design of extended short hairpin RNAs for HIV-1 inhibition
    • Liu YP, Haasnoot J, Berkhout B. Design of extended short hairpin RNAs for HIV-1 inhibition. Nucleic Acids Res 2007; 35:5683-5693.
    • (2007) Nucleic Acids Res , vol.35 , pp. 5683-5693
    • Liu, Y.P.1    Haasnoot, J.2    Berkhout, B.3
  • 118
    • 37549055424 scopus 로고    scopus 로고
    • The inhibitory efficacy of RNA POL III-expressed long hairpin RNAs targeted to untranslated regions of the HIV-1 5′ long terminal repeat
    • Barichievy S, Saayman S, von Eije KJ, Morris KV, Arbuthnot P, Weinberg MS. The inhibitory efficacy of RNA POL III-expressed long hairpin RNAs targeted to untranslated regions of the HIV-1 5′ long terminal repeat. Oligonucleotides 2007; 17:419-431.
    • (2007) Oligonucleotides , vol.17 , pp. 419-431
    • Barichievy, S.1    Saayman, S.2    von Eije, K.J.3    Morris, K.V.4    Arbuthnot, P.5    Weinberg, M.S.6
  • 119
    • 37549037041 scopus 로고    scopus 로고
    • Expression of long anti-HIV-1 hairpin RNAs for the generation of multiple siRNAs: Advantages and limitations
    • Sano M, Li H, Nakanishi M, Rossi JJ. Expression of long anti-HIV-1 hairpin RNAs for the generation of multiple siRNAs: advantages and limitations. Mol Ther 2008; 16:170-177.
    • (2008) Mol Ther , vol.16 , pp. 170-177
    • Sano, M.1    Li, H.2    Nakanishi, M.3    Rossi, J.J.4
  • 120
  • 121
    • 56349155978 scopus 로고    scopus 로고
    • Engineering and optimization of the miR-106b cluster for ectopic expression of multiplexed anti-HIV RNAs
    • Aagaard LA, Zhang J, von Eije KJ, et al. Engineering and optimization of the miR-106b cluster for ectopic expression of multiplexed anti-HIV RNAs. Gene Ther 2008; 15:1536-1549.
    • (2008) Gene Ther , vol.15 , pp. 1536-1549
    • Aagaard, L.A.1    Zhang, J.2    von Eije, K.J.3
  • 122
  • 123
    • 50149084664 scopus 로고    scopus 로고
    • Vector design for liver-specific expression of multiple interfering RNAs that target hepatitis B virus transcripts
    • Snyder LL, Esser JM, Pachuk CJ, Steel LF. Vector design for liver-specific expression of multiple interfering RNAs that target hepatitis B virus transcripts. Antiviral Res 2008; 80:36-44.
    • (2008) Antiviral Res , vol.80 , pp. 36-44
    • Snyder, L.L.1    Esser, J.M.2    Pachuk, C.J.3    Steel, L.F.4
  • 124
    • 0043210428 scopus 로고    scopus 로고
    • Conditional suppression of cellular genes: Lentivirus vector-mediated drug-inducible RNA interference
    • Wiznerowicz M, Trono D. Conditional suppression of cellular genes: lentivirus vector-mediated drug-inducible RNA interference. J Virol 2003; 77:8957-8961.
    • (2003) J Virol , vol.77 , pp. 8957-8961
    • Wiznerowicz, M.1    Trono, D.2
  • 125
    • 33847181641 scopus 로고    scopus 로고
    • Improved single-chain transactivators of the Tet-On gene expression system
    • Zhou X, Symons J, Hoppes R, et al. Improved single-chain transactivators of the Tet-On gene expression system. BMC Biotechnol 2007; 7:6.
