메뉴 건너뛰기




Volumn 41, Issue 21, 2013, Pages 9634-9650

Rational design of antisense oligonucleotides targeting single nucleotide polymorphisms for potent and allele selective suppression of mutant Huntingtin in the CNS

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; HUNTINGTIN; MESSENGER RNA; MUTANT PROTEIN; RIBONUCLEASE H;

EID: 84890057093     PISSN: 03051048     EISSN: 13624962     Source Type: Journal    
DOI: 10.1093/nar/gkt725     Document Type: Article
Times cited : (129)

References (49)
  • 1
    • 25844487226 scopus 로고    scopus 로고
    • Diseases of unstable repeat expansion: Mechanisms and common principles
    • Gatchel, J.R. and Zoghbi, H.Y. (2005) Diseases of unstable repeat expansion: mechanisms and common principles. Nat. Rev. Genet., 6, 743-755.
    • (2005) Nat. Rev. Genet. , vol.6 , pp. 743-755
    • Gatchel, J.R.1    Zoghbi, H.Y.2
  • 2
    • 77949512140 scopus 로고    scopus 로고
    • RNA targeting therapeutics: Molecular mechanisms of antisense oligonucleotides as a therapeutic platform
    • Bennett, C.F. and Swayze, E.E. (2010) RNA targeting therapeutics: molecular mechanisms of antisense oligonucleotides as a therapeutic platform. Annu. Rev. Pharmacol. Toxicol., 50, 259-293.
    • (2010) Annu. Rev. Pharmacol. Toxicol. , vol.50 , pp. 259-293
    • Bennett, C.F.1    Swayze, E.E.2
  • 3
    • 33645127443 scopus 로고    scopus 로고
    • Genetic medicines: Treatment strategies for hereditary disorders
    • O'Connor, T.P. and Crystal, R.G. (2006) Genetic medicines: treatment strategies for hereditary disorders. Nat. Rev. Genet., 7, 261-276.
    • (2006) Nat. Rev. Genet. , vol.7 , pp. 261-276
    • O'Connor, T.P.1    Crystal, R.G.2
  • 5
    • 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
  • 6
    • 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
  • 9
    • 84993912315 scopus 로고
    • Increased apoptosis and early embryonic lethality in mice nullizygous for the Huntington's disease gene homologue
    • Zeitlin, S., Liu, J.P., Chapman, D.L., Papaioannou, V.E. and Efstratiadis, A. (1995) Increased apoptosis and early embryonic lethality in mice nullizygous for the Huntington's disease gene homologue. Nat. Genet., 11, 155-163.
    • (1995) Nat. Genet. , vol.11 , pp. 155-163
    • Zeitlin, S.1    Liu, J.P.2    Chapman, D.L.3    Papaioannou, V.E.4    Efstratiadis, A.5
  • 12
    • 82955199935 scopus 로고    scopus 로고
    • Preclinical safety of RNAimediated HTT suppression in the rhesus macaque as a potential therapy for huntington's disease
    • McBride, J.L., Pitzer, M.R., Boudreau, R.L., Dufour, B., Hobbs, T., Ojeda, S.R. and Davidson, B.L. (2011) Preclinical safety of RNAimediated HTT suppression in the rhesus macaque as a potential therapy for huntington's disease. Mol. Ther., 19, 2152-2162.
    • (2011) Mol. Ther. , vol.19 , pp. 2152-2162
    • McBride, J.L.1    Pitzer, M.R.2    Boudreau, R.L.3    Dufour, B.4    Hobbs, T.5    Ojeda, S.R.6    Davidson, B.L.7
  • 13
    • 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, R.L., Spengler, R.M. and Davidson, B.L. (2011) Rational design of therapeutic siRNAs: minimizing off-targeting potential to improve the safety of RNAi therapy for Huntington's disease. Mol. Ther., 19, 2169-2177.
