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Volumn 19, Issue 12, 2011, Pages 2169-2177

Rational design of therapeutic siRNAs: Minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease

Author keywords

[No Author keywords available]

Indexed keywords

ARTIFICIAL MICRORNA; MICRORNA; NUCLEOTIDE; PARVOVIRUS VECTOR; SMALL INTERFERING RNA; UNCLASSIFIED DRUG;

EID: 82955233704     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2011.185     Document Type: Article
Times cited : (65)

References (41)
  • 1
    • 0036847540 scopus 로고    scopus 로고
    • Ribonuclease activity and RNA binding of recombinant human Dicer
    • DOI 10.1093/emboj/cdf578
    • Provost, P, Dishart, D, Doucet, J, Frendewey, D, Samuelsson, B and Rådmark, O (2002). Ribonuclease activity and RNA binding of recombinant human Dicer. EMBO J 21:5864-5874. (Pubitemid 35315284)
    • (2002) EMBO Journal , vol.21 , Issue.21 , pp. 5864-5874
    • Provost, P.1    Dishart, D.2    Doucet, J.3    Frendewey, D.4    Samuelsson, B.5    Radmark, O.6
  • 2
    • 77955902024 scopus 로고    scopus 로고
    • The widespread regulation of microRNA biogenesis, function and decay
    • Krol, J, Loedige, I and Filipowicz, W (2010). The widespread regulation of microRNA biogenesis, function and decay. Nat Rev Genet 11:597-610.
    • (2010) Nat Rev Genet , vol.11 , pp. 597-610
    • Krol, J.1    Loedige, I.2    Filipowicz, W.3
  • 5
    • 10744225153 scopus 로고    scopus 로고
    • Asymmetry in the assembly of the RNAi enzyme complex
    • DOI 10.1016/S0092-8674(03)00759-1
    • Schwarz, DS, Hutvágner, G, Du, T, Xu, Z, Aronin, N and Zamore, PD (2003). Asymmetry in the assembly of the RNAi enzyme complex. Cell 115:199-208. (Pubitemid 37329583)
    • (2003) Cell , vol.115 , Issue.2 , pp. 199-208
    • Schwarz, D.S.1    Hutvagner, G.2    Du, T.3    Xu, Z.4    Aronin, N.5    Zamore, P.D.6
  • 6
    • 0142165224 scopus 로고    scopus 로고
    • Functional siRNAs and miRNAs exhibit strand bias
    • DOI 10.1016/S0092-8674(03)00801-8
    • Khvorova, A, Reynolds, A and Jayasena, SD (2003). Functional siRNAs and miRNAs exhibit strand bias. Cell 115:209-216. (Pubitemid 37329584)
    • (2003) Cell , vol.115 , Issue.2 , pp. 209-216
    • Khvorova, A.1    Reynolds, A.2    Jayasena, S.D.3
  • 7
    • 77955644289 scopus 로고    scopus 로고
    • Mammalian microRNAs predominantly act to decrease target mRNA levels
    • Guo, H, Ingolia, NT, Weissman, JS and Bartel DP (2010). Mammalian microRNAs predominantly act to decrease target mRNA levels. Nature 466:835-840.
    • (2010) Nature , vol.466 , pp. 835-840
    • Guo, H.1    Ingolia, N.T.2    Weissman, J.S.3    Bartel, D.P.4
  • 8
    • 11844278458 scopus 로고    scopus 로고
    • Conserved seed pairing, often flanked by adenosines, indicates that thousands of human genes are microRNA targets
    • DOI 10.1016/j.cell.2004.12.035, PII S0092867404012607
    • Lewis, BP, Burge, CB and Bartel, DP (2005). Conserved seed pairing, often flanked by adenosines, indicates that thousands of human genes are microRNA targets. Cell 120:15-20. (Pubitemid 40094598)
    • (2005) Cell , vol.120 , Issue.1 , pp. 15-20
    • Lewis, B.P.1    Burge, C.B.2    Bartel, D.P.3
  • 9
    • 79954580272 scopus 로고    scopus 로고
    • Current prospects for RNA interference-based therapies
    • Davidson, BL and McCray, PB Jr (2011). Current prospects for RNA interference-based therapies. Nat Rev Genet 12:329-340.
    • (2011) Nat Rev Genet , vol.12 , pp. 329-340
    • Davidson, B.L.1    McCray Jr., P.B.2
  • 10
    • 0036251639 scopus 로고    scopus 로고
    • Effective expression of small interfering RNA in human cells
    • DOI 10.1038/nbt0502-505
    • Paul, CP, Good, PD, Winer, I and Engelke, DR (2002). Effective expression of small interfering RNA in human cells. Nat Biotechnol 20:505-508. (Pubitemid 34495939)
    • (2002) Nature Biotechnology , vol.20 , Issue.5 , pp. 505-508
    • Paul, C.P.1    Good, P.D.2    Winer, I.3    Engelke, D.R.4
  • 11
    • 0036294527 scopus 로고    scopus 로고
    • Both natural and designed micro RNAs can inhibit the expression of cognate mRNAs when expressed in human cells
    • DOI 10.1016/S1097-2765(02)00541-5
