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Volumn 112, Issue 30, 2015, Pages E4007-E4016

Alleviation of off-target effects from vector-encoded shRNAs via codelivered RNA decoys

Author keywords

AAV; Adeno associated viral vector; Off targeting; RNA interference; Short hairpin RNA

Indexed keywords

MICRORNA; RNA; SHORT HAIRPIN RNA; 3' UNTRANSLATED REGION; DNA; GREEN FLUORESCENT PROTEIN; MIRN122 MICRORNA, HUMAN; OLIGONUCLEOTIDE; SMALL INTERFERING RNA;

EID: 84938150485     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1510476112     Document Type: Article
Times cited : (36)

References (72)
  • 1
    • 84902096048 scopus 로고    scopus 로고
    • Development and applications of CRISPR-Cas9 for genome engineering
    • Hsu PD, Lander ES, Zhang F (2014) Development and applications of CRISPR-Cas9 for genome engineering. Cell 157(6):1262-1278.
    • (2014) Cell , vol.157 , Issue.6 , pp. 1262-1278
    • Hsu, P.D.1    Lander, E.S.2    Zhang, F.3
  • 2
    • 58749104364 scopus 로고    scopus 로고
    • The promises and pitfalls of RNA-interference-based therapeutics
    • Castanotto D, Rossi JJ (2009) The promises and pitfalls of RNA-interference-based therapeutics. Nature 457(7228):426-433.
    • (2009) Nature , vol.457 , Issue.7228 , pp. 426-433
    • Castanotto, D.1    Rossi, J.J.2
  • 3
    • 50549092566 scopus 로고    scopus 로고
    • Expression of shRNA from a tissuespecific pol II promoter is an effective and safe RNAi therapeutic
    • Giering JC, Grimm D, Storm TA, Kay MA (2008) Expression of shRNA from a tissuespecific pol II promoter is an effective and safe RNAi therapeutic. Mol Ther 16(9):1630-1636.
    • (2008) Mol Ther , vol.16 , Issue.9 , pp. 1630-1636
    • Giering, J.C.1    Grimm, D.2    Storm, T.A.3    Kay, M.A.4
  • 4
    • 33745299723 scopus 로고    scopus 로고
    • Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
    • Grimm D, et al. (2006) Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways. Nature 441(7092):537-541.
    • (2006) Nature , vol.441 , Issue.7092 , pp. 537-541
    • Grimm, D.1
  • 5
    • 74049124186 scopus 로고    scopus 로고
    • Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application
    • Jackson AL, Linsley PS (2010) Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application. Nat Rev Drug Discov 9(1):57-67.
    • (2010) Nat Rev Drug Discov , vol.9 , Issue.1 , pp. 57-67
    • Jackson, A.L.1    Linsley, P.S.2
  • 6
    • 0037685280 scopus 로고    scopus 로고
    • Expression profiling reveals off-target gene regulation by RNAi
    • Jackson AL, et al. (2003) Expression profiling reveals off-target gene regulation by RNAi. Nat Biotechnol 21(6):635-637.
    • (2003) Nat Biotechnol , vol.21 , Issue.6 , pp. 635-637
    • Jackson, A.L.1
  • 7
    • 33745612464 scopus 로고    scopus 로고
    • Widespread siRNA "off-target" transcript silencing mediated by seed region sequence complementarity
    • Jackson AL, et al. (2006) Widespread siRNA "off-target" transcript silencing mediated by seed region sequence complementarity. RNA 12(7):1179-1187.
    • (2006) RNA , vol.12 , Issue.7 , pp. 1179-1187
    • Jackson, A.L.1
  • 8
    • 73349086270 scopus 로고    scopus 로고
    • Off-target and a portion of target-specific siRNA mediated mRNA degradation is Ago2 'Slicer' independent and can be mediated by Ago1
    • Vickers TA, et al. (2009) Off-target and a portion of target-specific siRNA mediated mRNA degradation is Ago2 'Slicer' independent and can be mediated by Ago1. Nucleic Acids Res 37(20):6927-6941.