    • (2007) BMC Biotechnol , vol.7 , pp. 6
    • Zhou, X.1    Symons, J.2    Hoppes, R.3
  • 126
    • 34047168915 scopus 로고    scopus 로고
    • Current concepts in AIDS pathogenesis: Insights from the SIV/macaque model
    • Lackner AA, Veazey RS. Current concepts in AIDS pathogenesis: insights from the SIV/macaque model. Annu Rev Med 2007; 58:461-476.
    • (2007) Annu Rev Med , vol.58 , pp. 461-476
    • Lackner, A.A.1    Veazey, R.S.2
  • 127
    • 1542288934 scopus 로고    scopus 로고
    • The cytoplasmic body component TRIM5alpha restricts HIV-1 infection in Old World monkeys
    • Stremlau M, Owens CM, Perron MJ, Kiessling M, Autissier P, Sodroski J. The cytoplasmic body component TRIM5alpha restricts HIV-1 infection in Old World monkeys. Nature 2004; 427:848-853.
    • (2004) Nature , vol.427 , pp. 848-853
    • Stremlau, M.1    Owens, C.M.2    Perron, M.J.3    Kiessling, M.4    Autissier, P.5    Sodroski, J.6
  • 129
    • 1842588698 scopus 로고    scopus 로고
    • Development of a human adaptive immune system in cord blood cell-transplanted mice
    • Traggiai E, Chicha L, Mazzucchelli L, et al. Development of a human adaptive immune system in cord blood cell-transplanted mice. Science 2004; 304:104-107.
    • (2004) Science , vol.304 , pp. 104-107
    • Traggiai, E.1    Chicha, L.2    Mazzucchelli, L.3
  • 130
    • 10244237695 scopus 로고    scopus 로고
    • + cells injected into newborn RAG2-/-γc-/- mice: Functional inactivation of p53 in developing T cells
    • + cells injected into newborn RAG2-/-γc-/- mice: functional inactivation of p53 in developing T cells. Blood 2004; 104:3886-3893.
    • (2004) Blood , vol.104 , pp. 3886-3893
    • Gimeno, R.1    Weijer, K.2    Voordouw, A.3
  • 131
    • 32044454701 scopus 로고    scopus 로고
    • Experimental models to study development and function of the human immune system in vivo
    • Legrand N, Weijer K, Spits H. Experimental models to study development and function of the human immune system in vivo. J Immunol 2006; 176:2053-2058.
    • (2006) J Immunol , vol.176 , pp. 2053-2058
    • Legrand, N.1    Weijer, K.2    Spits, H.3
  • 132
    • 33846630040 scopus 로고    scopus 로고
    • Humanized mice in translational biomedical research
    • Shultz LD, Ishikawa F, Greiner DL. Humanized mice in translational biomedical research. Nat Rev Immunol 2007; 7:118-130.
    • (2007) Nat Rev Immunol , vol.7 , pp. 118-130
    • Shultz, L.D.1    Ishikawa, F.2    Greiner, D.L.3
  • 133
    • 34248547056 scopus 로고    scopus 로고
    • Human-hemato-lymphoid-system mice: Opportunities and challenges
    • Manz MG. Human-hemato-lymphoid-system mice: opportunities and challenges. Immunity 2007; 26:537-541.
    • (2007) Immunity , vol.26 , pp. 537-541
    • Manz, M.G.1
  • 134
    • 33745604934 scopus 로고    scopus 로고
    • Transient accumulation of human mature thymocytes and regulatory T cells with CD28 superagonist in "human immune system" Rag2(-/-)γc(-/-) mice
    • Legrand N, Cupedo T, van Lent AU, et al. Transient accumulation of human mature thymocytes and regulatory T cells with CD28 superagonist in "human immune system" Rag2(-/-)γc(-/-) mice. Blood 2006; 108:238-245.
    • (2006) Blood , vol.108 , pp. 238-245
    • Legrand, N.1    Cupedo, T.2    van Lent, A.U.3
  • 136
    • 33947596455 scopus 로고    scopus 로고
    • HIV-1 infection and pathogenesis in a novel humanized mouse model
    • Zhang L, Kovalev GI, Su L. HIV-1 infection and pathogenesis in a novel humanized mouse model. Blood 2007; 109:2978-2981.
