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Volumn 49, Issue 47, 2010, Pages 10166-10178

Allele-selective inhibition of mutant huntingtin expression with antisense oligonucleotides targeting the expanded CAG repeat

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDES; COOPERATIVE BINDING; HUNTINGTIN; HUNTINGTON'S DISEASE; IN-CELL; IN-VITRO; MUTANT HUNTINGTIN; NEURODEGENERATIVE DISEASE; PHOSPHOROTHIOATES; RIBONUCLEASE H; SELECTIVE INHIBITION;

EID: 78649379362     PISSN: 00062960     EISSN: 15204995     Source Type: Journal    
DOI: 10.1021/bi101208k     Document Type: Article
Times cited : (120)

References (77)
  • 1
    • 34547692622 scopus 로고    scopus 로고
    • Trinucleotide repeat disorders
    • Orr, H. T. and Zoghbi, H. Y. (2007) Trinucleotide repeat disorders Annu. Rev. Neurosci. 30, 575-621
    • (2007) Annu. Rev. Neurosci. , vol.30 , pp. 575-621
    • Orr, H.T.1    Zoghbi, H.Y.2
  • 2
    • 33846225133 scopus 로고    scopus 로고
    • Huntington's disease
    • Walker, F. O. (2007) Huntington's disease Lancet 369, 218-228
    • (2007) Lancet , vol.369 , pp. 218-228
    • Walker, F.O.1
  • 4
    • 33846214868 scopus 로고    scopus 로고
    • Molecular biology of Huntington's disease (HD) and HD-like disorders
    • in (, Ed.), pp, California Academic Press, Millbrae CA
    • Rubinsztein, D. C. (2003) Molecular biology of Huntington's disease (HD) and HD-like disorders., in Genetics of Movement Disorders (Pulst, S., Ed.), pp 365 - 377, California Academic Press, Millbrae CA.
    • (2003) Genetics of Movement Disorders , pp. 365-377
    • Rubinsztein, D.C.1    Pulst, S.2
  • 5
    • 0029997090 scopus 로고    scopus 로고
    • Phenotypic characterization of individuals with 30-40 CAG repeats in the Huntington disease (HD) gene reveals HD cases with 36 repeats and apparently normal elderly individuals with 36-39 repeats
    • Rubinsztein, D. C., Leggo, J., Coles, R., Almqvist, E., Biancalana, V., Cassiman, J. J., Chotai, K., Connarty, M., Crauford, D., and Curtis, A. 1996, Phenotypic characterization of individuals with 30-40 CAG repeats in the Huntington disease (HD) gene reveals HD cases with 36 repeats and apparently normal elderly individuals with 36-39 repeats Am. J. Hum. Genet. 59, 16-22
    • (1996) Am. J. Hum. Genet. , vol.59 , pp. 16-22
    • Rubinsztein, D.C.1    Leggo, J.2    Coles, R.3    Almqvist, E.4    Biancalana, V.5    Cassiman, J.J.6    Chotai, K.7    Connarty, M.8    Crauford, D.9    Curtis, A.10
  • 9
    • 3142679404 scopus 로고    scopus 로고
    • Huntington's disease: How does huntingtin, an anti-apoptotic protein, become toxic?
