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Volumn 7, Issue 1, 2010, Pages

Use of small RNAs for therapeutic gene silencing: Risk versus benefit

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; COMPLEMENTARY DNA; MICRORNA; SMALL INTERFERING RNA;

EID: 78649907430     PISSN: 17406749     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.ddtec.2010.07.003     Document Type: Review
Times cited : (1)

References (40)
  • 1
    • 0032545933 scopus 로고    scopus 로고
    • Potent and specific genetic interference by double-stranded RNA in caenorhabditis elegans
    • DOI 10.1038/35888
    • A. Fire Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans Nature 391 1998 806 811 (Pubitemid 28099681)
    • (1998) Nature , vol.391 , Issue.6669 , pp. 806-811
    • Fire, A.1    Xu, S.2    Montgomery, M.K.3    Kostas, S.A.4    Driver, S.E.5    Mello, C.C.6
  • 2
    • 58749104364 scopus 로고    scopus 로고
    • The promises and pitfalls of RNA-interference-based therapeutics
    • D. Castanotto The promises and pitfalls of RNA-interference-based therapeutics Nature 457 2009 426 433
    • (2009) Nature , vol.457 , pp. 426-433
    • Castanotto, D.1
  • 3
    • 33847036990 scopus 로고    scopus 로고
    • Strategies for silencing human disease using RNA interference
    • D.H. Kim Strategies for silencing human disease using RNA interference Nat. Rev. Genet. 8 2007 173 184
    • (2007) Nat. Rev. Genet. , vol.8 , pp. 173-184
    • Kim, D.H.1
  • 4
    • 0037685280 scopus 로고    scopus 로고
    • Expression profiling reveals off-target gene regulation by RNAi
    • A.L. Jackson Expression profiling reveals off-target gene regulation by RNAi Nat. Biotechnol. 21 2003 635 637
    • (2003) Nat. Biotechnol. , vol.21 , pp. 635-637
    • Jackson, A.L.1
  • 5
    • 4744373229 scopus 로고    scopus 로고
    • Noise amidst the silence: Off-target effects of siRNAs?
    • A.L. Jackson Noise amidst the silence: off-target effects of siRNAs? Trends Genet. 20 2004 521 524
    • (2004) Trends Genet. , vol.20 , pp. 521-524
    • Jackson, A.L.1
  • 6
    • 41649092475 scopus 로고    scopus 로고
    • RNA interference: Generic block on angiogenesis
    • R. Kalluri RNA interference: generic block on angiogenesis Nature 452 2008 543 545
    • (2008) Nature , vol.452 , pp. 543-545
    • Kalluri, R.1
  • 7
    • 58149232358 scopus 로고    scopus 로고
    • Artificial microRNAs as siRNA shuttles: Improved safety as compared to shRNAs in vitro and in vivo
    • R.L. Boudreau Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo Mol. Ther. 17 2009 169 175
    • (2009) Mol. Ther. , vol.17 , pp. 169-175
    • Boudreau, R.L.1
  • 8
    • 33745299723 scopus 로고    scopus 로고
    • Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways
    • D. Grimm Fatality in mice due to oversaturation of cellular microRNA/short hairpin RNA pathways Nature 441 2006 537 541
    • (2006) Nature , vol.441 , pp. 537-541
    • Grimm, D.1
  • 9
    • 44449121785 scopus 로고    scopus 로고
    • Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: Implications for the therapeutic development of RNAi
    • J.L. McBride Artificial miRNAs mitigate shRNA-mediated toxicity in the brain: implications for the therapeutic development of RNAi Proc. Natl. Acad. Sci. U. S. A. 105 2008 5868 5873
    • (2008) Proc. Natl. Acad. Sci. U. S. A. , vol.105 , pp. 5868-5873
    • McBride, J.L.1
  • 10
    • 78649906335 scopus 로고    scopus 로고
    • Argonaute proteins are key determinants of RNAi efficacy, toxicity and persistence in the adult mouse liver
    • (in press)
    • Grimm, D. et al. Argonaute proteins are key determinants of RNAi efficacy, toxicity and persistence in the adult mouse liver. J. Clin. Inv. (in press)
    • J. Clin. Inv.