    • (2011) Mol. Ther. , vol.19 , pp. 2169-2177
    • Boudreau, R.L.1    Spengler, R.M.2    Davidson, B.L.3
  • 15
    • 78649359013 scopus 로고    scopus 로고
    • Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism
    • Hu, J., Liu, J. and Corey, D.R. (2010) Allele-selective inhibition of huntingtin expression by switching to an miRNA-like RNAi mechanism. Chem. Biol., 17, 1183-1188.
    • (2010) Chem. Biol. , vol.17 , pp. 1183-1188
    • Hu, J.1    Liu, J.2    Corey, D.R.3
  • 18
    • 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, 710-719.
    • (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
  • 21
    • 0026774939 scopus 로고
    • Selective inhibition of mutant Ha-ras mRNA expression by antisense oligonucleotides
    • Monia, B.P., Johnston, J.F., Ecker, D.J., Zounes, M.A., Lima, W.F. and Freier, S.M. (1992) Selective inhibition of mutant Ha-ras mRNA expression by antisense oligonucleotides. J. Biol. Chem., 267, 19954-19962.
    • (1992) J. Biol. Chem. , vol.267 , pp. 19954-19962
    • Monia, B.P.1    Johnston, J.F.2    Ecker, D.J.3    Zounes, M.A.4    Lima, W.F.5    Freier, S.M.6
  • 22
    • 82955237522 scopus 로고    scopus 로고
    • Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene/ allele-specific silencing of mutant huntingtin
    • Carroll, J.B., Warby, S.C., Southwell, A.L., Doty, C.N., Greenlee, S., Skotte, N., Hung, G., Bennett, C.F., Freier, S.M. and Hayden, M.R. (2011) Potent and selective antisense oligonucleotides targeting single-nucleotide polymorphisms in the Huntington disease gene/ allele-specific silencing of mutant huntingtin. Mol. Ther., 19, 2178-2185.
    • (2011) Mol. Ther. , vol.19 , pp. 2178-2185
    • Carroll, J.B.1    Warby, S.C.2    Southwell, A.L.3    Doty, C.N.4    Greenlee, S.5    Skotte, N.6    Hung, G.7    Bennett, C.F.8    Freier, S.M.9    Hayden, M.R.10
  • 25
    • 0037470145 scopus 로고    scopus 로고
    • Efficient reduction of target RNAs by small interfering RNA and RNase H-dependent antisense agents
    • Vickers, T.A., Koo, S., Bennett, C.F., Crooke, S.T., Dean, N.M. and Baker, B.F. (2003) Efficient reduction of target RNAs by small interfering RNA and RNase H-dependent antisense agents. J. Biol. Chem., 278, 7108-7118.
    • (2003) J. Biol. Chem. , vol.278 , pp. 7108-7118
    • Vickers, T.A.1    Koo, S.2    Bennett, C.F.3    Crooke, S.T.4    Dean, N.M.5    Baker, B.F.6
  • 27
    • 84876466100 scopus 로고    scopus 로고
    • An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: A phase 1, randomised, first-in-man study
    • Miller, T.M., Pestronk, A., David, W., Rothstein, J., Simpson, E., Appel, S.H., Andres, P.L., Mahoney, K., Allred, P., Alexander, K. et al. (2013) An antisense oligonucleotide against SOD1 delivered intrathecally for patients with SOD1 familial amyotrophic lateral sclerosis: a phase 1, randomised, first-in-man study. Lancet Neurol., 12, 435-442.
    • (2013) Lancet Neurol. , vol.12 , pp. 435-442
    • Miller, T.M.1    Pestronk, A.2    David, W.3    Rothstein, J.4    Simpson, E.5    Appel, S.H.6    Andres, P.L.7    Mahoney, K.8    Allred, P.9    Alexander, K.10
  • 28
    • 84890098975 scopus 로고    scopus 로고
    • Getting a fix on SMA
    • Dolgin, E. (2012) Getting a fix on SMA. Nat. Med., 18, 1605-1605.