    • Rangone, H., Humbert, S., and Saudou, F. (2004) Huntington's disease: how does huntingtin, an anti-apoptotic protein, become toxic? Pathol. Biol. 52, 338-342
    • (2004) Pathol. Biol. , vol.52 , pp. 338-342
    • Rangone, H.1    Humbert, S.2    Saudou, F.3
  • 10
    • 77949902558 scopus 로고    scopus 로고
    • Collective roles of molecular chaperones in protein degradation pathways associated with neurodegenerative diseases
    • Luo, G. R. and Le, W. D. (2010) Collective roles of molecular chaperones in protein degradation pathways associated with neurodegenerative diseases Curr. Pharm. Biotechnol. 11, 180-187
    • (2010) Curr. Pharm. Biotechnol. , vol.11 , pp. 180-187
    • Luo, G.R.1    Le, W.D.2
  • 11
    • 3142514201 scopus 로고    scopus 로고
    • Protein aggregation and neurodegenerative disease
    • Ross, C. A. and Poirier, M. A. (2004) Protein aggregation and neurodegenerative disease Nature Med. 10, S10-S17
    • (2004) Nature Med. , vol.10
    • Ross, C.A.1    Poirier, M.A.2
  • 13
    • 0023673267 scopus 로고
    • Treatment of Huntington's disease with clonazepam
    • Stewart, J. T. (1988) Treatment of Huntington's disease with clonazepam South Med. J. 81, 102
    • (1988) South Med. J. , vol.81 , pp. 102
    • Stewart, J.T.1
  • 15
    • 39249084415 scopus 로고    scopus 로고
    • The antidepressant sertraline improves the phenotype, promotes neurogenesis and increases BDNF levels in the R6/2 Huntington's disease mouse model
    • Peng, Q., Masuda, N., Jiang, M., Li, Q., Zhao, M., Ross, C. A., and Duan, W. (2008) The antidepressant sertraline improves the phenotype, promotes neurogenesis and increases BDNF levels in the R6/2 Huntington's disease mouse model Exp. Neurol. 210, 154-163
    • (2008) Exp. Neurol. , vol.210 , pp. 154-163
    • Peng, Q.1    Masuda, N.2    Jiang, M.3    Li, Q.4    Zhao, M.5    Ross, C.A.6    Duan, W.7
  • 18
    • 20044377268 scopus 로고    scopus 로고
    • Early changes in Huntington's disease patient brains involve alterations in cytoskeletal and synaptic elements
    • DiProspero, N. A., Chen, E. Y., Charles, V., Plomann, M., Kordower, J. H., and Tagle, D. A. (2004) Early changes in Huntington's disease patient brains involve alterations in cytoskeletal and synaptic elements J. Neurocytol. 33, 517-533
    • (2004) J. Neurocytol. , vol.33 , pp. 517-533
    • Diprospero, N.A.1    Chen, E.Y.2    Charles, V.3    Plomann, M.4    Kordower, J.H.5    Tagle, D.A.6
  • 19
    • 17844378486 scopus 로고    scopus 로고
    • Axonal transport failure in neurodegenerative disorders: The case of Huntington's disease
    • Charrin, B. C., Saudou, F., and Humbert, S. (2005) Axonal transport failure in neurodegenerative disorders: the case of Huntington's disease Pathol. Biol. 53, 189-192
    • (2005) Pathol. Biol. , vol.53 , pp. 189-192
    • Charrin, B.C.1    Saudou, F.2    Humbert, S.3
  • 20
    • 69949170793 scopus 로고    scopus 로고
    • The pathogenic mechanisms of polyglutamine diseases and current therapeutic strategies
    • Bauer, P. O. and Nukina, N. (2009) The pathogenic mechanisms of polyglutamine diseases and current therapeutic strategies J. Neurochem. 110, 1737-1765
    • (2009) J. Neurochem. , vol.110 , pp. 1737-1765
    • Bauer, P.O.1    Nukina, N.2
  • 21
    • 77950216339 scopus 로고    scopus 로고
    • HD Therapeutics-CHDI Fifth Annual Conference
    • Gagnon, K. T. (2010) HD Therapeutics-CHDI Fifth Annual Conference, IDrugs 13, 219 - 223.
