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Volumn 44, Issue 5, 2015, Pages 2093-2109

Hepatotoxicity of high affinity gapmer antisense oligonucleotides is mediated by RNase H1 dependent promiscuous reduction of very long pre-mRNA transcripts

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; ANTISENSE OLIGONUCLEOTIDE; ASPARTATE AMINOTRANSFERASE; GAP ANTISENSE OLIGONUCLEOTIDE; LOCKED NUCLEIC ACID; MESSENGER RNA; MESSENGER RNA PRECURSOR; RIBONUCLEASE H; UNCLASSIFIED DRUG; OLIGONUCLEOTIDE; RIBONUCLEASE HI; RNA PRECURSOR; SMALL INTERFERING RNA; TRANSCRIPTOME;

EID: 84963812653     PISSN: 03051048     EISSN: 13624962     Source Type: Journal    
DOI: 10.1093/nar/gkv1210     Document Type: Article
Times cited : (141)

References (58)
  • 1
    • 0032989151 scopus 로고    scopus 로고
    • The clinical experience of antisense therapy to ICAM-1 in Crohn's disease
    • Yacyshyn, B., Bowen-Yacyshyn, M.B. and Shanahan, W. (1999) The clinical experience of antisense therapy to ICAM-1 in Crohn's disease. Curr. Opin. Mol. Ther., 1, 332-335.
    • (1999) Curr. Opin. Mol. Ther. , vol.1 , pp. 332-335
    • Yacyshyn, B.1    Bowen-Yacyshyn, M.B.2    Shanahan, W.3
  • 2
    • 84963833347 scopus 로고    scopus 로고
    • Crooke, S.T. (ed.) Taylor & Francis Group, LLC, Boca Raton
    • Bennett, F.C. (2007) In: Crooke, S.T. (ed.), Antisense Drug Technologies, Second Edition. Taylor & Francis Group, LLC, Boca Raton, Vol. 2, pp. 273-303.
    • (2007) Antisense Drug Technologies, Second Edition , vol.2 , pp. 273-303
    • Bennett, F.C.1
  • 5
    • 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
  • 6
    • 67649271530 scopus 로고    scopus 로고
    • Antisense oligonucleotide pharmacokinetics and metabolism
    • Geary, R.S. (2009) Antisense oligonucleotide pharmacokinetics and metabolism. Expert Opin. Drug Metab. Toxicol., 5, 381-391.
    • (2009) Expert Opin. Drug Metab. Toxicol. , vol.5 , pp. 381-391
    • Geary, R.S.1
  • 7
    • 10744226217 scopus 로고    scopus 로고
    • Pharmacokinetics of a tumor necrosis factor-alpha phosphorothioate 2'-O-(2-methoxyethyl) modified antisense oligonucleotide: Comparison across species
    • Geary, R.S., Yu, R.Z., Watanabe, T., Henry, S.P., Hardee, G.E., Chappell, A., Matson, J., Sasmor, H., Cummins, L. and Levin, A.A. (2003) Pharmacokinetics of a tumor necrosis factor-alpha phosphorothioate 2'-O-(2-methoxyethyl) modified antisense oligonucleotide: comparison across species. Drug Metab. Dispos., 31, 1419-1428.
    • (2003) Drug Metab. Dispos. , vol.31 , pp. 1419-1428
    • Geary, R.S.1    Yu, R.Z.2    Watanabe, T.3    Henry, S.P.4    Hardee, G.E.5    Chappell, A.6    Matson, J.7    Sasmor, H.8    Cummins, L.9    Levin, A.A.10
  • 8
    • 0347124829 scopus 로고    scopus 로고
    • Tissue disposition of a 2'-O-(2-methoxy) ethyl modified antisense oligonucleotides in monkeys
    • Yu, R.Z., Geary, R.S., Monteith, D.K., Matson, J., Truong, L., Fitchett, J. and Levin, A.A. (2004) Tissue disposition of a 2'-O-(2-methoxy) ethyl modified antisense oligonucleotides in monkeys. J. Pharm. Sci., 93, 48-59.