    • (2012) Nat. Med. , vol.18 , pp. 1605-1605
    • Dolgin, E.1
  • 30
    • 59449093219 scopus 로고    scopus 로고
    • Short antisense oligonucleotides with novel 20-40 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 20-40 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
  • 31
    • 79959479529 scopus 로고    scopus 로고
    • Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes
    • Koller, E., Vincent, T.M., Chappell, A., De, S., Manoharan, M. and Bennett, C.F. (2011) Mechanisms of single-stranded phosphorothioate modified antisense oligonucleotide accumulation in hepatocytes. Nucleic Acids Res., 39, 4795-4807.
    • (2011) Nucleic Acids Res. , vol.39 , pp. 4795-4807
    • Koller, E.1    Vincent, T.M.2    Chappell, A.3    De, S.4    Manoharan, M.5    Bennett, C.F.6
  • 32
    • 0034232357 scopus 로고    scopus 로고
    • The G x U wobble base pair. A fundamental building block of RNA structure crucial to RNA function in diverse biological systems
    • Varani, G. and McClain, W.H. (2000) The G x U wobble base pair. A fundamental building block of RNA structure crucial to RNA function in diverse biological systems. EMBO Rep., 1, 18-23.
    • (2000) EMBO Rep. , vol.1 , pp. 18-23
    • Varani, G.1    McClain, W.H.2
  • 33
    • 0035913728 scopus 로고    scopus 로고
    • Long-range cooperativity due to C5-propynylation of oligopyrimidines enhances specific recognition by uridine of ribo-adenosine over ribo-guanosine
    • Barnes, T.W. III and Turner, D.H. (2001) Long-range cooperativity due to C5-propynylation of oligopyrimidines enhances specific recognition by uridine of ribo-adenosine over ribo-guanosine. J. Am. Chem. Soc., 123, 9186-9187.
    • (2001) J. Am. Chem. Soc. , vol.123 , pp. 9186-9187
    • Barnes III, T.W.1    Turner, D.H.2
  • 34
    • 0033554848 scopus 로고    scopus 로고
    • Thermodynamics of RNA-RNA duplexes with 2-or 4-thiouridines: Implications for antisense design and targeting a group i intron
    • Testa, S.M., Disney, M.D., Turner, D.H. and Kierzek, R. (1999) Thermodynamics of RNA-RNA duplexes with 2-or 4-thiouridines: implications for antisense design and targeting a group I intron. Biochemistry, 38, 16655-16662.
    • (1999) Biochemistry , vol.38 , pp. 16655-16662
    • Testa, S.M.1    Disney, M.D.2    Turner, D.H.3    Kierzek, R.4
  • 35
    • 80054756151 scopus 로고    scopus 로고
    • Synthesis, improved antisense activity and structural rationale for the divergent RNA affinities of 30-fluoro hexitol nucleic acid (FHNA and Ara-FHNA) modified oligonucleotides
    • Egli, M., Pallan, P.S., Allerson, C.R., Prakash, T.P., Berdeja, A., Yu, J., Lee, S., Watt, A., Gaus, H., Bhat, B. et al. (2011) Synthesis, improved antisense activity and structural rationale for the divergent RNA affinities of 30-fluoro hexitol nucleic acid (FHNA and Ara-FHNA) modified oligonucleotides. J. Am. Chem. Soc., 133, 16642-16649.
    • (2011) J. Am. Chem. Soc. , vol.133 , pp. 16642-16649
    • Egli, M.1    Pallan, P.S.2    Allerson, C.R.3    Prakash, T.P.4    Berdeja, A.5    Yu, J.6    Lee, S.7    Watt, A.8    Gaus, H.9    Bhat, B.10
  • 36
    • 2342427578 scopus 로고    scopus 로고
    • Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs
    • Wu, H., Lima, W.F., Zhang, H., Fan, A., Sun, H. and Crooke, S.T. (2004) Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs. J. Biol. Chem., 279, 17181-17189.