    • (2010) IDrugs , vol.13 , pp. 219-223
    • Gagnon, K.T.1
  • 22
    • 0029055717 scopus 로고
    • Targeted disruption of the Huntington's disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes
    • Nasir, J., Floresco, S. B., O'Kusky, J. R., Diewert, V. M., Richman, J. M., Zeisler, J., Borowski, A., Marth, J. D., Phillips, A. G., and Hayden, M. R. (1995) Targeted disruption of the Huntington's disease gene results in embryonic lethality and behavioral and morphological changes in heterozygotes Cell 81, 811-823
    • (1995) Cell , vol.81 , pp. 811-823
    • Nasir, J.1    Floresco, S.B.2    O'Kusky, J.R.3    Diewert, V.M.4    Richman, J.M.5    Zeisler, J.6    Borowski, A.7    Marth, J.D.8    Phillips, A.G.9    Hayden, M.R.10
  • 26
    • 72449187177 scopus 로고    scopus 로고
    • Therapeutic gene silencing strategies for polyglutamine disorders
    • Scholefield, J. and Wood, M. J. (2010) Therapeutic gene silencing strategies for polyglutamine disorders Trends Genet. 26, 29-38
    • (2010) Trends Genet. , vol.26 , pp. 29-38
    • Scholefield, J.1    Wood, M.J.2
  • 28
    • 47549105506 scopus 로고    scopus 로고
    • Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts
    • van Bilsen, P. H., Jaspers, L., Lombardi, M. S., Odekerken, J. C., Burright, E. N., and Kaemmerer, W. F. (2008) Identification and allele-specific silencing of the mutant huntingtin allele in Huntington's disease patient-derived fibroblasts Hum. Gene Ther. 19, 710-718
    • (2008) Hum. Gene Ther. , vol.19 , pp. 710-718
    • Van Bilsen, P.H.1    Jaspers, L.2    Lombardi, M.S.3    Odekerken, J.C.4    Burright, E.N.5    Kaemmerer, W.F.6
  • 29
    • 33645160450 scopus 로고    scopus 로고
    • Allele-specific RNA interference for neurological disease
    • Rodriguez-Lebron, E. and Paulson, H. L. (2006) Allele-specific RNA interference for neurological disease Gene Ther. 13, 576-581
    • (2006) Gene Ther. , vol.13 , pp. 576-581
    • Rodriguez-Lebron, E.1    Paulson, H.L.2
  • 30
    • 57249086448 scopus 로고    scopus 로고
    • Allele-specific silencing of mutant Huntington's disease gene
    • Zhang, Y., Engelman, J., and Friedlander, R. M. (2009) Allele-specific silencing of mutant Huntington's disease gene J. Neurochem. 108, 82-90
    • (2009) J. Neurochem. , vol.108 , pp. 82-90
    • Zhang, Y.1    Engelman, J.2    Friedlander, R.M.3
  • 34
    • 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., and Corey, D. R. (2009) Allele-selective inhibition of mutant huntingtin by peptide nucleic acid-peptide conjugates, locked nucleic acid, and small interfering RNA Ann. N.Y. Acad. Sci. 1175, 24-31
    • (2009) Ann. N.Y. Acad. Sci. , vol.1175 , pp. 24-31
    • Hu, J.1    Matsui, M.2    Corey, D.R.3
  • 35
    • 71749087536 scopus 로고    scopus 로고
    • Cellular localization and allele-selective inhibition of mutant huntingtin protein by peptide nucleic acid oligomers containing the fluorescent nucleobase [bis- o -(aminoethoxy)phenyl]pyrrolocytosine
    • Hu, J., Dodd, D. W., Hudson, R. H., and Corey, D. R. (2009) Cellular localization and allele-selective inhibition of mutant huntingtin protein by peptide nucleic acid oligomers containing the fluorescent nucleobase [bis- o -(aminoethoxy)phenyl]pyrrolocytosine Bioorg. Med. Chem. Lett. 19, 6181-6184
    • (2009) Bioorg. Med. Chem. Lett. , vol.19 , pp. 6181-6184
    • Hu, J.1    Dodd, D.W.2    Hudson, R.H.3    Corey, D.R.4
  • 36
    • 67650828361 scopus 로고    scopus 로고
    • Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA
    • Wheeler, T. M., Sobczak, K., Lueck, J. D., Osborne, R. J., Lin, X., Dirksen, R. T., and Thornton, C. A. (2009) Reversal of RNA dominance by displacement of protein sequestered on triplet repeat RNA Science 325, 336-339
    • (2009) Science , vol.325 , pp. 336-339
    • Wheeler, T.M.1    Sobczak, K.2    Lueck, J.D.3    Osborne, R.J.4    Lin, X.5    Dirksen, R.T.6    Thornton, C.A.7
  • 37
    • 36849094017 scopus 로고    scopus 로고
    • Chemical modification: The key to clinical application of RNA interference?