    • (2004) J. Pharm. Sci. , vol.93 , pp. 48-59
    • Yu, R.Z.1    Geary, R.S.2    Monteith, D.K.3    Matson, J.4    Truong, L.5    Fitchett, J.6    Levin, A.A.7
  • 9
  • 10
    • 50349084487 scopus 로고    scopus 로고
    • Antisense oligonucleotide directed to human apolipoprotein B-100 reduces lipoprotein(a) levels and oxidized phospholipids on human apolipoprotein B-100 particles in lipoprotein(a) transgenic mice
    • Merki, E., Graham, M.J., Mullick, A.E., Miller, E.R., Crooke, R.M., Pitas, R.E., Witztum, J.L. and Tsimikas, S. (2008) Antisense oligonucleotide directed to human apolipoprotein B-100 reduces lipoprotein(a) levels and oxidized phospholipids on human apolipoprotein B-100 particles in lipoprotein(a) transgenic mice. Circulation, 118, 743-753.
    • (2008) Circulation , vol.118 , pp. 743-753
    • Merki, E.1    Graham, M.J.2    Mullick, A.E.3    Miller, E.R.4    Crooke, R.M.5    Pitas, R.E.6    Witztum, J.L.7    Tsimikas, S.8
  • 11
    • 0030806664 scopus 로고    scopus 로고
    • Evaluation of the toxicity of ISIS 2302, a phosphorothioate oligonucleotide in a 4-week study in CD-1 mice
    • Henry, S.P., Taylor, J., Midgley, L., Levin, A.A. and Kornbrust, D.J. (1997) Evaluation of the toxicity of ISIS 2302, a phosphorothioate oligonucleotide in a 4-week study in CD-1 mice. Antisense Nucleic Acid Drug Dev., 7, 473-481.
    • (1997) Antisense Nucleic Acid Drug Dev. , vol.7 , pp. 473-481
    • Henry, S.P.1    Taylor, J.2    Midgley, L.3    Levin, A.A.4    Kornbrust, D.J.5
  • 12
    • 33750207033 scopus 로고    scopus 로고
    • Potent reduction of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B
    • Kastelein, J.J., Wedel, M.K., Baker, B.F., Su, J., Bradley, J.D., Yu, R.Z., Chuang, E., Graham, M.J. and Crooke, R.M. (2006) Potent reduction of apolipoprotein B and low-density lipoprotein cholesterol by short-term administration of an antisense inhibitor of apolipoprotein B. Circulation, 114, 1729-1735.
    • (2006) Circulation , vol.114 , pp. 1729-1735
    • Kastelein, J.J.1    Wedel, M.K.2    Baker, B.F.3    Su, J.4    Bradley, J.D.5    Yu, R.Z.6    Chuang, E.7    Graham, M.J.8    Crooke, R.M.9
  • 15
    • 0032909507 scopus 로고    scopus 로고
    • Synthesis of 3'-C- and 4'-C-Branched Oligodeoxynucleotides and the Development of Locked Nucleic Acid (LNA)
    • Wengel, J. (1999) Synthesis of 3'-C- and 4'-C-Branched Oligodeoxynucleotides and the Development of Locked Nucleic Acid (LNA). Acc. Chem. Res., 32, 301-310.
    • (1999) Acc. Chem. Res. , vol.32 , pp. 301-310
    • Wengel, J.1
  • 16
    • 84878247636 scopus 로고    scopus 로고
    • Preclinical evaluation of the toxicological effects of a novel constrained ethyl modified antisense compound targeting signal transducer and activator of transcription 3 in mice and cynomolgus monkeys
    • Burel, S.A., Han, S.R., Lee, H.S., Norris, D.A., Lee, B.S., Machemer, T., Park, S.Y., Zhou, T., He, G., Kim, Y. et al. (2013) Preclinical evaluation of the toxicological effects of a novel constrained ethyl modified antisense compound targeting signal transducer and activator of transcription 3 in mice and cynomolgus monkeys. Nucleic Acid Ther., 23, 213-227.
    • (2013) Nucleic Acid Ther. , vol.23 , pp. 213-227
    • Burel, S.A.1    Han, S.R.2    Lee, H.S.3    Norris, D.A.4    Lee, B.S.5    Machemer, T.6    Park, S.Y.7    Zhou, T.8    He, G.9    Kim, Y.10
  • 17
    • 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
  • 19
    • 84887841103 scopus 로고    scopus 로고
    • First-in-human Phase i study of EZN-4176, a locked nucleic acid antisense oligonucleotide to exon 4 of the androgen receptor mRNA in patients with castration-resistant prostate cancer
    • Bianchini, D., Omlin, A., Pezaro, C., Lorente, D., Ferraldeschi, R., Mukherji, D., Crespo, M., Figueiredo, I., Miranda, S., Riisnaes, R. et al. (2013) First-in-human Phase I study of EZN-4176, a locked nucleic acid antisense oligonucleotide to exon 4 of the androgen receptor mRNA in patients with castration-resistant prostate cancer. Br. J. Cancer, 109, 2579-2586.