    • (2004) J. Biol. Chem. , vol.279 , pp. 17181-17189
    • Wu, H.1    Lima, W.F.2    Zhang, H.3    Fan, A.4    Sun, H.5    Crooke, S.T.6
  • 38
    • 35348978302 scopus 로고    scopus 로고
    • Structure of human RNase H1 complexed with an RNA/DNA hybrid: Insight into HIV reverse transcription
    • Nowotny, M., Gaidamakov, S.A., Ghirlando, R., Cerritelli, S.M., Crouch, R.J. and Yang, W. (2007) Structure of human RNase H1 complexed with an RNA/DNA hybrid: insight into HIV reverse transcription. Mol. Cell, 28, 264-276.
    • (2007) Mol. Cell , vol.28 , pp. 264-276
    • Nowotny, M.1    Gaidamakov, S.A.2    Ghirlando, R.3    Cerritelli, S.M.4    Crouch, R.J.5    Yang, W.6
  • 39
    • 0033213981 scopus 로고    scopus 로고
    • Properties of cloned and expressed human RNase H1
    • Wu, H., Lima, W.F. and Crooke, S.T. (1999) Properties of cloned and expressed human RNase H1. J. Biol. Chem., 274, 28270-28278.
    • (1999) J. Biol. Chem. , vol.274 , pp. 28270-28278
    • Wu, H.1    Lima, W.F.2    Crooke, S.T.3
  • 42
    • 33645545684 scopus 로고    scopus 로고
    • 20-fluoroarabino-and arabinonucleic acid show different conformations, resulting in deviating RNA affinities and processing of their heteroduplexes with RNA by RNase H
    • Li, F., Sarkhel, S., Wilds, C.J., Wawrzak, Z., Prakash, T.P., Manoharan, M. and Egli, M. (2006) 20-fluoroarabino-and arabinonucleic acid show different conformations, resulting in deviating RNA affinities and processing of their heteroduplexes with RNA by RNase H. Biochemistry, 45, 4141-4152.
    • (2006) Biochemistry , vol.45 , pp. 4141-4152
    • Li, F.1    Sarkhel, S.2    Wilds, C.J.3    Wawrzak, Z.4    Prakash, T.P.5    Manoharan, M.6    Egli, M.7
  • 43
    • 0025762698 scopus 로고
    • Properties of 2'-fluorothymidine-containing oligonucleotides: Interaction with restriction endonuclease EcoRV
    • Williams, D.M., Benseler, F. and Eckstein, F. (1991) Properties of 2'-fluorothymidine-containing oligonucleotides: interaction with restriction endonuclease EcoRV. Biochemistry, 30, 4001-4009.
    • (1991) Biochemistry , vol.30 , pp. 4001-4009
    • Williams, D.M.1    Benseler, F.2    Eckstein, F.3
  • 44
    • 84864199342 scopus 로고    scopus 로고
    • Structure and nuclease resistance of 20, 40-constrained 20-O-methoxyethyl (cMOE) and 20-O-ethyl (cEt) modified DNAs
    • Pallan, P.S., Allerson, C.R., Berdeja, A., Seth, P.P., Swayze, E.E., Prakash, T.P. and Egli, M. (2012) Structure and nuclease resistance of 20, 40-constrained 20-O-methoxyethyl (cMOE) and 20-O-ethyl (cEt) modified DNAs. Chem. Commun., 48, 8195-8197.
    • (2012) Chem. Commun. , vol.48 , pp. 8195-8197
    • Pallan, P.S.1    Allerson, C.R.2    Berdeja, A.3    Seth, P.P.4    Swayze, E.E.5    Prakash, T.P.6    Egli, M.7
  • 46
    • 77955643169 scopus 로고    scopus 로고
    • Molecular mechanisms and potential therapeutical targets in Huntington's disease
    • Zuccato, C., Valenza, M. and Cattaneo, E. (2010) Molecular mechanisms and potential therapeutical targets in Huntington's disease. Physiol. Rev., 90, 905-981.
    • (2010) Physiol. Rev. , vol.90 , pp. 905-981
    • Zuccato, C.1    Valenza, M.2    Cattaneo, E.3


* 이 정보는 Elsevier사의 SCOPUS DB에서 KISTI가 분석하여 추출한 것입니다.