    • Corey, D. R. (2007) Chemical modification: the key to clinical application of RNA interference? J. Clin. Invest. 117, 3615-3622
    • (2007) J. Clin. Invest. , vol.117 , pp. 3615-3622
    • Corey, D.R.1
  • 39
    • 77949485460 scopus 로고    scopus 로고
    • Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: A randomised, double-blind, placebo-controlled trial
    • Raal, F. J., Santos, R. D., Blom, D. J., Marais, A. D., Charng, M. J., Cromwell, W. C., Lachmann, R. H., Gaudet, D., Tan, J. L., and Chasan-Taber, S. 2010, Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial Lancet 375, 998-1006
    • (2010) Lancet , vol.375 , pp. 998-1006
    • Raal, F.J.1    Santos, R.D.2    Blom, D.J.3    Marais, A.D.4    Charng, M.J.5    Cromwell, W.C.6    Lachmann, R.H.7    Gaudet, D.8    Tan, J.L.9    Chasan-Taber, S.10
  • 40
    • 77951069859 scopus 로고    scopus 로고
    • Effect of mipomersen, an apolipoprotein B synthesis inhibitor, on low-density lipoprotein cholesterol in patients with familial hypercholesterolemia
    • Akdim, F., Visser, M. E., Tribble, D. L., Baker, B. F., Stroes, E. S., Yu, R., Flaim, J. D., Su, J., Stein, E. A., and Kastelein, J. J. (2010) Effect of mipomersen, an apolipoprotein B synthesis inhibitor, on low-density lipoprotein cholesterol in patients with familial hypercholesterolemia Am. J. Cardiol. 105, 1413-1419
    • (2010) Am. J. Cardiol. , vol.105 , pp. 1413-1419
    • Akdim, F.1    Visser, M.E.2    Tribble, D.L.3    Baker, B.F.4    Stroes, E.S.5    Yu, R.6    Flaim, J.D.7    Su, J.8    Stein, E.A.9    Kastelein, J.J.10
  • 41
    • 77950332314 scopus 로고    scopus 로고
    • Efficacy and safety of mipomersen, an antisense inhibitor of apolipoprotein B, in hypercholesterolemic subjects receiving stable statin therapy
    • Akdim, F., Stroes, E. S., Sijbrands, E. J., Tribble, D. L., Trip, M. D., Jukema, J. W., Flaim, J. D., Su, J., Yu, R., and Baker, B. F. 2010, Efficacy and safety of mipomersen, an antisense inhibitor of apolipoprotein B, in hypercholesterolemic subjects receiving stable statin therapy J. Am. Coll. Cardiol. 55, 1611-1618
    • (2010) J. Am. Coll. Cardiol. , vol.55 , pp. 1611-1618
    • Akdim, F.1    Stroes, E.S.2    Sijbrands, E.J.3    Tribble, D.L.4    Trip, M.D.5    Jukema, J.W.6    Flaim, J.D.7    Su, J.8    Yu, R.9    Baker, B.F.10
  • 42
  • 43
    • 0032560835 scopus 로고    scopus 로고
    • Stability and structural features of the duplexes containing nucleoside analogues with a fixed N-type conformation, 2′- O,4′- C -methyleneribonucleosides
    • Obika, S., Nanbu, D., Hari, Y., Andoh, J., Morio, K., Doi, T., and Imanishi, T. (1998) Stability and structural features of the duplexes containing nucleoside analogues with a fixed N-type conformation, 2′- O,4′- C -methyleneribonucleosides Tetrahedron Lett. 39, 5401-5404
    • (1998) Tetrahedron Lett. , vol.39 , pp. 5401-5404
    • Obika, S.1    Nanbu, D.2    Hari, Y.3    Andoh, J.4    Morio, K.5    Doi, T.6    Imanishi, T.7
  • 44
    • 0032473890 scopus 로고    scopus 로고
    • LNA (Locked Nucleic Acids): Synthesis of the adenine, cytosine, guanine, 5-methylcytosine, thymine and uracil bicyclonucleoside monomers, oligomerisation, and unprecedented nucleic acid recognition
    • Koshkin, A. A., Singh, S. K., Nielsen, P., Rajwanshi, V. K., Kumar, R., Meldgaard, M., Olsen, C. E., and Wengel, J. (1998) LNA (Locked Nucleic Acids): Synthesis of the adenine, cytosine, guanine, 5-methylcytosine, thymine and uracil bicyclonucleoside monomers, oligomerisation, and unprecedented nucleic acid recognition Tetrahedron 54, 3607-3630