    • Zeng, Y, Wagner, EJ and Cullen, BR (2002). Both natural and designed micro RNAs can inhibit the expression of cognate mRNAs when expressed in human cells. Mol Cell 9:1327-1333. (Pubitemid 34722311)
    • (2002) Molecular Cell , vol.9 , Issue.6 , pp. 1327-1333
    • Zeng, Y.1    Wagner, E.J.2    Cullen, B.R.3
  • 19
    • 72549097028 scopus 로고    scopus 로고
    • SiDirect 2.0: Updated software for designing functional siRNA with reduced seed-dependent off-target effect
    • Naito, Y, Yoshimura, J, Morishita, S and Ui-Tei, K (2009). siDirect 2.0: updated software for designing functional siRNA with reduced seed-dependent off-target effect. BMC Bioinformatics 10:392.
    • (2009) BMC Bioinformatics , vol.10 , pp. 392
    • Naito, Y.1    Yoshimura, J.2    Morishita, S.3    Ui-Tei, K.4
  • 20
    • 74049124186 scopus 로고    scopus 로고
    • Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application
    • Jackson, AL and Linsley, PS (2010). Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application. Nat Rev Drug Discov 9:57-67.
    • (2010) Nat Rev Drug Discov , vol.9 , pp. 57-67
    • Jackson, A.L.1    Linsley, P.S.2
  • 21
    • 78650031174 scopus 로고    scopus 로고
    • Huntington's disease: From molecular pathogenesis to clinical treatment
    • Ross, CA and Tabrizi, SJ (2011). Huntington's disease: from molecular pathogenesis to clinical treatment. Lancet Neurol 10:83-98.
    • (2011) Lancet Neurol , vol.10 , pp. 83-98
    • Ross, C.A.1    Tabrizi, S.J.2
  • 23
    • 25144464388 scopus 로고    scopus 로고
    • Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice
    • DOI 10.1016/j.ymthe.2005.05.006, PII S1525001605002005
    • Rodriguez-Lebron, E, Denovan-Wright, EM, Nash, K, Lewin, AS and Mandel, RJ (2005). Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Mol Ther 12:618-633. (Pubitemid 41350143)
    • (2005) Molecular Therapy , vol.12 , Issue.4 , pp. 618-633
    • Rodriguez-Lebron, E.1    Denovan-Wright, E.M.2    Nash, K.3    Lewin, A.S.4    Mandel, R.J.5
  • 24
    • 42549134402 scopus 로고    scopus 로고
    • AAV vector-mediated RNAi of mutant Huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease
    • DOI 10.1038/mt.2008.50, PII MT200850
    • Franich, NR, Fitzsimons, HL, Fong, DM, Klugmann, M, During, MJ and Young, D (2008). AAV vector-mediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease. Mol Ther 16:947-956. (Pubitemid 351587106)
    • (2008) Molecular Therapy , vol.16 , Issue.5 , pp. 947-956
    • Franich, N.R.1    Fitzsimons, H.L.2    Fong, D.M.3    Klugmann, M.4    During, M.J.5    Young, D.6
  • 25
    • 67349100160 scopus 로고    scopus 로고
    • Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice
    • Boudreau, RL, McBride, JL, Martins, I, Shen, S, Xing, Y, Carter, BJ et al. (2009). Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. Mol Ther 17:1053-1063.
    • (2009) Mol Ther , vol.17 , pp. 1053-1063
    • Boudreau, R.L.1    McBride, J.L.2    Martins, I.3    Shen, S.4    Xing, Y.5    Carter, B.J.6
  • 27
    • 50649121877 scopus 로고    scopus 로고
    • Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs
    • Boudreau, RL, Monteys, AM and Davidson, BL (2008). Minimizing variables among hairpin-based RNAi vectors reveals the potency of shRNAs. RNA 14:1834-1844.
    • (2008) RNA , vol.14 , pp. 1834-1844
    • Boudreau, R.L.1    Monteys, A.M.2    Davidson, B.L.3
  • 29
    • 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 and Davidson, BL (2009). Artificial microRNAs as siRNA shuttles: improved safety as compared to shRNAs in vitro and in vivo. Mol Ther 17:169-175.
    • (2009) Mol Ther , vol.17 , pp. 169-175
    • Boudreau, R.L.1    Martins, I.2    Davidson, B.L.3
  • 30
    • 34250309149 scopus 로고    scopus 로고
    • Comparison of approaches for rational siRNA design leading to a new efficient and transparent method
    • Matveeva, O, Nechipurenko, Y, Rossi, L, Moore, B, Saetrom, P, Ogurtsov, AY et al. (2007). Comparison of approaches for rational siRNA design leading to a new efficient and transparent method. Nucleic Acids Res 35:e63.
    • (2007) Nucleic Acids Res , vol.35
    • Matveeva, O.1    Nechipurenko, Y.2    Rossi, L.3    Moore, B.4    Saetrom, P.5    Ogurtsov, A.Y.6