    • (2009) Nucleic Acids Res , vol.37 , Issue.20 , pp. 6927-6941
    • Vickers, T.A.1
  • 9
    • 84857781943 scopus 로고    scopus 로고
    • A genomewide homologous recombination screen identifies the RNA-binding protein RBMX as a component of the DNA-damage response
    • Adamson B, Smogorzewska A, Sigoillot FD, King RW, Elledge SJ (2012) A genomewide homologous recombination screen identifies the RNA-binding protein RBMX as a component of the DNA-damage response. Nat Cell Biol 14(3):318-328.
    • (2012) Nat Cell Biol , vol.14 , Issue.3 , pp. 318-328
    • Adamson, B.1    Smogorzewska, A.2    Sigoillot, F.D.3    King, R.W.4    Elledge, S.J.5
  • 10
    • 80052890070 scopus 로고    scopus 로고
    • Off-target effects dominate a large-scale RNAi screen for modulators of the TGF-p pathway and reveal microRNA regulation of TGFBR2
    • Schultz N, et al. (2011) Off-target effects dominate a large-scale RNAi screen for modulators of the TGF-p pathway and reveal microRNA regulation of TGFBR2. Silence 2:3.
    • (2011) Silence , vol.2 , pp. 3
    • Schultz, N.1
  • 12
    • 79959366354 scopus 로고    scopus 로고
    • Thermodynamic stability of small hairpin RNAs highly influences the loading process of different mammalian Argonautes
    • Gu S, et al. (2011) Thermodynamic stability of small hairpin RNAs highly influences the loading process of different mammalian Argonautes. Proc Natl Acad Sci USA 108(22):9208-9213.
    • (2011) Proc Natl Acad Sci USA , vol.108 , Issue.22 , pp. 9208-9213
    • Gu, S.1
  • 13
    • 84856723932 scopus 로고    scopus 로고
    • The N domain of Argonaute drives duplex unwinding during RISC assembly
    • Kwak PB, Tomari Y (2012) The N domain of Argonaute drives duplex unwinding during RISC assembly. Nat Struct Mol Biol 19(2):145-151.
    • (2012) Nat Struct Mol Biol , vol.19 , Issue.2 , pp. 145-151
    • Kwak, P.B.1    Tomari, Y.2
  • 14
    • 81255176919 scopus 로고    scopus 로고
    • Argonaute proteins regulate microRNA stability: Increased microRNA abundance by Argonaute proteins is due to microRNA stabilization
    • Winter J, Diederichs S (2011) Argonaute proteins regulate microRNA stability: Increased microRNA abundance by Argonaute proteins is due to microRNA stabilization. RNA Biol 8(6):1149-1157.
    • (2011) RNA Biol , vol.8 , Issue.6 , pp. 1149-1157
    • Winter, J.1    Diederichs, S.2
  • 15
    • 84886037550 scopus 로고    scopus 로고
    • Argonaute-3 activates the let-7a passenger strand microRNA
    • Winter J, Diederichs S (2013) Argonaute-3 activates the let-7a passenger strand microRNA. RNA Biol 10(10):1631-1643.
    • (2013) RNA Biol , vol.10 , Issue.10 , pp. 1631-1643
    • Winter, J.1    Diederichs, S.2
  • 16
    • 41649089309 scopus 로고    scopus 로고
    • ∗ species has substantial influence on microRNA and 3' UTR evolution
    • ∗ species has substantial influence on microRNA and 3' UTR evolution. Nat Struct Mol Biol 15(4):354-363.
    • (2008) Nat Struct Mol Biol , vol.15 , Issue.4 , pp. 354-363
    • Okamura, K.1
  • 17
    • 0142165224 scopus 로고    scopus 로고
    • Functional siRNAs and miRNAs exhibit strand bias
    • Khvorova A, Reynolds A, Jayasena SD (2003) Functional siRNAs and miRNAs exhibit strand bias. Cell 115(2):209-216.
    • (2003) Cell , vol.115 , Issue.2 , pp. 209-216
    • Khvorova, A.1    Reynolds, A.2    Jayasena, S.D.3
  • 18
    • 10744225153 scopus 로고    scopus 로고
    • Asymmetry in the assembly of the RNAi enzyme complex
    • Schwarz DS, et al. (2003) Asymmetry in the assembly of the RNAi enzyme complex. Cell 115(2):199-208.
