메뉴 건너뛰기




Volumn 18, Issue 12, 2011, Pages 1139-1149

Using non-coding small RNAs to develop therapies for Huntington's disease

Author keywords

allele specific; Huntington's disease; mutant HD; non coding small RNAs; therapy; wild type HD

Indexed keywords

HUNTINGTIN; MESSENGER RNA; SMALL INTERFERING RNA; SMALL UNTRANSLATED RNA;

EID: 83255177161     PISSN: 09697128     EISSN: 14765462     Source Type: Journal    
DOI: 10.1038/gt.2011.170     Document Type: Review
Times cited : (38)

References (86)
  • 1
    • 0027480960 scopus 로고
    • A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes
    • The Huntington's Disease Collaborative Research Group
    • The Huntington's Disease Collaborative Research Group. A novel gene containing a trinucleotide repeat that is expanded and unstable on Huntington's disease chromosomes. Cell 1993; 72: 971-983.
    • (1993) Cell , vol.72 , pp. 971-983
  • 2
    • 68549104413 scopus 로고    scopus 로고
    • Progress and challenges in RNA interference therapy for Huntington disease
    • Harper SQ. Progress and challenges in RNA interference therapy for Huntington disease. Arch Neurol 2009; 66: 933-938.
    • (2009) Arch Neurol , vol.66 , pp. 933-938
    • Harper, S.Q.1
  • 3
    • 33645135860 scopus 로고    scopus 로고
    • RNAi: A potential therapy for the dominantly inherited nucleotide repeat diseases
    • Denovan-Wright EM, Davidson BL. RNAi: a potential therapy for the dominantly inherited nucleotide repeat diseases. Gene Therapy 2006; 13: 525-531.
    • (2006) Gene Therapy , vol.13 , pp. 525-531
    • Denovan-Wright, E.M.1    Davidson, B.L.2
  • 5
    • 26844494370 scopus 로고    scopus 로고
    • Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA
    • DOI 10.1016/j.neures.2005.06.021, PII S0168010205001926
    • Wang YL, Liu W, Wada E, Murata M, Wada K, Kanazawa I. Clinico-pathological rescue of a model mouse of Huntington's disease by siRNA. Neurosci Res 2005; 53: 241-249. (Pubitemid 41463777)
    • (2005) Neuroscience Research , vol.53 , Issue.3 , pp. 241-249
    • Wang, Y.-L.1    Liu, W.2    Wada, E.3    Murata, M.4    Wada, K.5    Kanazawa, I.6
  • 6
    • 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, Young D. AAV vectormediated RNAi of mutant huntingtin expression is neuroprotective in a novel genetic rat model of Huntington's disease. Mol Ther 2008; 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
  • 8
    • 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. Nonallele-specific silencing of mutant and wild-type huntingtin demonstrates therapeutic efficacy in Huntington's disease mice. Mol Ther 2009; 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
  • 10
    • 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, Mandel RJ. Intrastriatal rAAV-mediated delivery of anti-huntingtin shRNAs induces partial reversal of disease progression in R6/1 Huntington's disease transgenic mice. Mol Ther 2005; 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
  • 14
    • 34248146697 scopus 로고    scopus 로고
    • High-capacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo
    • DOI 10.1089/hum.2006.160
    • Huang B, Schiefer J, Sass C, Landwehrmeyer GB, Kosinski CM, Kochanek S. Highcapacity adenoviral vector-mediated reduction of huntingtin aggregate load in vitro and in vivo. Hum Gene Ther 2007; 18: 303-311. (Pubitemid 46709342)
    • (2007) Human Gene Therapy , vol.18 , Issue.4 , pp. 303-311
    • Huang, B.1    Schiefer, J.2    Sass, C.3    Landwehrmeyer, G.B.4    Kosinski, C.M.5    Kochanek, S.6
  • 15
    • 83255176538 scopus 로고    scopus 로고
    • Silencing Huntington's disease (HD) gene with RNAi
    • Erdmann VA and Barciszewski J (eds) Springer: Berlin
    • Zhang Y, Friedlander RM. Silencing Huntington's disease (HD) gene with RNAi. In: Erdmann VA and Barciszewski J (eds). RNA Technologies and Their Applications. Springer: Berlin, 2010, pp 131-160.