    • (1998) Tetrahedron , vol.54 , pp. 3607-3630
    • Koshkin, A.A.1    Singh, S.K.2    Nielsen, P.3    Rajwanshi, V.K.4    Kumar, R.5    Meldgaard, M.6    Olsen, C.E.7    Wengel, J.8
  • 45
    • 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
    • Braasch, D. A., Liu, Y., and Corey, D. R. (2002) Antisense inhibition of gene expression in cells by oligonucleotides incorporating locked nucleic acids: effect of mRNA target sequence and chimera design Nucleic Acids Res. 30, 5160-5167
    • (2002) Nucleic Acids Res. , vol.30 , pp. 5160-5167
    • Braasch, D.A.1    Liu, Y.2    Corey, D.R.3
  • 46
    • 33745012353 scopus 로고    scopus 로고
    • Thermodynamic, Counterion, and Hydration Effects for the Incorporation of Locked Nucleic Acid Nucleotides into DNA Duplexes
    • Kaur, H., Arora, A., Wengel, J., Maiti, S., Arora, A., Wengel, J., and Maiti, S. (2006) Thermodynamic, Counterion, and Hydration Effects for the Incorporation of Locked Nucleic Acid Nucleotides into DNA Duplexes Biochemistry 45, 7347-7355
    • (2006) Biochemistry , vol.45 , pp. 7347-7355
    • Kaur, H.1    Arora, A.2    Wengel, J.3    Maiti, S.4    Arora, A.5    Wengel, J.6    Maiti, S.7
  • 47
    • 0037033135 scopus 로고    scopus 로고
    • 2′- O,4′- C -ethylene-bridged nucleic acids (ENA): Highly nuclease-resistant and thermodynamically stable oligonucleotides for antisense drug
    • Morita, K., Hasegawa, C., Kaneko, M., Tsutsumi, S., Sone, J., Ishikawa, T., Imanishi, T., and Koizumi, M. (2002) 2′- O,4′- C -ethylene-bridged nucleic acids (ENA): highly nuclease-resistant and thermodynamically stable oligonucleotides for antisense drug Bioorg. Med. Chem. Lett. 12, 73-76
    • (2002) Bioorg. Med. Chem. Lett. , vol.12 , pp. 73-76
    • Morita, K.1    Hasegawa, C.2    Kaneko, M.3    Tsutsumi, S.4    Sone, J.5    Ishikawa, T.6    Imanishi, T.7    Koizumi, M.8
  • 48
    • 0037901806 scopus 로고    scopus 로고
    • Triplex formation with 2′- O,4′- C -ethylene-bridged nucleic acids (ENA) having C 3′-endo conformation at physiological pH
    • Koizumi, M., Morita, K., Daigo, M., Tsutsumu, S., Abe, K., Obika, S., and Imanishi, T. (2003) Triplex formation with 2′- O,4′- C -ethylene-bridged nucleic acids (ENA) having C 3′-endo conformation at physiological pH Nucleic Acids Res. 31, 3267-3273
    • (2003) Nucleic Acids Res. , vol.31 , pp. 3267-3273
    • Koizumi, M.1    Morita, K.2    Daigo, M.3    Tsutsumu, S.4    Abe, K.5    Obika, S.6    Imanishi, T.7
  • 49
    • 59449093219 scopus 로고    scopus 로고
    • Short antisense oligonucleotides with novel 2′-4′ conformationaly restricted nucleoside analogues show improved potency without increased toxicity in animals
    • Seth, P. P., Siwkowski, A., Allerson, C. R., Vasquez, G., Lee, S., Prakash, T. P., Wancewicz, E. V., Witchell, D., and Swayze, E. E. (2009) Short antisense oligonucleotides with novel 2′-4′ conformationaly restricted nucleoside analogues show improved potency without increased toxicity in animals J. Med. Chem. 52, 10-13
    • (2009) J. Med. Chem. , vol.52 , pp. 10-13
    • Seth, P.P.1    Siwkowski, A.2    Allerson, C.R.3    Vasquez, G.4    Lee, S.5    Prakash, T.P.6    Wancewicz, E.V.7    Witchell, D.8    Swayze, E.E.9
  • 50
    • 77649230488 scopus 로고    scopus 로고