    • (2003) Cell , vol.115 , Issue.2 , pp. 199-208
    • Schwarz, D.S.1
  • 19
    • 14644393684 scopus 로고    scopus 로고
    • Perspective: Machines for RNAi
    • Tomari Y, Zamore PD (2005) Perspective: Machines for RNAi. Genes Dev 19(5):517-529.
    • (2005) Genes Dev , vol.19 , Issue.5 , pp. 517-529
    • Tomari, Y.1    Zamore, P.D.2
  • 20
    • 79952697845 scopus 로고    scopus 로고
    • Functional identification of optimized RNAi triggers using a massively parallel sensor assay
    • Fellmann C, et al. (2011) Functional identification of optimized RNAi triggers using a massively parallel sensor assay. Mol Cell 41(6):733-746.
    • (2011) Mol Cell , vol.41 , Issue.6 , pp. 733-746
    • Fellmann, C.1
  • 21
    • 84055222215 scopus 로고    scopus 로고
    • Dicer is dispensable for asymmetric RISC loading in mammals
    • Betancur JG, Tomari Y (2012) Dicer is dispensable for asymmetric RISC loading in mammals. RNA 18(1):24-30.
    • (2012) RNA , vol.18 , Issue.1 , pp. 24-30
    • Betancur, J.G.1    Tomari, Y.2
  • 22
    • 84876591872 scopus 로고    scopus 로고
    • Multiple sensors ensure guide strand selection in human RNAi pathways
    • Noland CL, Doudna JA (2013) Multiple sensors ensure guide strand selection in human RNAi pathways. RNA 19(5):639-648.
    • (2013) RNA , vol.19 , Issue.5 , pp. 639-648
    • Noland, C.L.1    Doudna, J.A.2
  • 23
    • 79959872313 scopus 로고    scopus 로고
    • SiRNA repositioning for guide strand selection by human Dicer complexes
    • Noland CL, Ma E, Doudna JA (2011) siRNA repositioning for guide strand selection by human Dicer complexes. Mol Cell 43(1):110-121.
    • (2011) Mol Cell , vol.43 , Issue.1 , pp. 110-121
    • Noland, C.L.1    Ma, E.2    Doudna, J.A.3
  • 24
    • 67749129050 scopus 로고    scopus 로고
    • Asymmetry in siRNA design
    • Grimm D (2009) Asymmetry in siRNA design. Gene Ther 16(7):827-829.
    • (2009) Gene Ther , vol.16 , Issue.7 , pp. 827-829
    • Grimm, D.1
  • 25
    • 84869037267 scopus 로고    scopus 로고
    • The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo
    • Gu S, et al. (2012) The loop position of shRNAs and pre-miRNAs is critical for the accuracy of dicer processing in vivo. Cell 151(4):900-911.
    • (2012) Cell , vol.151 , Issue.4 , pp. 900-911
    • Gu, S.1
  • 26
    • 82955233704 scopus 로고    scopus 로고
    • Rational design of therapeutic siRNAs: Minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease
    • Boudreau RL, Spengler RM, Davidson BL (2011) Rational design of therapeutic siRNAs: Minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease. Mol Ther 19(12):2169-2177.
    • (2011) Mol Ther , vol.19 , Issue.12 , pp. 2169-2177
    • Boudreau, R.L.1    Spengler, R.M.2    Davidson, B.L.3
  • 27
    • 72049087544 scopus 로고    scopus 로고
    • Asymmetrically designed siRNAs and shRNAs enhance the strand specificity and efficacy in RNAi
    • Ding H, Liao G, Wang H, Zhou Y (2007) Asymmetrically designed siRNAs and shRNAs enhance the strand specificity and efficacy in RNAi. J RNAi Gene Silencing 4(1):269-280.
    • (2007) J RNAi Gene Silencing , vol.4 , Issue.1 , pp. 269-280
    • Ding, H.1    Liao, G.2    Wang, H.3    Zhou, Y.4
  • 28
    • 79961102375 scopus 로고    scopus 로고
    • The effects of stem length and core placement on shRNA activity
    • McIntyre GJ, Yu YH, Lomas M, Fanning GC (2011) The effects of stem length and core placement on shRNA activity. BMC Mol Biol 12:34.