    • (2007) Blood , vol.109 , pp. 2978-2981
    • Zhang, L.1    Kovalev, G.I.2    Su, L.3
  • 137
    • 33846104485 scopus 로고    scopus 로고
    • null mice develop human lymphoid systems and induce long-lasting HIV-1 infection with specific humoral immune responses
    • null mice develop human lymphoid systems and induce long-lasting HIV-1 infection with specific humoral immune responses. Blood 2007; 109:212-218.
    • (2007) Blood , vol.109 , pp. 212-218
    • Watanabe, S.1    Terashima, K.2    Ohta, S.3
  • 138
    • 33751003823 scopus 로고    scopus 로고
    • HIV-1 infection and CD4 T cell depletion in the humanized Rag2-/-γc-/- (RAG-hu) mouse model
    • Berges BK, Wheat WH, Palmer BE, Connick E, Akkina R. HIV-1 infection and CD4 T cell depletion in the humanized Rag2-/-γc-/- (RAG-hu) mouse model. Retrovirology 2006; 3:76.
    • (2006) Retrovirology , vol.3 , pp. 76
    • Berges, B.K.1    Wheat, W.H.2    Palmer, B.E.3    Connick, E.4    Akkina, R.5
  • 139
    • 34248335883 scopus 로고    scopus 로고
    • Use of a novel chimeric mouse model with a functionally active human immune system to study human immunodeficiency virus type 1 infection
    • An DS, Poon B, Ho Tsong FR, et al. Use of a novel chimeric mouse model with a functionally active human immune system to study human immunodeficiency virus type 1 infection. Clin Vaccine Immunol 2007; 14:391-396.
    • (2007) Clin Vaccine Immunol , vol.14 , pp. 391-396
    • An, D.S.1    Poon, B.2    Ho Tsong, F.R.3
  • 140
    • 40849104785 scopus 로고    scopus 로고
    • Mucosal transmission of R5 and X4 tropic HIV-1 via vaginal and rectal routes in humanized Rag2(-/-)γc(-/-) (RAG-hu) mice
    • Berges BK, Akkina SR, Folkvord JM, Connick E, Akkina R. Mucosal transmission of R5 and X4 tropic HIV-1 via vaginal and rectal routes in humanized Rag2(-/-)γc(-/-) (RAG-hu) mice. Virology 2008; 373:342-351.
    • (2008) Virology , vol.373 , pp. 342-351
    • Berges, B.K.1    Akkina, S.R.2    Folkvord, J.M.3    Connick, E.4    Akkina, R.5
  • 141
    • 41549135641 scopus 로고    scopus 로고
    • Summary of presentations at the NIH/NIAID New Humanized Rodent Models 2007 Workshop
    • Goldstein H. Summary of presentations at the NIH/NIAID New Humanized Rodent Models 2007 Workshop. AIDS Res Ther 2008; 5:3.
    • (2008) AIDS Res Ther , vol.5 , pp. 3
    • Goldstein, H.1
  • 142
    • 0025175787 scopus 로고
    • Gene therapy begins
    • Culliton BJ. Gene therapy begins. Science 1990; 249:1372.
    • (1990) Science , vol.249 , pp. 1372
    • Culliton, B.J.1
  • 143
    • 0033579358 scopus 로고    scopus 로고
    • Gene therapy death prompts review of adenovirus vector
    • Marshall E. Gene therapy death prompts review of adenovirus vector. Science 1999; 286:2244-2245.
    • (1999) Science , vol.286 , pp. 2244-2245
    • Marshall, E.1
  • 144
    • 0037129435 scopus 로고    scopus 로고
    • Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy
    • Hacein-Bey-Abina S, Le Deist F, Carlier F, et al. Sustained correction of X-linked severe combined immunodeficiency by ex vivo gene therapy. N Engl J Med 2002; 346:1185-1193.