    • Antisense oligonucleotides containing conformationally constrained 2′,4′-(N -methoxy)aminomethylene and 2′,4′- aminooxymethylene and 2′- O,4′- C -aminomethylene bridged nucleoside analogues show improved potency in animal models
    • Prakash, T. P., Siwkowski, A., Allerson, C. R., Migawa, M. T., Lee, S., Gaus, H. J., Black, C., Seth, P. P., Swayze, E. E., and Bhat, B. (2010) Antisense oligonucleotides containing conformationally constrained 2′,4′-(N -methoxy)aminomethylene and 2′,4′- aminooxymethylene and 2′- O,4′- C -aminomethylene bridged nucleoside analogues show improved potency in animal models J. Med. Chem. 53, 1636-1650
    • (2010) J. Med. Chem. , vol.53 , pp. 1636-1650
    • Prakash, T.P.1    Siwkowski, A.2    Allerson, C.R.3    Migawa, M.T.4    Lee, S.5    Gaus, H.J.6    Black, C.7    Seth, P.P.8    Swayze, E.E.9    Bhat, B.10
  • 51
    • 34447129727 scopus 로고    scopus 로고
    • Five- and six-membered conformationally locked 2′,4′- carbocyclic ribo-thymidines: Synthesis, structure, and biochemical studies
    • Srivastava, P., Barman, J., Pathmasiri, W., Plashkevych, O., Wenska, M., and Chattopadhyaya, J. (2007) Five- and six-membered conformationally locked 2′,4′-carbocyclic ribo-thymidines: synthesis, structure, and biochemical studies J. Am. Chem. Soc. 129, 8362-8379
    • (2007) J. Am. Chem. Soc. , vol.129 , pp. 8362-8379
    • Srivastava, P.1    Barman, J.2    Pathmasiri, W.3    Plashkevych, O.4    Wenska, M.5    Chattopadhyaya, J.6
  • 54
    • 0027479864 scopus 로고
    • Uniformly modified 2′-deoxy-2′-fluoro phosphorothioate oligonucleotides as nuclease-resistant antisense compounds with high affinity and specificity for RNA targets
    • Kawasaki, A. M., Casper, M. D., Freier, S. M., Lesnik, E. A., Zounes, M. C., Cummins, L. L., Gonzalez, C., and Cook, P. D. (1993) Uniformly modified 2′-deoxy-2′-fluoro phosphorothioate oligonucleotides as nuclease-resistant antisense compounds with high affinity and specificity for RNA targets J. Med. Chem. 36, 831-841
    • (1993) J. Med. Chem. , vol.36 , pp. 831-841
    • Kawasaki, A.M.1    Casper, M.D.2    Freier, S.M.3    Lesnik, E.A.4    Zounes, M.C.5    Cummins, L.L.6    Gonzalez, C.7    Cook, P.D.8
  • 55
    • 46249131334 scopus 로고    scopus 로고
    • 2′F-Arabinonucleic acids (2′F-ANA)-History, properties, and new frontiers
    • Watts, J. K. and Damha, M. J. (2008) 2′F-Arabinonucleic acids (2′F-ANA)-History, properties, and new frontiers Can. J. Chem. 86, 641-656
    • (2008) Can. J. Chem. , vol.86 , pp. 641-656
    • Watts, J.K.1    Damha, M.J.2
  • 56
    • 0032539186 scopus 로고    scopus 로고
    • Hybrids of RNA and arabinonucleic acids (ANA and 2′F-ANA) are substrates of ribonuclease H
    • Damha, M. J., Wilds, C. J., Noronha, A., Brukner, I., Borkow, G., Arion, D., and Parniak, M. A. (1998) Hybrids of RNA and arabinonucleic acids (ANA and 2′F-ANA) are substrates of ribonuclease H J. Am. Chem. Soc. 120, 12976-12977
    • (1998) J. Am. Chem. Soc. , vol.120 , pp. 12976-12977
    • Damha, M.J.1    Wilds, C.J.2    Noronha, A.3    Brukner, I.4    Borkow, G.5    Arion, D.6    Parniak, M.A.7
  • 58
    • 23844465175 scopus 로고    scopus 로고
    • Thermodynamic stability of RNA structures formed by CNG trinucleotide repeats. Implication for prediction of RNA structure
    • Broda, M., Kierzek, E., Gdaniec, Z., Kulinski, T., and Kierzek, R. (2005) Thermodynamic stability of RNA structures formed by CNG trinucleotide repeats. Implication for prediction of RNA structure Biochemistry 44, 10873-10882