    • (2011) BMC Mol Biol , vol.12 , pp. 34
    • McIntyre, G.J.1    Yu, Y.H.2    Lomas, M.3    Fanning, G.C.4
  • 29
    • 84890012407 scopus 로고    scopus 로고
    • Robust RNAi enhancement via human Argonaute-2 overexpression from plasmids, viral vectors and cell lines
    • Börner K, et al. (2013) Robust RNAi enhancement via human Argonaute-2 overexpression from plasmids, viral vectors and cell lines. Nucleic Acids Res 41(21):e199.
    • (2013) Nucleic Acids Res , vol.41 , Issue.21 , pp. e199
    • Börner, K.1
  • 30
    • 84876030403 scopus 로고    scopus 로고
    • Dicer-independent processing of short hairpin RNAs
    • Liu YP, Schopman NC, Berkhout B (2013) Dicer-independent processing of short hairpin RNAs. Nucleic Acids Res 41(6):3723-3733.
    • (2013) Nucleic Acids Res , vol.41 , Issue.6 , pp. 3723-3733
    • Liu, Y.P.1    Schopman, N.C.2    Berkhout, B.3
  • 31
    • 77956995245 scopus 로고    scopus 로고
    • Conserved vertebrate mir-451 provides a platform for Dicerindependent, Ago2-mediated microRNA biogenesis
    • Yang JS, et al. (2010) Conserved vertebrate mir-451 provides a platform for Dicerindependent, Ago2-mediated microRNA biogenesis. Proc Natl Acad Sci USA 107(34):15163-15168.
    • (2010) Proc Natl Acad Sci USA , vol.107 , Issue.34 , pp. 15163-15168
    • Yang, J.S.1
  • 32
    • 84859969412 scopus 로고    scopus 로고
    • Functional parameters of Dicer-independent microRNA biogenesis
    • Yang JS, Maurin T, Lai EC (2012) Functional parameters of Dicer-independent microRNA biogenesis. RNA 18(5):945-957.
    • (2012) RNA , vol.18 , Issue.5 , pp. 945-957
    • Yang, J.S.1    Maurin, T.2    Lai, E.C.3
  • 33
    • 77953897182 scopus 로고    scopus 로고
    • A novel miRNA processing pathway independent of Dicer requires Argonaute2 catalytic activity
    • Cifuentes D, et al. (2010) A novel miRNA processing pathway independent of Dicer requires Argonaute2 catalytic activity. Science 328(5986):1694-1698.
    • (2010) Science , vol.328 , Issue.5986 , pp. 1694-1698
    • Cifuentes, D.1
  • 34
    • 36849048778 scopus 로고    scopus 로고
    • Dual role for argonautes in microRNA processing and posttranscriptional regulation of microRNA expression
    • Diederichs S, Haber DA (2007) Dual role for argonautes in microRNA processing and posttranscriptional regulation of microRNA expression. Cell 131(6):1097-1108.
    • (2007) Cell , vol.131 , Issue.6 , pp. 1097-1108
    • Diederichs, S.1    Haber, D.A.2
  • 35
    • 84860370660 scopus 로고    scopus 로고
    • A potent 2'-O-methylated RNA-based microRNA inhibitor with unique secondary structures
    • Haraguchi T, et al. (2012) A potent 2'-O-methylated RNA-based microRNA inhibitor with unique secondary structures. Nucleic Acids Res 40(8):e58.
    • (2012) Nucleic Acids Res , vol.40 , Issue.8 , pp. e58
    • Haraguchi, T.1
  • 36
    • 64549109671 scopus 로고    scopus 로고
    • Vectors expressing efficient RNA decoys achieve the long-term suppression of specific microRNA activity in mammalian cells
    • Haraguchi T, Ozaki Y, Iba H (2009) Vectors expressing efficient RNA decoys achieve the long-term suppression of specific microRNA activity in mammalian cells. Nucleic Acids Res 37(6):e43.