    • Grimm, D.1
  • 11
    • 55849135596 scopus 로고    scopus 로고
    • MicroRNA-directed transcriptional gene silencing in mammalian cells
    • D.H. Kim MicroRNA-directed transcriptional gene silencing in mammalian cells Proc. Natl. Acad. Sci. U. S. A. 105 2008 16230 16235
    • (2008) Proc. Natl. Acad. Sci. U. S. A. , vol.105 , pp. 16230-16235
    • Kim, D.H.1
  • 12
    • 26944498261 scopus 로고    scopus 로고
    • Approaches for chemically synthesized siRNA and vector-mediated RNAi
    • M. Amarzguioui Approaches for chemically synthesized siRNA and vector-mediated RNAi FEBS Lett. 579 2005 5974 5981
    • (2005) FEBS Lett. , vol.579 , pp. 5974-5981
    • Amarzguioui, M.1
  • 13
    • 42549096043 scopus 로고    scopus 로고
    • Expression strategies for short hairpin RNA interference triggers
    • J.J. Rossi Expression strategies for short hairpin RNA interference triggers Hum. Gene Ther. 19 2008 313 317
    • (2008) Hum. Gene Ther. , vol.19 , pp. 313-317
    • Rossi, J.J.1
  • 14
    • 50649121877 scopus 로고    scopus 로고
    • Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs
    • R.L. Boudreau Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs RNA 14 2008 1834 1844
    • (2008) RNA , vol.14 , pp. 1834-1844
    • Boudreau, R.L.1
  • 15
    • 50549092566 scopus 로고    scopus 로고
    • Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic
    • J.C. Giering Expression of shRNA from a tissue-specific pol II promoter is an effective and safe RNAi therapeutic Mol. Ther. 16 2008 1630 1636
    • (2008) Mol. Ther. , vol.16 , pp. 1630-1636
    • Giering, J.C.1
  • 16
    • 0036787671 scopus 로고    scopus 로고
    • SiRNA-mediated gene silencing in vitro and in vivo
    • H. Xia siRNA-mediated gene silencing in vitro and in vivo Nat. Biotechnol. 20 2002 1006 1010
    • (2002) Nat. Biotechnol. , vol.20 , pp. 1006-1010
    • Xia, H.1
  • 17
    • 66349125020 scopus 로고    scopus 로고
    • Small interfering RNA-induced TLR3 activation inhibits blood and lymphatic vessel growth
    • W.G. Cho Small interfering RNA-induced TLR3 activation inhibits blood and lymphatic vessel growth Proc. Natl. Acad. Sci. U. S. A. 106 2009 7137 7142
    • (2009) Proc. Natl. Acad. Sci. U. S. A. , vol.106 , pp. 7137-7142
    • Cho, W.G.1
  • 18
    • 41649115210 scopus 로고    scopus 로고
    • Sequence- and target-independent angiogenesis suppression by siRNA via TLR3
    • M.E. Kleinman Sequence- and target-independent angiogenesis suppression by siRNA via TLR3 Nature 452 2008 591 597
    • (2008) Nature , vol.452 , pp. 591-597
    • Kleinman, M.E.1
  • 19
    • 74049124186 scopus 로고    scopus 로고
    • Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application
    • A.L. Jackson Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application Nat. Rev. Drug Discov. 9 2010 57 67
    • (2010) Nat. Rev. Drug Discov. , vol.9 , pp. 57-67
    • Jackson, A.L.1
  • 20
    • 0037089135 scopus 로고    scopus 로고
    • Positional effects of short interfering RNAs targeting the human coagulation trigger Tissue Factor
    • T. Holen Positional effects of short interfering RNAs targeting the human coagulation trigger Tissue Factor Nucleic Acids Res. 30 2002 1757 1766
    • (2002) Nucleic Acids Res. , vol.30 , pp. 1757-1766
    • Holen, T.1
  • 21
    • 0037111232 scopus 로고    scopus 로고
    • Small interfering RNA-mediated gene silencing in T lymphocytes
    • M.T. McManus Small interfering RNA-mediated gene silencing in T lymphocytes J. Immunol. 169 2002 5754 5760