    • (2013) Br. J. Cancer , vol.109 , pp. 2579-2586
    • Bianchini, D.1    Omlin, A.2    Pezaro, C.3    Lorente, D.4    Ferraldeschi, R.5    Mukherji, D.6    Crespo, M.7    Figueiredo, I.8    Miranda, S.9    Riisnaes, R.10
  • 21
    • 84905108842 scopus 로고    scopus 로고
    • A phase i study of EZN-3042, a novel survivin messenger ribonucleic acid (mRNA) antagonist, administered in combination with chemotherapy in children with relapsed acute lymphoblastic leukemia (ALL): A report from the therapeutic advances in childhood leukemia and lymphoma (TACL) consortium
    • Raetz, E.A., Morrison, D., Romanos-Sirakis, E., Gaynon, P., Sposto, R., Bhojwani, D., Bostrom, B.C., Brown, P., Eckroth, E., Cassar, J. et al. (2014) A phase I study of EZN-3042, a novel survivin messenger ribonucleic acid (mRNA) antagonist, administered in combination with chemotherapy in children with relapsed acute lymphoblastic leukemia (ALL): a report from the therapeutic advances in childhood leukemia and lymphoma (TACL) consortium. J. Pediatr. Hematol. Oncol., 36, 458-463.
    • (2014) J. Pediatr. Hematol. Oncol. , vol.36 , pp. 458-463
    • Raetz, E.A.1    Morrison, D.2    Romanos-Sirakis, E.3    Gaynon, P.4    Sposto, R.5    Bhojwani, D.6    Bostrom, B.C.7    Brown, P.8    Eckroth, E.9    Cassar, J.10
  • 24
    • 84868347571 scopus 로고    scopus 로고
    • Structure activity relationships of alpha-L-LNA modified phosphorothioate gapmer antisense oligonucleotides in animals
    • Seth, P.P., Jazayeri, A., Yu, J., Allerson, C.R., Bhat, B. and Swayze, E.E. (2012) Structure activity relationships of alpha-L-LNA modified phosphorothioate gapmer antisense oligonucleotides in animals. Mol. Ther. Nucleic Acids, 1, e47.
    • (2012) Mol. Ther. Nucleic Acids , vol.1 , pp. e47
    • Seth, P.P.1    Jazayeri, A.2    Yu, J.3    Allerson, C.R.4    Bhat, B.5    Swayze, E.E.6
  • 28
    • 79954438975 scopus 로고    scopus 로고
    • The novel antisense Bcl-2 inhibitor SPC2996 causes rapid leukemic cell clearance and immune activation in chronic lymphocytic leukemia
    • Durig, J., Duhrsen, U., Klein-Hitpass, L., Worm, J., Hansen, J.B., Orum, H. and Wissenbach, M. (2011) The novel antisense Bcl-2 inhibitor SPC2996 causes rapid leukemic cell clearance and immune activation in chronic lymphocytic leukemia. Leukemia, 25, 638-647.
    • (2011) Leukemia , vol.25 , pp. 638-647
    • Durig, J.1    Duhrsen, U.2    Klein-Hitpass, L.3    Worm, J.4    Hansen, J.B.5    Orum, H.6    Wissenbach, M.7
  • 32
    • 0033555551 scopus 로고    scopus 로고
    • Characterization of a potent and specific class of antisense oligonucleotide inhibitor of human protein kinase C-α expression
    • McKay, R.A., Miraglia, L.J., Cummins, L.L., Owens, S.R., Sasmor, H. and Dean, N.M. (1999) Characterization of a potent and specific class of antisense oligonucleotide inhibitor of human protein kinase C-α expression. J. Biol. Chem., 274, 1715-1722.