    • (2009) Nucleic Acids Res , vol.37 , Issue.6 , pp. e43
    • Haraguchi, T.1    Ozaki, Y.2    Iba, H.3
  • 37
    • 1542369977 scopus 로고    scopus 로고
    • From virus evolution to vector revolution: Use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy
    • Grimm D, Kay MA (2003) From virus evolution to vector revolution: Use of naturally occurring serotypes of adeno-associated virus (AAV) as novel vectors for human gene therapy. Curr Gene Ther 3(4):281-304.
    • (2003) Curr Gene Ther , vol.3 , Issue.4 , pp. 281-304
    • Grimm, D.1    Kay, M.A.2
  • 38
    • 12144289381 scopus 로고    scopus 로고
    • Alternative approaches for efficient inhibition of hepatitis C virus RNA replication by small interfering RNAs
    • Krönke J, et al. (2004) Alternative approaches for efficient inhibition of hepatitis C virus RNA replication by small interfering RNAs. J Virol 78(7):3436-3446.
    • (2004) J Virol , vol.78 , Issue.7 , pp. 3436-3446
    • Krönke, J.1
  • 39
    • 84890928825 scopus 로고    scopus 로고
    • Prospects for nucleic acid-based therapeutics against hepatitis C virus
    • Lee CH, Kim JH, Lee SW (2013) Prospects for nucleic acid-based therapeutics against hepatitis C virus. World J Gastroenterol 19(47):8949-8962.
    • (2013) World J Gastroenterol , vol.19 , Issue.47 , pp. 8949-8962
    • Lee, C.H.1    Kim, J.H.2    Lee, S.W.3
  • 40
    • 84890590684 scopus 로고    scopus 로고
    • Formulated minimal-length synthetic small hairpin RNAs are potent inhibitors of hepatitis C virus in mice with humanized livers
    • Ma H, et al. (2014) Formulated minimal-length synthetic small hairpin RNAs are potent inhibitors of hepatitis C virus in mice with humanized livers. Gastroenterology 146(1):63-66.
    • (2014) Gastroenterology , vol.146 , Issue.1 , pp. 63-66
    • Ma, H.1
  • 41
    • 84890963077 scopus 로고    scopus 로고
    • Adenovirus vectors lacking virus-associated RNA expression enhance shRNA activity to suppress hepatitis C virus replication
    • Pei Z, et al. (2013) Adenovirus vectors lacking virus-associated RNA expression enhance shRNA activity to suppress hepatitis C virus replication. Sci Rep 3:3575.
    • (2013) Sci Rep , vol.3 , pp. 3575
    • Pei, Z.1
  • 42
    • 10744230911 scopus 로고    scopus 로고
    • Inhibition of intracellular hepatitis C virus replication by synthetic and vector-derived small interfering RNAs
    • Yokota T, et al. (2003) Inhibition of intracellular hepatitis C virus replication by synthetic and vector-derived small interfering RNAs. EMBO Rep 4(6):602-608.
    • (2003) EMBO Rep , vol.4 , Issue.6 , pp. 602-608
    • Yokota, T.1
  • 43
    • 44949131860 scopus 로고    scopus 로고
    • In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses
    • Grimm D, et al. (2008) In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses. J Virol 82(12):5887-5911.
    • (2008) J Virol , vol.82 , Issue.12 , pp. 5887-5911
    • Grimm, D.1
  • 44
    • 84862805889 scopus 로고    scopus 로고
    • Long-term, efficient inhibition of microRNA function in mice using rAAV vectors
    • Xie J, et al. (2012) Long-term, efficient inhibition of microRNA function in mice using rAAV vectors. Nat Methods 9(4):403-409.
    • (2012) Nat Methods , vol.9 , Issue.4 , pp. 403-409
    • Xie, J.1
  • 45
    • 24644483623 scopus 로고    scopus 로고
    • Modulation of hepatitis C virus RNA abundance by a liver-specific MicroRNA
    • Jopling CL, Yi M, Lancaster AM, Lemon SM, Sarnow P (2005) Modulation of hepatitis C virus RNA abundance by a liver-specific MicroRNA. Science 309(5740):1577-1581.