    • (2010) RNA Technologies and Their Applications , pp. 131-160
    • Zhang, Y.1    Friedlander, R.M.2
  • 16
    • 47549105506 scopus 로고    scopus 로고
    • Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts
    • Van Bilsen PH, Jaspers L, Lombardi MS, Odekerken JC, Burright EN, Kaemmerer WF. Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts. Hum Gene Ther 2008; 19: 710-719.
    • (2008) Hum Gene Ther , vol.19 , pp. 710-719
    • Van Bilsen, P.H.1    Jaspers, L.2    Lombardi, M.S.3    Odekerken, J.C.4    Burright, E.N.5    Kaemmerer, W.F.6
  • 17
    • 57249086448 scopus 로고    scopus 로고
    • Allele-specific silencing of mutant Huntington's disease gene
    • Zhang Y, Engelman J, Friedlander RM. Allele-specific silencing of mutant Huntington's disease gene. J Neurochem 2009; 108: 82-90.
    • (2009) J Neurochem , vol.108 , pp. 82-90
    • Zhang, Y.1    Engelman, J.2    Friedlander, R.M.3
  • 18
    • 70349469318 scopus 로고    scopus 로고
    • Allele-selective inhibition of mutant huntingtin by peptide nucleic acid-peptide conjugates, locked nucleic acid, and small interfering RNA
    • Hu J, Matsui M, Corey DR. Allele-selective inhibition of mutant huntingtin by peptide nucleic acid-peptide conjugates, locked nucleic acid, and small interfering RNA. Ann NY Acad Sci 2009; 1175: 24-31.
    • (2009) Ann NY Acad Sci , vol.1175 , pp. 24-31
    • Hu, J.1    Matsui, M.2    Corey, D.R.3
  • 21
    • 67349263503 scopus 로고    scopus 로고
    • A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference
    • Lombardi MS, Jaspers L, Spronkmans C, Gellera C, Taroni F, Di Maria E et al. A majority of Huntington's disease patients may be treatable by individualized allele-specific RNA interference. Exp Neurol 2009; 217: 312-319.
    • (2009) Exp Neurol , vol.217 , pp. 312-319
    • Lombardi, M.S.1    Jaspers, L.2    Spronkmans, C.3    Gellera, C.4    Taroni, F.5    Di Maria, E.6
  • 22
    • 79956023002 scopus 로고    scopus 로고
    • Mutant CAG repeats of huntingtin transcript fold into hairpins, form nuclear foci and are targets for RNA interference
    • De Mezer M, Wojciechowska M, Napierala M, Sobczak K, Krzyzosiak WJ. Mutant CAG repeats of huntingtin transcript fold into hairpins, form nuclear foci and are targets for RNA interference. Nucleic Acids Res 2011; 39: 3852-3863.
    • (2011) Nucleic Acids Res , vol.39 , pp. 3852-3863
    • De Mezer, M.1    Wojciechowska, M.2    Napierala, M.3    Sobczak, K.4    Krzyzosiak, W.J.5
  • 23
    • 66149181896 scopus 로고    scopus 로고
    • Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs
    • Hu J, Matsui M, Gagnon KT, Schwartz JC, Gabillet S, Arar K et al. Allele-specific silencing of mutant huntingtin and ataxin-3 genes by targeting expanded CAG repeats in mRNAs. Nat Biotechnol 2009; 27: 478-484.
    • (2009) Nat Biotechnol , vol.27 , pp. 478-484
    • Hu, J.1    Matsui, M.2    Gagnon, K.T.3    Schwartz, J.C.4    Gabillet, S.5    Arar, K.6
  • 24
    • 78649379362 scopus 로고    scopus 로고
    • Alleleselective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat
    • Gagnon KT, Pendergraff HM, Deleavey GF, Swayze EE, Potier P, Randolph J et al. Alleleselective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat. Biochemistry 2010; 49: 10166-10178.
    • (2010) Biochemistry , vol.49 , pp. 10166-10178
    • Gagnon, K.T.1    Pendergraff, H.M.2    Deleavey, G.F.3    Swayze, E.E.4    Potier, P.5    Randolph, J.6
  • 25
    • 78649359013 scopus 로고    scopus 로고
    • Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism
    • Hu J, Liu J, Corey DR. Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism. Chem Biol 2010; 17: 1183-1188.