    • (1999) J. Biol. Chem. , vol.274 , pp. 1715-1722
    • McKay, R.A.1    Miraglia, L.J.2    Cummins, L.L.3    Owens, S.R.4    Sasmor, H.5    Dean, N.M.6
  • 33
    • 0001677717 scopus 로고
    • Controlling the false discovery rate: A practical and powerful approach to multiple testing
    • Benjamini, Y. and Hochberg, Y. (1995) Controlling the false discovery rate: a practical and powerful approach to multiple testing. J. R. Statistical Soc., 57, 289-300.
    • (1995) J. R. Statistical Soc. , vol.57 , pp. 289-300
    • Benjamini, Y.1    Hochberg, Y.2
  • 34
    • 4744373229 scopus 로고    scopus 로고
    • Noise amidst the silence: Off-target effects of siRNAs?
    • Jackson, A.L. and Linsley, P.S. (2004) Noise amidst the silence: off-target effects of siRNAs? Trends Genet., 20, 521-524.
    • (2004) Trends Genet. , vol.20 , pp. 521-524
    • Jackson, A.L.1    Linsley, P.S.2
  • 37
    • 74049124186 scopus 로고    scopus 로고
    • Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application
    • Jackson, A.L. and Linsley, P.S. (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
  • 38
    • 33745612464 scopus 로고    scopus 로고
    • Widespread siRNA 'off-target' transcript silencing mediated by seed region sequence complementarity
    • Jackson, A.L., Burchard, J., Schelter, J., Chau, B.N., Cleary, M., Lim, L. and Linsley, P.S. (2006) Widespread siRNA 'off-target' transcript silencing mediated by seed region sequence complementarity. RNA, 12, 1179-1187.
    • (2006) RNA , vol.12 , 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
  • 39
    • 57049175013 scopus 로고    scopus 로고
    • Detecting microRNA binding and siRNA off-target effects from expression data
    • van Dongen, S., Abreu-Goodger, C. and Enright, A.J. (2008) Detecting microRNA binding and siRNA off-target effects from expression data. Nat. Methods, 5, 1023-1025.
    • (2008) Nat. Methods , vol.5 , pp. 1023-1025
    • Van Dongen, S.1    Abreu-Goodger, C.2    Enright, A.J.3
  • 42
    • 33751348056 scopus 로고    scopus 로고
    • Ablation in mice of the mTORC components raptor, rictor, or mLST8 reveals that mTORC2 is required for signaling to Akt-FOXO and PKCalpha, but not S6K1
    • Guertin, D.A., Stevens, D.M., Thoreen, C.C., Burds, A.A., Kalaany, N.Y., Moffat, J., Brown, M., Fitzgerald, K.J. and Sabatini, D.M. (2006) Ablation in mice of the mTORC components raptor, rictor, or mLST8 reveals that mTORC2 is required for signaling to Akt-FOXO and PKCalpha, but not S6K1. Dev. Cell, 11, 859-871.
    • (2006) Dev. Cell , vol.11 , pp. 859-871
    • Guertin, D.A.1    Stevens, D.M.2    Thoreen, C.C.3    Burds, A.A.4    Kalaany, N.Y.5    Moffat, J.6    Brown, M.7    Fitzgerald, K.J.8    Sabatini, D.M.9
  • 43
    • 36348963611 scopus 로고    scopus 로고
    • A deletion mutation of the protein tyrosine phosphatase kappa (Ptprk) gene is responsible for T-helper immunodeficiency (thid) in the LEC rat
    • Asano, A., Tsubomatsu, K., Jung, C.G., Sasaki, N. and Agui, T. (2007) A deletion mutation of the protein tyrosine phosphatase kappa (Ptprk) gene is responsible for T-helper immunodeficiency (thid) in the LEC rat. Mamm. Genome, 18, 779-786.
    • (2007) Mamm. Genome , vol.18 , pp. 779-786
    • Asano, A.1    Tsubomatsu, K.2    Jung, C.G.3    Sasaki, N.4    Agui, T.5
  • 45
    • 39749108409 scopus 로고    scopus 로고
    • Development of hepatocellular carcinoma in Iqgap2-deficient mice is IQGAP1 dependent
    • Schmidt, V.A., Chiariello, C.S., Capilla, E., Miller, F. and Bahou, W.F. (2008) Development of hepatocellular carcinoma in Iqgap2-deficient mice is IQGAP1 dependent. Mol. Cell. Biol., 28, 1489-1502.