    • (2005) Science , vol.309 , Issue.5740 , pp. 1577-1581
    • Jopling, C.L.1    Yi, M.2    Lancaster, A.M.3    Lemon, S.M.4    Sarnow, P.5
  • 46
    • 74249112787 scopus 로고    scopus 로고
    • Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection
    • Lanford RE, et al. (2010) Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection. Science 327(5962):198-201.
    • (2010) Science , vol.327 , Issue.5962 , pp. 198-201
    • Lanford, R.E.1
  • 47
    • 84877258007 scopus 로고    scopus 로고
    • Treatment of HCV infection by targeting microRNA
    • Janssen HL, et al. (2013) Treatment of HCV infection by targeting microRNA. N Engl J Med 368(18):1685-1694.
    • (2013) N Engl J Med , vol.368 , Issue.18 , pp. 1685-1694
    • Janssen, H.L.1
  • 48
    • 84920116302 scopus 로고    scopus 로고
    • In vitro antiviral activity and preclinical and clinical resistance profile of miravirsen, a novel anti-hepatitis C virus therapeutic targeting the human factor miR-122
    • Ottosen S, et al. (2015) In vitro antiviral activity and preclinical and clinical resistance profile of miravirsen, a novel anti-hepatitis C virus therapeutic targeting the human factor miR-122. Antimicrob Agents Chemother 59(1):599-608.
    • (2015) Antimicrob Agents Chemother , vol.59 , Issue.1 , pp. 599-608
    • Ottosen, S.1
  • 49
    • 84922244588 scopus 로고    scopus 로고
    • MIR-122-A key factor and therapeutic target in liver disease
    • Bandiera S, Pfeffer S, Baumert TF, Zeisel MB (2015) miR-122-a key factor and therapeutic target in liver disease. J Hepatol 62(2):448-457.
    • (2015) J Hepatol , vol.62 , Issue.2 , pp. 448-457
    • Bandiera, S.1    Pfeffer, S.2    Baumert, T.F.3    Zeisel, M.B.4
  • 50
    • 84855212109 scopus 로고    scopus 로고
    • New targets for antiviral therapy of chronic hepatitis C
    • Bühler S, Bartenschlager R (2012) New targets for antiviral therapy of chronic hepatitis C. Liver Int 32(Suppl 1):9-16.
    • (2012) Liver Int , vol.32 , pp. 9-16
    • Bühler, S.1    Bartenschlager, R.2
  • 51
    • 84919683883 scopus 로고    scopus 로고
    • Pegylated interferon based therapy with second-wave direct-acting antivirals in genotype 1 chronic hepatitis C
    • Welch NM, Jensen DM (2015) Pegylated interferon based therapy with second-wave direct-acting antivirals in genotype 1 chronic hepatitis C. Liver Int 35(Suppl 1):11-17.
    • (2015) Liver Int , vol.35 , pp. 11-17
    • Welch, N.M.1    Jensen, D.M.2
  • 52
    • 0032545933 scopus 로고    scopus 로고
    • Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
    • Fire A, et al. (1998) Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature 391(6669):806-811.
    • (1998) Nature , vol.391 , Issue.6669 , pp. 806-811
    • Fire, A.1
  • 53
    • 58149232358 scopus 로고    scopus 로고
    • Artificial microRNAs as siRNA shuttles: Improved safety as compared to shRNAs in vitro and in vivo
    • Boudreau RL, Martins I, Davidson BL (2009) Artificial microRNAs as siRNA shuttles: Improved safety as compared to shRNAs in vitro and in vivo. Mol Ther 17(1):169-175.
    • (2009) Mol Ther , vol.17 , Issue.1 , pp. 169-175
    • Boudreau, R.L.1    Martins, I.2    Davidson, B.L.3
  • 54
    • 50649121877 scopus 로고    scopus 로고
    • Minimizing variables among hairpinbased RNAi vectors reveals the potency of shRNAs
    • Boudreau RL, Monteys AM, Davidson BL (2008) Minimizing variables among hairpinbased RNAi vectors reveals the potency of shRNAs. RNA 14(9):1834-1844.