    • (2010) Chem Biol , vol.17 , pp. 1183-1188
    • Hu, J.1    Liu, J.2    Corey, D.R.3
  • 26
    • 80052250736 scopus 로고    scopus 로고
    • Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats
    • Fiszer A, Mykowska A, Krzyzosiak WJ. Inhibition of mutant huntingtin expression by RNA duplex targeting expanded CAG repeats. Nucleic Acids Res 2011; 39: 5578-5585.
    • (2011) Nucleic Acids Res , vol.39 , pp. 5578-5585
    • Fiszer, A.1    Mykowska, A.2    Krzyzosiak, W.J.3
  • 31
    • 0033999321 scopus 로고    scopus 로고
    • Intraneuronal aggregate formation and cell death after viral expression of expanded polyglutamine tracts in the adult rat brain
    • Senut MC, Suhr ST, Kaspar B, Gage FH. Intraneuronal aggregate formation and cell death after viral expression of expanded polyglutamine tracts in the adult rat brain. J Neurosci 2000; 20: 219-229. (Pubitemid 30220509)
    • (2000) Journal of Neuroscience , vol.20 , Issue.1 , pp. 219-229
    • Senut, M.-C.1    Suhr, S.T.2    Kaspar, B.3    Gage, F.H.4
  • 32
    • 0036580677 scopus 로고    scopus 로고
    • Lentiviral-Mediated Delivery of Mutant Huntingtin in the Striatum of Rats Induces a Selective Neuropathology Modulated by Polyglutamine Repeat Size, Huntingtin Expression Levels, and Protein Length
    • De Almeida LP, Ross CA, Zala D, Aebischer P, Deglon N. Lentiviral-mediated delivery of mutant huntingtin in the striatum of rats induces a selective neuropathology modulated by polyglutamine repeat size, huntingtin expression levels, and protein length. J Neurosci 2002; 22: 3473-3483. (Pubitemid 37465753)
    • (2002) Journal of Neuroscience , vol.22 , Issue.9 , pp. 3473-3483
    • De Almeida, L.P.1    Ross, C.A.2    Zala, D.3    Aebischer, P.4    Deglon, N.5
  • 34
    • 46749157501 scopus 로고    scopus 로고
    • Full-length human mutant huntingtin with a stable polyglutamine repeat can elicit progressive and selective neuropathogenesis in BACHD mice
    • Gray M, Shirasaki DI, Cepeda C, Andre VM, Wilburn B, Lu XH et al. Full-length human mutant huntingtin with a stable polyglutamine repeat can elicit progressive and selective neuropathogenesis in BACHD mice. J Neurosci 2008; 28: 6182-6195.
    • (2008) J Neurosci , vol.28 , pp. 6182-6195
    • Gray, M.1    Shirasaki, D.I.2    Cepeda, C.3    Andre, V.M.4    Wilburn, B.5    Lu, X.H.6
  • 35
    • 56249087909 scopus 로고    scopus 로고
    • Progressive synaptic pathology of motor cortical neurons in a BAC transgenic mouse model of Huntington's disease
    • Spampanato J, Gu X, Yang XW, Mody I. Progressive synaptic pathology of motor cortical neurons in a BAC transgenic mouse model of Huntington's disease. Neuroscience 2008; 157: 606-620.
    • (2008) Neuroscience , vol.157 , pp. 606-620
    • Spampanato, J.1    Gu, X.2    Yang, X.W.3    Mody, I.4
  • 38
    • 61549104503 scopus 로고    scopus 로고
    • CAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroup
    • Warby SC, Montpetit A, Hayden AR, Carroll JB, Butland SL, Visscher H et al. CAG expansion in the Huntington disease gene is associated with a specific and targetable predisposing haplogroup. Am J Hum Genet 2009; 84: 351-366.
    • (2009) Am J Hum Genet , vol.84 , pp. 351-366
    • Warby, S.C.1    Montpetit, A.2    Hayden, A.R.3    Carroll, J.B.4    Butland, S.L.5    Visscher, H.6
  • 40
    • 12244266688 scopus 로고
    • Ancestral differences in the distribution of the delta 2642 glutamic acid polymorphism is associated with varying CAG repeat lengths on normal chromosomes: Insights into the genetic evolution of Huntington disease
    • Almqvist E, Spence N, Nichol K, Andrew SE, Vesa J, Peltonen L et al. Ancestral differences in the distribution of the delta 2642 glutamic acid polymorphism is associated with varying CAG repeat lengths on normal chromosomes: insights into the genetic evolution of Huntington disease. Hum Mol Genet 1995; 4: 207-214.