  • 32
    • 34250805982 scopus 로고    scopus 로고
    • MicroRNA Targeting Specificity in Mammals: Determinants beyond Seed Pairing
    • DOI 10.1016/j.molcel.2007.06.017, PII S1097276507004078
    • Grimson, A, Farh, KK, Johnston, WK, Garrett-Engele, P, Lim, LP and Bartel, DP (2007). MicroRNA targeting specificity in mammals: determinants beyond seed pairing. Mol Cell 27:91-105. (Pubitemid 46991392)
    • (2007) Molecular Cell , vol.27 , Issue.1 , pp. 91-105
    • Grimson, A.1    Farh, K.K.-H.2    Johnston, W.K.3    Garrett-Engele, P.4    Lim, L.P.5    Bartel, D.P.6
  • 34
    • 43349107172 scopus 로고    scopus 로고
    • Small interfering RNA therapy in cancer: Mechanism, potential targets, and clinical applications
    • DOI 10.1517/14728222.12.5.637
    • Huang, C, Li, M, Chen, C and Yao, Q (2008). Small interfering RNA therapy in cancer: mechanism, potential targets, and clinical applications. Expert Opin Ther Targets 12:637-645. (Pubitemid 351699481)
    • (2008) Expert Opinion on Therapeutic Targets , vol.12 , Issue.5 , pp. 637-645
    • Huang, C.1    Li, M.2    Chen, C.3    Yao, Q.4
  • 35
    • 58249106361 scopus 로고    scopus 로고
    • MicroRNA-like off-target transcript regulation by siRNAs is species specific
    • Burchard, J, Jackson, AL, Malkov, V, Needham, RH, Tan, Y, Bartz, SR et al. (2009). MicroRNA-like off-target transcript regulation by siRNAs is species specific. RNA 15:308-315.
    • (2009) RNA , vol.15 , pp. 308-315
    • Burchard, J.1    Jackson, A.L.2    Malkov, V.3    Needham, R.H.4    Tan, Y.5    Bartz, S.R.6
  • 37
    • 77957241115 scopus 로고    scopus 로고
    • A screen of chemical modifications identifies position-specific modification by UNA to most potently reduce siRNA off-target effects
    • Bramsen, JB, Pakula, MM, Hansen, TB, Bus, C, Langkjær, N, Odadzic, D et al. (2010). A screen of chemical modifications identifies position-specific modification by UNA to most potently reduce siRNA off-target effects. Nucleic Acids Res 38:5761-5773.
    • (2010) Nucleic Acids Res , vol.38 , pp. 5761-5773
    • Bramsen, J.B.1    Pakula, M.M.2    Hansen, T.B.3    Bus, C.4    Langkjær, N.5    Odadzic, D.6
  • 38
    • 79953144826 scopus 로고    scopus 로고
    • Improved specificity of gene silencing by siRNAs containing unlocked nucleobase analogs
    • Vaish, N, Chen, F, Seth, S, Fosnaugh, K, Liu, Y, Adami, R et al. (2011). Improved specificity of gene silencing by siRNAs containing unlocked nucleobase analogs. Nucleic Acids Res 39:1823-1832.
    • (2011) Nucleic Acids Res , vol.39 , pp. 1823-1832
    • Vaish, N.1    Chen, F.2    Seth, S.3    Fosnaugh, K.4    Liu, Y.5    Adami, R.6
  • 39
    • 0036431701 scopus 로고    scopus 로고
    • Insect cells as a factory to produce adeno-associated virus type 2 vectors
    • DOI 10.1089/10430340260355347
    • Urabe, M, Ding, C and Kotin, RM (2002). Insect cells as a factory to produce adenoassociated virus type 2 vectors. Hum Gene Ther 13:1935-1943. (Pubitemid 35341035)
    • (2002) Human Gene Therapy , vol.13 , Issue.16 , pp. 1935-1943
    • Urabe, M.1    Ding, C.2    Kotin, R.M.3
  • 40
    • 41349115432 scopus 로고    scopus 로고
    • The DAVID gene functional classification tool: A novel biological module-centric algorithm to functionally analyze large gene lists
    • Huang da, W, Sherman, BT, Tan, Q, Collins, JR, Alvord, WG, Roayaei, J et al. (2007). The DAVID Gene Functional Classification Tool: a novel biological module-centric algorithm to functionally analyze large gene lists. Genome Biol 8:R183.
    • (2007) Genome Biol , vol.8
    • Da Huang, W.1    Sherman, B.T.2    Tan, Q.3    Collins, J.R.4    Alvord, W.G.5    Roayaei, J.6
  • 41
    • 63249135662 scopus 로고    scopus 로고
    • Allegro: Analyzing expression and sequence in concert to discover regulatory programs
    • Halperin, Y, Linhart, C, Ulitsky, I and Shamir, R (2009). Allegro: analyzing expression and sequence in concert to discover regulatory programs. Nucleic Acids Res 37:1566-1579.
    • (2009) Nucleic Acids Res , vol.37 , pp. 1566-1579
    • Halperin, Y.1    Linhart, C.2    Ulitsky, I.3    Shamir, R.4


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