    • (2008) RNA , vol.14 , Issue.9 , pp. 1834-1844
    • Boudreau, R.L.1    Monteys, A.M.2    Davidson, B.L.3
  • 55
    • 84872556705 scopus 로고    scopus 로고
    • Potent microRNA suppression by RNA Pol II-transcribed 'Tough Decoy' inhibitors
    • Bak RO, Hollensen AK, Primo MN, Sørensen CD, Mikkelsen JG (2013) Potent microRNA suppression by RNA Pol II-transcribed 'Tough Decoy' inhibitors. RNA 19 (2) 280-293.
    • (2013) RNA , vol.19 , Issue.2 , pp. 280-293
    • Bak, R.O.1    Hollensen, A.K.2    Primo, M.N.3    Sørensen, C.D.4    Mikkelsen, J.G.5
  • 56
    • 84860628872 scopus 로고    scopus 로고
    • Engineering and evolution of synthetic adeno-associated virus (AAV) gene therapy vectors via DNA family shuffling
    • Kienle E, et al. (2012) Engineering and evolution of synthetic adeno-associated virus (AAV) gene therapy vectors via DNA family shuffling. J Vis Exp Apr. 2(62):3819.
    • (2012) J Vis Exp Apr , vol.2 , Issue.62 , pp. 3819
    • Kienle, E.1
  • 57
    • 84867564182 scopus 로고    scopus 로고
    • Right- and left-loop short shRNAs have distinct and unusual mechanisms of gene silencing
    • Dallas A, et al. (2012) Right- and left-loop short shRNAs have distinct and unusual mechanisms of gene silencing. Nucleic Acids Res 40(18):9255-9271.
    • (2012) Nucleic Acids Res , vol.40 , Issue.18 , pp. 9255-9271
    • Dallas, A.1
  • 58
    • 84874965759 scopus 로고    scopus 로고
    • Suppression of microRNAs by dual-targeting and clustered Tough Decoy inhibitors
    • Hollensen AK, Bak RO, Haslund D, Mikkelsen JG (2013) Suppression of microRNAs by dual-targeting and clustered Tough Decoy inhibitors. RNA Biol 10(3):406-414.
    • (2013) RNA Biol , vol.10 , Issue.3 , pp. 406-414
    • Hollensen, A.K.1    Bak, R.O.2    Haslund, D.3    Mikkelsen, J.G.4
  • 59
    • 18744382144 scopus 로고    scopus 로고
    • Hepatitis C virus replicons escape RNA interference induced by a short interfering RNA directed against the NS5b coding region
    • Wilson JA, Richardson CD (2005) Hepatitis C virus replicons escape RNA interference induced by a short interfering RNA directed against the NS5b coding region. J Virol 79(11):7050-7058.
    • (2005) J Virol , vol.79 , Issue.11 , pp. 7050-7058
    • Wilson, J.A.1    Richardson, C.D.2
  • 60
    • 84876055691 scopus 로고    scopus 로고
    • The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA
    • Lisowski L, Elazar M, Chu K, Glenn JS, Kay MA (2013) The anti-genomic (negative) strand of Hepatitis C Virus is not targetable by shRNA. Nucleic Acids Res 41(6):3688-3698.
    • (2013) Nucleic Acids Res , vol.41 , Issue.6 , pp. 3688-3698
    • Lisowski, L.1    Elazar, M.2    Chu, K.3    Glenn, J.S.4    Kay, M.A.5
  • 61
    • 33846966669 scopus 로고    scopus 로고
    • Positional effects and strand preference of RNA interference against hepatitis C virus target sequences
    • Smith RM, Smolic R, Volarevic M, Wu GY (2007) Positional effects and strand preference of RNA interference against hepatitis C virus target sequences. J Viral Hepat 14(3):194-212.
    • (2007) J Viral Hepat , vol.14 , Issue.3 , pp. 194-212
    • Smith, R.M.1    Smolic, R.2    Volarevic, M.3    Wu, G.Y.4
  • 62
    • 67349251607 scopus 로고    scopus 로고
    • Rapid creation and quantitative monitoring of high coverage shRNA libraries
    • Bassik MC, et al. (2009) Rapid creation and quantitative monitoring of high coverage shRNA libraries. Nat Methods 6(6):443-445.