    • (1995) Hum Mol Genet , vol.4 , pp. 207-214
    • Almqvist, E.1    Spence, N.2    Nichol, K.3    Andrew, S.E.4    Vesa, J.5    Peltonen, L.6
  • 41
    • 0028986598 scopus 로고
    • Haplotype analysis of the delta 2642 and (CAG)n polymorphisms in the Huntington's disease (HD) gene provides an explanation for an apparent 'founder' HD haplotype
    • Rubinsztein DC, Leggo J, Goodburn S, Barton DE, Ferguson-Smith MA. Haplotype analysis of the delta 2642 and (CAG)n polymorphisms in the Huntington's disease (HD) gene provides an explanation for an apparent 'founder' HD haplotype. Hum Mol Genet 1995; 4: 203-206.
    • (1995) Hum Mol Genet , vol.4 , pp. 203-206
    • Rubinsztein, D.C.1    Leggo, J.2    Goodburn, S.3    Barton, D.E.4    Ferguson-Smith, M.A.5
  • 44
    • 0142009656 scopus 로고    scopus 로고
    • RNA structure of trinucleotide repeats associated with human neurological diseases
    • DOI 10.1093/nar/gkg766
    • Sobczak K, de Mezer M, Michlewski G, Krol J, Krzyzosiak WJ. RNA structure of trinucleotide repeats associated with human neurological diseases. Nucleic Acids Res 2003; 31: 5469-5482. (Pubitemid 37441917)
    • (2003) Nucleic Acids Research , vol.31 , Issue.19 , pp. 5469-5482
    • Sobczak, K.1    De Mezer, M.2    Michlewski, G.3    Krol, J.4    Krzyzosiak, W.J.5
  • 45
    • 0029053371 scopus 로고
    • Trinucleotide repeats that expand in human disease form hairpin structures in vitro
    • Gacy AM, Goellner G, Juranic N, Macura S, McMurray CT. Trinucleotide repeats that expand in human disease form hairpin structures in vitro. Cell 1995; 81: 533-540.
    • (1995) Cell , vol.81 , pp. 533-540
    • Gacy, A.M.1    Goellner, G.2    Juranic, N.3    MacUra, S.4    McMurray, C.T.5
  • 46
    • 23844465175 scopus 로고    scopus 로고
    • Thermodynamic stability of RNA structures formed by CNG trinucleotide repeats. Implication for prediction of RNA structure
    • DOI 10.1021/bi0502339
    • Broda M, Kierzek E, Gdaniec Z, Kulinski T, Kierzek R. Thermodynamic stability of RNA structures formed by CNG trinucleotide repeats. Implication for prediction of RNA structure. Biochemistry 2005; 44: 10873-10882. (Pubitemid 41153648)
    • (2005) Biochemistry , vol.44 , Issue.32 , pp. 10873-10882
    • Broda, M.1    Kierzek, E.2    Gdaniec, Z.3    Kulinski, T.4    Kierzek, R.5
  • 48
    • 3042561985 scopus 로고    scopus 로고
    • Molecular architecture of CAG repeats in human disease related transcripts
    • DOI 10.1016/j.jmb.2004.05.021, PII S0022283604005868
    • Michlewski G, Krzyzosiak WJ. Molecular architecture of CAG repeats in human disease related transcripts. J Mol Biol 2004; 340: 665-679. (Pubitemid 38829630)
    • (2004) Journal of Molecular Biology , vol.340 , Issue.4 , pp. 665-679
    • Michlewski, G.1    Krzyzosiak, W.J.2
  • 49
    • 33847077134 scopus 로고    scopus 로고
    • Ribonuclease Dicer Cleaves Triplet Repeat Hairpins into Shorter Repeats that Silence Specific Targets
    • DOI 10.1016/j.molcel.2007.01.031, PII S1097276507000548
    • Krol J, Fiszer A, Mykowska A, Sobczak K, de Mezer M, Krzyzosiak WJ. Ribonuclease dicer cleaves triplet repeat hairpins into shorter repeats that silence specific targets. Mol Cell 2007; 25: 575-586. (Pubitemid 46274449)
    • (2007) Molecular Cell , vol.25 , Issue.4 , pp. 575-586
    • Krol, J.1    Fiszer, A.2    Mykowska, A.3    Sobczak, K.4    De Mezer, M.5    Krzyzosiak, W.J.6
  • 50
    • 1842406027 scopus 로고    scopus 로고