    • (2002) J. Immunol. , vol.169 , pp. 5754-5760
    • McManus, M.T.1
  • 22
    • 34548548614 scopus 로고    scopus 로고
    • Combinatorial delivery of small interfering RNAs reduces RNAi efficacy by selective incorporation into RISC
    • D. Castanotto Combinatorial delivery of small interfering RNAs reduces RNAi efficacy by selective incorporation into RISC Nucleic Acids Res. 35 2007 5154 5164
    • (2007) Nucleic Acids Res. , vol.35 , pp. 5154-5164
    • Castanotto, D.1
  • 23
    • 33750027210 scopus 로고    scopus 로고
    • Competition for RISC binding predicts in vitro potency of siRNA
    • E. Koller Competition for RISC binding predicts in vitro potency of siRNA Nucleic Acids Res. 34 2006 4467 4476
    • (2006) Nucleic Acids Res. , vol.34 , pp. 4467-4476
    • Koller, E.1
  • 24
    • 36148972467 scopus 로고    scopus 로고
    • Reduced levels of Ago2 expression result in increased siRNA competition in mammalian cells
    • T.A. Vickers Reduced levels of Ago2 expression result in increased siRNA competition in mammalian cells Nucleic Acids Res. 35 2007 6598 6610
    • (2007) Nucleic Acids Res. , vol.35 , pp. 6598-6610
    • Vickers, T.A.1
  • 25
    • 12844285548 scopus 로고    scopus 로고
    • Overexpression of exportin 5 enhances RNA interference mediated by short hairpin RNAs and microRNAs
    • R. Yi Overexpression of exportin 5 enhances RNA interference mediated by short hairpin RNAs and microRNAs RNA 11 2005 220 226
    • (2005) RNA , vol.11 , pp. 220-226
    • Yi, R.1
  • 26
    • 0347361541 scopus 로고    scopus 로고
    • Exportin-5 mediates the nuclear export of pre-microRNAs and short hairpin RNAs
    • R. Yi Exportin-5 mediates the nuclear export of pre-microRNAs and short hairpin RNAs Genes Dev. 17 2003 3011 3016
    • (2003) Genes Dev. , vol.17 , pp. 3011-3016
    • Yi, R.1
  • 27
    • 77949446144 scopus 로고    scopus 로고
    • Cellular toxicity following application of adeno-associated viral vector-mediated RNA interference in the nervous system
    • E.M. Ehlert Cellular toxicity following application of adeno-associated viral vector-mediated RNA interference in the nervous system BMC Neurosci. 11 2010 20
    • (2010) BMC Neurosci. , vol.11 , pp. 20
    • Ehlert, E.M.1
  • 28
    • 48249097174 scopus 로고    scopus 로고
    • Coexpression of Argonaute-2 enhances RNA interference toward perfect match binding sites
    • S. Diederichs Coexpression of Argonaute-2 enhances RNA interference toward perfect match binding sites Proc. Natl. Acad. Sci. USA 105 2008 9284 9289
    • (2008) Proc. Natl. Acad. Sci. USA , vol.105 , pp. 9284-9289
    • Diederichs, S.1
  • 29
    • 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
    • D. Grimm 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 2003 281 304
    • (2003) Curr. Gene Ther. , vol.3 , pp. 281-304
    • Grimm, D.1
  • 30
    • 33845656937 scopus 로고    scopus 로고
    • Long-term inhibition of hepatitis B virus in transgenic mice by double-stranded adeno-associated virus 8-delivered short hairpin RNA
    • C.C. Chen Long-term inhibition of hepatitis B virus in transgenic mice by double-stranded adeno-associated virus 8-delivered short hairpin RNA Gene Ther. 14 2007 11 19
    • (2007) Gene Ther. , vol.14 , pp. 11-19
    • Chen, C.C.1
  • 31
    • 63649148742 scopus 로고    scopus 로고
    • Comparative study of anti-hepatitis B virus RNA interference by double-stranded adeno-associated virus serotypes 7, 8, and 9