    • (2005) Biochemistry , vol.44 , pp. 10873-10882
    • Broda, M.1    Kierzek, E.2    Gdaniec, Z.3    Kulinski, T.4    Kierzek, R.5
  • 60
    • 0033572750 scopus 로고    scopus 로고
    • Thermal stability of PNA/DNA and DNA/DNA duplexes by differential scanning calorimetry
    • Chakrabarti, M. C. and Schwarz, F. P. (1999) Thermal stability of PNA/DNA and DNA/DNA duplexes by differential scanning calorimetry Nucleic Acids Res. 27, 4801-4806
    • (1999) Nucleic Acids Res. , vol.27 , pp. 4801-4806
    • Chakrabarti, M.C.1    Schwarz, F.P.2
  • 61
    • 38549149739 scopus 로고    scopus 로고
    • Thermodynamics of DNA-RNA heteroduplex formation: Effects of locked nucleic acid nucleotides incorporated into the DNA strand
    • Kaur, H., Wengel, J., and Maiti, S. (2008) Thermodynamics of DNA-RNA heteroduplex formation: effects of locked nucleic acid nucleotides incorporated into the DNA strand Biochemistry 47, 1218-1227
    • (2008) Biochemistry , vol.47 , pp. 1218-1227
    • Kaur, H.1    Wengel, J.2    Maiti, S.3
  • 63
    • 27744483960 scopus 로고    scopus 로고
    • SiRNA-mediated inhibition of endogenous Huntington disease gene expression induces an aberrant configuration of the ER network in vitro
    • Omi, K., Hachiya, N. S., Tokunaga, K., and Kaneko, K. (2005) siRNA-mediated inhibition of endogenous Huntington disease gene expression induces an aberrant configuration of the ER network in vitro Biochem. Biophys. Res. Commun. 338, 1229-1235
    • (2005) Biochem. Biophys. Res. Commun. , vol.338 , pp. 1229-1235
    • Omi, K.1    Hachiya, N.S.2    Tokunaga, K.3    Kaneko, K.4
  • 64
    • 0033564415 scopus 로고    scopus 로고
    • Analysis of the cooperative thermal unfolding of the td intron of bacteriophage T4
    • Brion, P., Michel, F., Schroeder, R., and Westhof, E. (1999) Analysis of the cooperative thermal unfolding of the td intron of bacteriophage T4 Nucleic Acids Res. 27, 2494-2502
    • (1999) Nucleic Acids Res. , vol.27 , pp. 2494-2502
    • Brion, P.1    Michel, F.2    Schroeder, R.3    Westhof, E.4
  • 65
    • 1542303744 scopus 로고    scopus 로고
    • Analysis of thermal melting curves
    • Mergny, J.-L. and Lacroix, L. (2003) Analysis of thermal melting curves Oligonucleotides 13, 515-537
    • (2003) Oligonucleotides , vol.13 , pp. 515-537
    • Mergny, J.-L.1    Lacroix, L.2
  • 66
    • 0032078424 scopus 로고    scopus 로고
    • The effect of two antipodal fluorine-induced sugar puckers on the conformation and stability of the Dickerson-Drew dodecamer duplex [d(CGCGAATTCGCG)]2
    • Ikeda, H., Fernandez, R., Wilk, A., Barchi, J. J., Jr., Huang, X., and Marquez, V. E. (1998) The effect of two antipodal fluorine-induced sugar puckers on the conformation and stability of the Dickerson-Drew dodecamer duplex [d(CGCGAATTCGCG)]2 Nucleic Acids Res. 26, 2237-2244
    • (1998) Nucleic Acids Res. , vol.26 , pp. 2237-2244
    • Ikeda, H.1    Fernandez, R.2    Wilk, A.3    Barchi, Jr.J.J.4    Huang, X.5    Marquez, V.E.6
  • 67
    • 0037460131 scopus 로고    scopus 로고
    • Efficient RNase H-directed cleavage of RNA promoted by antisense DNA or 2′F-ANA constructs containing acyclic nucleotide inserts
    • Mangos, M. M., Min, K.-L., Viazovkina, E., Galarneau, A., Elzagheid, M. I., Parniak, M. A., and Damha, M. J. (2002) Efficient RNase H-directed cleavage of RNA promoted by antisense DNA or 2′F-ANA constructs containing acyclic nucleotide inserts J. Am. Chem. Soc. 125, 654-661