    • CUG repeats present in myotonin kinase RNA form metastable 'slippery' hairpins
    • DOI 10.1074/jbc.272.49.31079
    • Napierala M, Krzyzosiak WJ. CUG repeats present in myotonin kinase RNA form metastable "slippery" hairpins. J Biol Chem 1997; 272: 31079-31085. (Pubitemid 27527556)
    • (1997) Journal of Biological Chemistry , vol.272 , Issue.49 , pp. 31079-31085
    • Napierala, M.1    Krzyzosiak, W.J.2
  • 51
    • 0036924811 scopus 로고    scopus 로고
    • Antisense inhibition of gene expression in cells by oligonucleotides incorporating locked nucleic acids: Effect of mRNA target sequence and chimera design
    • DOI 10.1093/nar/gkf651
    • Braasch DA, Liu Y, Corey DR. Antisense inhibition of gene expression in cells by oligonucleotides incorporating locked nucleic acids: effect of mRNA target sequence and chimera design. Nucleic Acids Res 2002; 30: 5160-5167. (Pubitemid 36033034)
    • (2002) Nucleic Acids Research , vol.30 , Issue.23 , pp. 5160-5167
    • Braasch, D.A.1    Liu, Y.2    Corey, D.R.3
  • 52
    • 0033607615 scopus 로고    scopus 로고
    • Antisense PNA tridecamers targeted to the coding region of Ha-ras mRNA arrest polypeptide chain elongation
    • Dias N, Dheur S, Nielsen PE, Gryaznov S, Van Aerschot A, Herdewijn P et al. Antisense PNA tridecamers targeted to the coding region of Ha-ras mRNA arrest polypeptide chain elongation. J Mol Biol 1999; 294: 403-416.
    • (1999) J Mol Biol , vol.294 , pp. 403-416
    • Dias, N.1    Dheur, S.2    Nielsen, P.E.3    Gryaznov, S.4    Van Aerschot, A.5    Herdewijn, P.6
  • 54
    • 17844387370 scopus 로고    scopus 로고
    • A computational study of off-target effects of RNA interference
    • DOI 10.1093/nar/gki324
    • Qiu S, Adema CM, Lane T. A computational study of off-target effects of RNA interference. Nucleic Acids Res 2005; 33: 1834-1847. (Pubitemid 41748330)
    • (2005) Nucleic Acids Research , vol.33 , Issue.6 , pp. 1834-1847
    • Qiu, S.1    Adema, C.M.2    Lane, T.3
  • 56
    • 33745612464 scopus 로고    scopus 로고
    • Widespread siRNA "off-target" transcript silencing mediated by seed region sequence complementarity
    • DOI 10.1261/rna.25706
    • Jackson AL, Burchard J, Schelter J, Chau BN, Cleary M, Lim L et al. Widespread siRNA "off-target" transcript silencing mediated by seed region sequence complementarity. RNA 2006; 12: 1179-1187. (Pubitemid 43990543)
    • (2006) RNA , vol.12 , Issue.7 , pp. 1179-1187
    • Jackson, A.L.1    Burchard, J.2    Schelter, J.3    Chau, B.N.4    Cleary, M.5    Lim, L.6    Linsley, P.S.7
  • 58
    • 1642374097 scopus 로고    scopus 로고
    • Specificity of microRNA target selection in translational repression
    • DOI 10.1101/gad.1184404
    • Doench JG, Sharp PA. Specificity of microRNA target selection in translational repression. Genes Dev 2004; 18: 504-511. (Pubitemid 38372808)
    • (2004) Genes and Development , vol.18 , Issue.5 , pp. 504-511
    • Doench, J.G.1    Sharp, P.A.2
  • 59
  • 60
    • 10744225153 scopus 로고    scopus 로고
    • Asymmetry in the assembly of the RNAi enzyme complex
    • DOI 10.1016/S0092-8674(03)00759-1
    • Schwarz DS, Hutvagner G, Du T, Xu Z, Aronin N, Zamore PD. Asymmetry in the assembly of the RNAi enzyme complex. Cell 2003; 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
  • 61
    • 0142165224 scopus 로고    scopus 로고
    • Functional siRNAs and miRNAs exhibit strand bias
    • DOI 10.1016/S0092-8674(03)00801-8