    • C.C. Chen Comparative study of anti-hepatitis B virus RNA interference by double-stranded adeno-associated virus serotypes 7, 8, and 9 Mol. Ther. 17 2009 352 359
    • (2009) Mol. Ther. , vol.17 , pp. 352-359
    • Chen, C.C.1
  • 32
    • 59949096548 scopus 로고    scopus 로고
    • Essential and overlapping functions for mammalian Argonautes in microRNA silencing
    • H. Su Essential and overlapping functions for mammalian Argonautes in microRNA silencing Genes Dev. 23 2009 304 317
    • (2009) Genes Dev. , vol.23 , pp. 304-317
    • Su, H.1
  • 33
    • 50849117820 scopus 로고    scopus 로고
    • Importance of translation and nonnucleolytic ago proteins for on-target RNA interference
    • L. Wu Importance of translation and nonnucleolytic ago proteins for on-target RNA interference Curr. Biol. 18 2008 1327 1332
    • (2008) Curr. Biol. , vol.18 , pp. 1327-1332
    • Wu, L.1
  • 34
    • 61649128003 scopus 로고    scopus 로고
    • Rational design leads to more potent RNA interference against hepatitis B virus: Factors effecting silencing efficiency
    • K. Keck Rational design leads to more potent RNA interference against hepatitis B virus: factors effecting silencing efficiency Mol. Ther. 17 2009 538 547
    • (2009) Mol. Ther. , vol.17 , pp. 538-547
    • Keck, K.1
  • 35
    • 70349887777 scopus 로고    scopus 로고
    • Combinatorial RNAi against HIV-1 using extended short hairpin RNAs
    • Y.P. Liu Combinatorial RNAi against HIV-1 using extended short hairpin RNAs Mol. Ther. 17 2009 1712 1723
    • (2009) Mol. Ther. , vol.17 , pp. 1712-1723
    • Liu, Y.P.1
  • 36
    • 77957807722 scopus 로고    scopus 로고
    • Deriving four functional anti-HIV siRNAs from a single Pol III-generated transcript comprising two adjacent long hairpin RNA precursors
    • (in press)
    • Saayman, S. et al. Deriving four functional anti-HIV siRNAs from a single Pol III-generated transcript comprising two adjacent long hairpin RNA precursors. Nucleic Acids Res. (in press)
    • Nucleic Acids Res.
    • Saayman, S.1
  • 37
    • 52049117445 scopus 로고    scopus 로고
    • Silencing of hepatic fatty acid transporter protein 5 in vivo reverses diet-induced non-alcoholic fatty liver disease and improves hyperglycemia
    • H. Doege Silencing of hepatic fatty acid transporter protein 5 in vivo reverses diet-induced non-alcoholic fatty liver disease and improves hyperglycemia J. Biol. Chem. 283 2008 22186 22192
    • (2008) J. Biol. Chem. , vol.283 , pp. 22186-22192
    • Doege, H.1
  • 38
    • 44949131860 scopus 로고    scopus 로고
    • In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses
    • D. Grimm In vitro and in vivo gene therapy vector evolution via multispecies interbreeding and retargeting of adeno-associated viruses J. Virol. 82 2008 5887 5911
    • (2008) J. Virol. , vol.82 , pp. 5887-5911
    • Grimm, D.1
  • 39
    • 62549159967 scopus 로고    scopus 로고
    • Tailoring the AAV vector capsid for gene therapy
    • L.H. Vandenberghe Tailoring the AAV vector capsid for gene therapy Gene Ther. 16 2009 311 319
    • (2009) Gene Ther. , vol.16 , pp. 311-319
    • Vandenberghe, L.H.1
  • 40
    • 74149092180 scopus 로고    scopus 로고
    • Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice
    • S. Beer Low-level shRNA cytotoxicity can contribute to MYC-induced hepatocellular carcinoma in adult mice Mol. Ther. 18 2010 161 170
    • (2010) Mol. Ther. , vol.18 , pp. 161-170
    • Beer, S.1


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