    • (2002) N Engl J Med , vol.346 , pp. 1185-1193
    • Hacein-Bey-Abina, S.1    Le Deist, F.2    Carlier, F.3
  • 145
    • 0142084745 scopus 로고    scopus 로고
    • LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1
    • Hacein-Bey-Abina S, Von Kalle C, Schmidt M, et al. LMO2-associated clonal T cell proliferation in two patients after gene therapy for SCID-X1. Science 2003; 302:415-419.
    • (2003) Science , vol.302 , pp. 415-419
    • Hacein-Bey-Abina, S.1    Von Kalle, C.2    Schmidt, M.3
  • 146
    • 35148817461 scopus 로고    scopus 로고
    • Gene therapy clinical trials worldwide to 2007 - an update
    • Edelstein ML, Abedi MR, Wixon J. Gene therapy clinical trials worldwide to 2007 - an update. J Gene Med 2007; 9:833-842.
    • (2007) J Gene Med , vol.9 , pp. 833-842
    • Edelstein, M.L.1    Abedi, M.R.2    Wixon, J.3
  • 148
    • 77949734759 scopus 로고    scopus 로고
    • Hematopoietic stem cell gene therapy trial with lentiviral vector in X-linked adrenoleukodystrophy
    • Cartier-Lacave N. Hematopoietic stem cell gene therapy trial with lentiviral vector in X-linked adrenoleukodystrophy. Hum Gene Ther 2008; 19:1061
    • (2008) Hum Gene Ther , vol.19 , pp. 1061
    • Cartier-Lacave, N.1
  • 149
    • 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, Cesana D, Schmidt M, et al. Hematopoietic stem cell gene transfer in a tumor-prone mouse model uncovers low genotoxicity of lentiviral vector integration. Nat Biotechnol 2006; 24:687-696.
    • (2006) Nat Biotechnol , vol.24 , pp. 687-696
    • Montini, E.1    Cesana, D.2    Schmidt, M.3
  • 150
    • 28144462457 scopus 로고    scopus 로고
    • Genome-wide analysis of retroviral DNA integration
    • Bushman F, Lewinski M, Ciuffi A, et al. Genome-wide analysis of retroviral DNA integration. Nat Rev Microbiol 2005; 3:848-858.
    • (2005) Nat Rev Microbiol , vol.3 , pp. 848-858
    • Bushman, F.1    Lewinski, M.2    Ciuffi, A.3
  • 151
    • 20844455902 scopus 로고    scopus 로고
    • Regulatory considerations for novel gene therapy products: A review of the process leading to the first clinical lentiviral vector
    • Manilla P, Rebello T, Afable C, et al. Regulatory considerations for novel gene therapy products: a review of the process leading to the first clinical lentiviral vector. Hum Gene Ther 2005; 16:17-25.
    • (2005) Hum Gene Ther , vol.16 , pp. 17-25
    • Manilla, P.1    Rebello, T.2    Afable, C.3
  • 152
    • 54949095836 scopus 로고    scopus 로고
    • Misinterpreting the therapeutic effects of small interfering RNA caused by immune stimulation
    • Robbins M, Judge A, Ambegia E, et al. Misinterpreting the therapeutic effects of small interfering RNA caused by immune stimulation. Hum Gene Ther 2008; 19:991-999.
    • (2008) Hum Gene Ther , vol.19 , pp. 991-999
    • Robbins, M.1    Judge, A.2    Ambegia, E.3
  • 153
    • 41649115210 scopus 로고    scopus 로고
    • Sequence- and target-independent angiogenesis suppression by siRNA via TLR3
    • Kleinman ME, Yamada K, Takeda A, et al. Sequence- and target-independent angiogenesis suppression by siRNA via TLR3. Nature 2008; 452:591-597.
    • (2008) Nature , vol.452 , pp. 591-597
    • Kleinman, M.E.1    Yamada, K.2    Takeda, A.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.