    • Khvorova A, Reynolds A, Jayasena SD. Functional siRNAs and miRNAs exhibit strand bias. Cell 2003; 115: 209-216. (Pubitemid 37329584)
    • (2003) Cell , vol.115 , Issue.2 , pp. 209-216
    • Khvorova, A.1    Reynolds, A.2    Jayasena, S.D.3
  • 63
    • 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, Bartel DP. MicroRNA targeting specificity in mammals: determinants beyond seed pairing. Mol Cell 2007; 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
  • 64
    • 4344566457 scopus 로고    scopus 로고
    • Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system
    • DOI 10.1016/j.ymthe.2004.05.024, PII S1525001604001911
    • Burger C, Gorbatyuk OS, Velardo MJ, Peden CS, Williams P, Zolotukhin S et al. Recombinant AAV viral vectors pseudotyped with viral capsids from serotypes 1, 2, and 5 display differential efficiency and cell tropism after delivery to different regions of the central nervous system. Mol Ther 2004; 10: 302-317. (Pubitemid 39117875)
    • (2004) Molecular Therapy , vol.10 , Issue.2 , pp. 302-317
    • Burger, C.1    Gorbatyuk, O.S.2    Velardo, M.J.3    Peden, C.S.4    Williams, P.5    Zolotukhin, S.6    Reier, P.J.7    Mandel, R.J.8    Muzyczka, N.9
  • 65
    • 2942689239 scopus 로고    scopus 로고
    • Transduction profiles of recombinant adeno-associated virus vectors derived from serotypes 2 and 5 in the nigrostriatal system of rats
    • DOI 10.1128/JVI.78.13.6808-6817.2004
    • Paterna JC, Feldon J, Bueler H. Transduction profiles of recombinant adeno-associated virus vectors derived from serotypes 2 and 5 in the nigrostriatal system of rats. J Virol 2004; 78: 6808-6817. (Pubitemid 38781518)
    • (2004) Journal of Virology , vol.78 , Issue.13 , pp. 6808-6817
    • Paterna, J.-C.1    Feldon, J.2    Bueler, H.3
  • 69
    • 33751099034 scopus 로고    scopus 로고
    • RNAi therapeutics: A potential new class of pharmaceutical drugs
    • DOI 10.1038/nchembio839, PII NCHEMBIO839
    • Bumcrot D, Manoharan M, Koteliansky V, Sah DW. RNAi therapeutics: a potential new class of pharmaceutical drugs. Nat Chem Biol 2006; 2: 711-719. (Pubitemid 44764213)
    • (2006) Nature Chemical Biology , vol.2 , Issue.12 , pp. 711-719
    • Bumcrot, D.1    Manoharan, M.2    Koteliansky, V.3    Sah, D.W.Y.4
  • 71
    • 3142720243 scopus 로고    scopus 로고
    • Synthesis and physical and physiological properties of 4′-thioRNA: Application to post-modification of RNA aptamer toward NF-κB
    • DOI 10.1093/nar/gkh705
    • Hoshika S, Minakawa N, Matsuda A. Synthesis and physical and physiological properties of 4'-thioRNA: application to post-modification of RNA aptamer toward NFkappaB. Nucleic Acids Res 2004; 32: 3815-3825. (Pubitemid 39199074)
    • (2004) Nucleic Acids Research , vol.32 , Issue.13 , pp. 3815-3825
    • Hoshika, S.1    Minakawa, N.2    Matsuda, A.3
  • 72
    • 33644860608 scopus 로고    scopus 로고
    • Improving RNA interference in mammalian cells by 4′-thio-modified small interfering RNA (siRNA): Effect on siRNA activity and nuclease stability when used in combination with 2′-O-alkyl modifications
    • DOI 10.1021/jm050822c
    • Dande P, Prakash TP, Sioufi N, Gaus H, Jarres R, Berdeja A et al. Improving RNA interference in mammalian cells by 4'-thio-modified small interfering RNA (siRNA): effect on siRNA activity and nuclease stability when used in combination with 2'-Oalkyl modifications. J Med Chem 2006; 49: 1624-1634. (Pubitemid 43376507)