    • (2009) Nat Methods , vol.6 , Issue.6 , pp. 443-445
    • Bassik, M.C.1
  • 63
    • 84874113985 scopus 로고    scopus 로고
    • A systematic mammalian genetic interaction map reveals pathways underlying ricin susceptibility
    • Bassik MC, et al. (2013) A systematic mammalian genetic interaction map reveals pathways underlying ricin susceptibility. Cell 152(4):909-922.
    • (2013) Cell , vol.152 , Issue.4 , pp. 909-922
    • Bassik, M.C.1
  • 64
    • 84879319702 scopus 로고    scopus 로고
    • Integrated platform for genome-wide screening and construction of high-density genetic interaction maps in mammalian cells
    • Kampmann M, Bassik MC, Weissman JS (2013) Integrated platform for genome-wide screening and construction of high-density genetic interaction maps in mammalian cells. Proc Natl Acad Sci USA 110(25):E2317-E2326.
    • (2013) Proc Natl Acad Sci USA , vol.110 , Issue.25 , pp. E2317-E2326
    • Kampmann, M.1    Bassik, M.C.2    Weissman, J.S.3
  • 65
    • 77956355233 scopus 로고    scopus 로고
    • Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver
    • Grimm D, et al. (2010) Argonaute proteins are key determinants of RNAi efficacy, toxicity, and persistence in the adult mouse liver. J Clin Invest 120(9):3106-3119.
    • (2010) J Clin Invest , vol.120 , Issue.9 , pp. 3106-3119
    • Grimm, D.1
  • 66
    • 74149092180 scopus 로고    scopus 로고
    • Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice
    • Beer S, et al. (2010) Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice. Mol Ther 18(1):161-170.
    • (2010) Mol Ther , vol.18 , Issue.1 , pp. 161-170
    • Beer, S.1
  • 67
    • 33845656937 scopus 로고    scopus 로고
    • Long-term inhibition of hepatitis B virus in transgenic mice by doublestranded adeno-associated virus 8-delivered short hairpin RNA
    • Chen CC, et al. (2007) Long-term inhibition of hepatitis B virus in transgenic mice by doublestranded adeno-associated virus 8-delivered short hairpin RNA. Gene Ther 14(1):11-19.
    • (2007) Gene Ther , vol.14 , Issue.1 , pp. 11-19
    • Chen, C.C.1
  • 68
    • 0031875874 scopus 로고    scopus 로고
    • Adeno-associated virus vectors can be efficiently produced without helper virus
    • Matsushita T, et al. (1998) Adeno-associated virus vectors can be efficiently produced without helper virus. Gene Ther 5(7):938-945.
    • (1998) Gene Ther , vol.5 , Issue.7 , pp. 938-945
    • Matsushita, T.1
  • 69
    • 0037015049 scopus 로고    scopus 로고
    • Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy
    • Gao GP, et al. (2002) Novel adeno-associated viruses from rhesus monkeys as vectors for human gene therapy. Proc Natl Acad Sci USA 99(18):11854-11859.
    • (2002) Proc Natl Acad Sci USA , vol.99 , Issue.18 , pp. 11854-11859
    • Gao, G.P.1
  • 70
    • 0036430191 scopus 로고    scopus 로고
    • Production methods for gene transfer vectors based on adenoassociated virus serotypes
    • Grimm D (2002) Production methods for gene transfer vectors based on adenoassociated virus serotypes. Methods 28(2):146-157.
    • (2002) Methods , vol.28 , Issue.2 , pp. 146-157
    • Grimm, D.1
  • 71
    • 61549137192 scopus 로고    scopus 로고
    • HCV replicons: Overview and basic protocols
    • Lohmann V (2009) HCV replicons: Overview and basic protocols. Methods Mol Biol 510:145-163.
    • (2009) Methods Mol Biol , vol.510 , pp. 145-163
    • Lohmann, V.1
  • 72
    • 3242736704 scopus 로고    scopus 로고
    • Human Argonaute2 mediates RNA cleavage targeted by miRNAs and siRNAs
    • Meister G, et al. (2004) Human Argonaute2 mediates RNA cleavage targeted by miRNAs and siRNAs. Mol Cell 15(2):185-197.
    • (2004) Mol Cell , vol.15 , Issue.2 , pp. 185-197
    • Meister, G.1


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