    • (2002) J. Am. Chem. Soc. , vol.125 , pp. 654-661
    • Mangos, M.M.1    Min, K.-L.2    Viazovkina, E.3    Galarneau, A.4    Elzagheid, M.I.5    Parniak, M.A.6    Damha, M.J.7
  • 68
    • 33751430285 scopus 로고    scopus 로고
    • LNA-modified oligonucleotides effectively drive intramolecular-stable hairpin to intermolecular-duplex state
    • Kaur, H., Wengel, J., and Maiti, S. (2007) LNA-modified oligonucleotides effectively drive intramolecular-stable hairpin to intermolecular-duplex state Biochem. Biophys. Res. Commun. 352, 118-122
    • (2007) Biochem. Biophys. Res. Commun. , vol.352 , pp. 118-122
    • Kaur, H.1    Wengel, J.2    Maiti, S.3
  • 69
    • 1542269161 scopus 로고    scopus 로고
    • Progress in antisense technology
    • Crooke, S. T. (2004) Progress in antisense technology Annu. Rev. Med. 55, 61-95
    • (2004) Annu. Rev. Med. , vol.55 , pp. 61-95
    • Crooke, S.T.1
  • 70
    • 0033045917 scopus 로고    scopus 로고
    • Correlation of Toxicity and Pharmacokinetic Properties of a Phosphorothioate Oligonucleotide Designed to Inhibit ICAM-1
    • Henry, S. P., Templin, M. V., Gillett, N., Rojko, J., and Levin, A. A. (1999) Correlation of Toxicity and Pharmacokinetic Properties of a Phosphorothioate Oligonucleotide Designed to Inhibit ICAM-1 Toxicol. Pathol. 27, 95-100
    • (1999) Toxicol. Pathol. , vol.27 , pp. 95-100
    • Henry, S.P.1    Templin, M.V.2    Gillett, N.3    Rojko, J.4    Levin, A.A.5
  • 71
    • 79959624824 scopus 로고    scopus 로고
    • Preclinical toxicity and toxicokinetics of GTI-2040, a phosphorothioate oligonucleotide targeting ribonucleotide reductase R2
    • doi: 10.1007/s00280-010-1473-z
    • Ferdinandi, E. S., Vassilakos, A., Lee, Y., Lightfoot, J., Fitsialos, D., Wright, J. A., and Young, A. H. (2010) Preclinical toxicity and toxicokinetics of GTI-2040, a phosphorothioate oligonucleotide targeting ribonucleotide reductase R2, Cancer Chemother. Pharmacol. 66, doi: 10.1007/s00280-010-1473-z.
    • (2010) Cancer Chemother. Pharmacol. , vol.66
    • Ferdinandi, E.S.1    Vassilakos, A.2    Lee, Y.3    Lightfoot, J.4    Fitsialos, D.5    Wright, J.A.6    Young, A.H.7
  • 73
    • 3042561985 scopus 로고    scopus 로고
    • Molecular architecture of CAG repeats in human disease related transcripts
    • Michlewski, G. and Krzyzosiak, W. J. (2004) Molecular architecture of CAG repeats in human disease related transcripts J. Mol. Biol. 340, 665-679
    • (2004) J. Mol. Biol. , vol.340 , pp. 665-679
    • Michlewski, G.1    Krzyzosiak, W.J.2
  • 74
    • 77955894067 scopus 로고    scopus 로고
    • Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model
    • Hua, Y., Sahashi, K., Hung, G., Rigo, F., Passini, M. A., Bennett, C. F., and Krainer, A. R. (2010) Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model Genes Dev. 24, 1634-1644
    • (2010) Genes Dev. , vol.24 , pp. 1634-1644
    • Hua, Y.1    Sahashi, K.2    Hung, G.3    Rigo, F.4    Passini, M.A.5    Bennett, C.F.6    Krainer, A.R.7
  • 76
    • 52049090811 scopus 로고    scopus 로고
    • Chemically modified siRNA: Tools and applications
    • Watts, J. K., Deleavey, G. F., and Damha, M. J. (2008) Chemically modified siRNA: tools and applications Drug Discovery Today 13, 842-855
    • (2008) Drug Discovery Today , vol.13 , pp. 842-855
    • Watts, J.K.1    Deleavey, G.F.2    Damha, M.J.3


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