    • (2008) Mol. Cell. Biol. , vol.28 , pp. 1489-1502
    • Schmidt, V.A.1    Chiariello, C.S.2    Capilla, E.3    Miller, F.4    Bahou, W.F.5
  • 47
    • 55949087070 scopus 로고    scopus 로고
    • Analysis of human disease genes in the context of gene essentiality
    • Park, D., Park, J., Park, S.G., Park, T. and Choi, S.S. (2008) Analysis of human disease genes in the context of gene essentiality. Genomics, 92, 414-418.
    • (2008) Genomics , vol.92 , pp. 414-418
    • Park, D.1    Park, J.2    Park, S.G.3    Park, T.4    Choi, S.S.5
  • 48
    • 84946543730 scopus 로고    scopus 로고
    • Identification and characterization of modified antisense oligonucleotides targeting dmpk in mice and nonhuman primates for the treatment of Myotonic Dystrophy type 1
    • Pandey, S.K., Wheeler, T.M., Justice, S.L., Kim, A., Younis, H., Gattis, D., Jauvin, D., Puymirat, J., Swayze, E.E., Freier, S.M. et al. (2015) Identification and characterization of modified antisense oligonucleotides targeting dmpk in mice and nonhuman primates for the treatment of Myotonic Dystrophy type 1. J. Pharmacol. Exp. Ther., 355, 310-321.
    • (2015) J. Pharmacol. Exp. Ther. , vol.355 , pp. 310-321
    • Pandey, S.K.1    Wheeler, T.M.2    Justice, S.L.3    Kim, A.4    Younis, H.5    Gattis, D.6    Jauvin, D.7    Puymirat, J.8    Swayze, E.E.9    Freier, S.M.10
  • 49
    • 84878243943 scopus 로고    scopus 로고
    • Inhibition of vascular permeability by antisense-mediated inhibition of plasma kallikrein and coagulation factor 12
    • Bhattacharjee, G., Revenko, A.S., Crosby, J.R., May, C., Gao, D., Zhao, C., Monia, B.P. and MacLeod, A.R. (2013) Inhibition of vascular permeability by antisense-mediated inhibition of plasma kallikrein and coagulation factor 12. Nucleic Acid Ther., 23, 175-187.
    • (2013) Nucleic Acid Ther. , vol.23 , pp. 175-187
    • Bhattacharjee, G.1    Revenko, A.S.2    Crosby, J.R.3    May, C.4    Gao, D.5    Zhao, C.6    Monia, B.P.7    MacLeod, A.R.8
  • 50
    • 78649471947 scopus 로고    scopus 로고
    • Inhibition of the intrinsic coagulation pathway factor XI by antisense oligonucleotides: A novel antithrombotic strategy with lowered bleeding risk
    • Zhang, H., Lowenberg, E.C., Crosby, J.R., MacLeod, A.R., Zhao, C., Gao, D., Black, C., Revenko, A.S., Meijers, J.C., Stroes, E.S. et al. (2010) Inhibition of the intrinsic coagulation pathway factor XI by antisense oligonucleotides: a novel antithrombotic strategy with lowered bleeding risk. Blood, 116, 4684-4692.
    • (2010) Blood , vol.116 , pp. 4684-4692
    • Zhang, H.1    Lowenberg, E.C.2    Crosby, J.R.3    MacLeod, A.R.4    Zhao, C.5    Gao, D.6    Black, C.7    Revenko, A.S.8    Meijers, J.C.9    Stroes, E.S.10
  • 51
    • 25144497136 scopus 로고    scopus 로고
    • The influence of locked nucleic acid residues on the thermodynamic properties of 2'-O-methyl RNA/RNA heteroduplexes
    • Kierzek, E., Ciesielska, A., Pasternak, K., Mathews, D.H., Turner, D.H. and Kierzek, R. (2005) The influence of locked nucleic acid residues on the thermodynamic properties of 2'-O-methyl RNA/RNA heteroduplexes. Nucleic Acids Res., 33, 5082-5093.
    • (2005) Nucleic Acids Res. , vol.33 , pp. 5082-5093
    • Kierzek, E.1    Ciesielska, A.2    Pasternak, K.3    Mathews, D.H.4    Turner, D.H.5    Kierzek, R.6
  • 52
  • 53


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