    • (2006) Journal of Medicinal Chemistry , vol.49 , Issue.5 , pp. 1624-1634
    • Dande, P.1    Prakash, T.P.2    Sioufi, N.3    Gaus, H.4    Jarres, R.5    Berdeja, A.6    Swayze, E.E.7    Griffey, R.H.8    Bhat, B.9
  • 75
    • 0037440005 scopus 로고    scopus 로고
    • Tolerance for mutations and chemical modifications in a siRNA
    • DOI 10.1093/nar/gkg147
    • Amarzguioui M, Holen T, Babaie E, Prydz H. Tolerance for mutations and chemical modifications in a siRNA. Nucleic Acids Res 2003; 31: 589-595. (Pubitemid 36175948)
    • (2003) Nucleic Acids Research , vol.31 , Issue.2 , pp. 589-595
    • Amarzguioui, M.1    Holen, T.2    Babaie, E.3    Prydz, H.4
  • 76
    • 33745630675 scopus 로고    scopus 로고
    • Stearylated octaarginine and artificial virus-like particles for transfection of siRNA into primary rat neurons
    • DOI 10.1261/rna.2252206
    • Tonges L, Lingor P, Egle R, Dietz GP, Fahr A, Bahr M. Stearylated octaarginine and artificial virus-like particles for transfection of siRNA into primary rat neurons. RNA 2006; 12: 1431-1438. (Pubitemid 43990566)
    • (2006) RNA , vol.12 , Issue.7 , pp. 1431-1438
    • Tonges, L.1    Lingor, P.2    Egle, R.3    Dietz, G.P.H.4    Fahr, A.5    Bahr, M.6
  • 79
    • 25144514032 scopus 로고    scopus 로고
    • Peptide-based strategy for siRNA delivery into mammalian cells
    • Simeoni F, Morris MC, Heitz F, Divita G. Peptide-based strategy for siRNA delivery into mammalian cells. Methods Mol Biol 2005; 309: 251-260.
    • (2005) Methods Mol Biol , vol.309 , pp. 251-260
    • Simeoni, F.1    Morris, M.C.2    Heitz, F.3    Divita, G.4
  • 83
    • 8544231403 scopus 로고    scopus 로고
    • Highly efficient small interfering RNA delivery to primary mammalian neurons induces microRNA-like effects before mRNA degradation
    • DOI 10.1523/JNEUROSCI.3643-04.2004
    • Davidson TJ, Harel S, Arboleda VA, Prunell GF, Shelanski ML, Greene LA et al. Highly efficient small interfering RNA delivery to primary mammalian neurons induces MicroRNA-like effects before mRNA degradation. J Neurosci 2004; 24: 10040-10046. (Pubitemid 39492027)
    • (2004) Journal of Neuroscience , vol.24 , Issue.45 , pp. 10040-10046
    • Davidson, T.J.1    Harel, S.2    Arboleda, V.A.3    Prunell, G.F.4    Shelanski, M.L.5    Greene, L.A.6    Troy, C.M.7
  • 84
    • 0842346326 scopus 로고    scopus 로고
    • Conjugate for efficient delivery of short interfering RNA (siRNA) into mammalian cells
    • DOI 10.1016/S0014-5793(03)01505-9
    • Muratovska A, Eccles MR. Conjugate for efficient delivery of short interfering RNA (siRNA) into mammalian cells. FEBS Lett 2004; 558: 63-68. (Pubitemid 38167411)
    • (2004) FEBS Letters , vol.558 , Issue.1-3 , pp. 63-68
    • Muratovska, A.1    Eccles, M.R.2
  • 85
    • 63849138722 scopus 로고    scopus 로고
    • Potential application of RNAi for understanding and therapy of neurodegenerative diseases
    • Schwartz EI. Potential application of RNAi for understanding and therapy of neurodegenerative diseases. Front Biosci 2009; 14: 297-320.
    • (2009) Front Biosci , vol.14 , pp. 297-320
    • Schwartz, E.I.1
  • 86
    • 80054741160 scopus 로고    scopus 로고
    • RNAi: A potential new class of therapeutic for human genetic disease
    • Seyhan AA. RNAi: a potential new class of therapeutic for human genetic disease. Hum Genet 2011; 130: 583-605.
    • (2011) Hum Genet , vol.130 , pp. 583-605
    • Seyhan, A.A.1


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.