Comparison of hepatic transcription profiles of locked ribonucleic acid antisense oligonucleotides: Evidence of distinct pathways contributing to non-target mediated toxicity in mice

Toxicological Sciences

Volumn 138, Issue 1, 2014, Pages 234-248

  Kakiuchi Kiyota, Satoko ^a   Koza Taylor, Petra H ^a   Mantena, Srinivasa R ^a   Nelms, Linda F ^a   Enayetallah, Ahmed E ^a   Hollingshead, Brett D ^a   Burdick, Andrew D ^a   Reed, Lori A ^a   Warneke, James A ^a   Whiteley, Lawrence O ^a   Ryan, Anne M ^a   Mathialagan, Nagappan ^a  

^a PFIZER INC   (United States)

Author keywords

Antisense oligonucleotide; Clathrin mediated endocytosis; Hepatotoxicity; LNA gapmers; Microarray analysis

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; CLATHRIN-MEDIATED ENDOCYTOSIS; HEPATOTOXICITY; LNA GAPMERS; MICROARRAY ANALYSIS;

ANIMALS; CLATHRIN; DRUG-INDUCED LIVER INJURY; ENDOCYTOSIS; GENE EXPRESSION PROFILING; INJECTIONS, SUBCUTANEOUS; LIVER; MALE; MICE; MICE, INBRED STRAINS; MOLECULAR SEQUENCE DATA; OLIGONUCLEOTIDES; OLIGONUCLEOTIDES, ANTISENSE; TRANSCRIPTOME;

EID: 84894339791     PISSN: 10966080     EISSN: 10960929     Source Type: Journal    
DOI: 10.1093/toxsci/kft278     Document Type: Article

References (28)

1. 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.
2. Burel, S. A., Machemer, T., Ragone, F. L., Kato, H., Cauntay, P., Greenlee, S., Salim, A., Gaarde, W. A., Hung, G., Peralta, R., et al. (2012). Unique O-methoxyethyl ribose-DNA chimeric oligonucleotide induces an atypical melanoma differentiation-associated gene 5-dependent induction of type I interferon response. J. Pharmacol. Exp. Ther. 342, 150-162.
3. Casey, C. A., Lee, S. M., Aziz-Seible, R., McVicker, B. L. (2008). Impaired receptor-mediated endocytosis: Its role in alcohol-induced apoptosis. J. Gastroenterol. Hepatol. 23, S46-S49.
4. Cheng, J., Grassart, A., and Drubin, D. G. (2012). Myosin 1E coordinates actin assembly and cargo trafficking during clathrin-mediated endocytosis. Mol. Biol. Cell 23, 2891-2904.
5. Chindelevitch, L., Ziemek, D., Enayetallah, A. E., Randhawa, R., Sidders, B., Brockel, C., Huang, E. S. (2012). Causal reasoning on biological networks: Interpreting transcriptional changes. Bioinformatics 28, 1114-1121
6. Conner, S. D., and Schmid, S. L. (2003). Regulated portals of entry into the cell. Nature 422, 37-44.
7. Enayetallah, A. E., Ziemek, D., Leininger, M. T., Randhawa, R., Yang, J., Manion, T. B., Mather, D. E., Zavadoski, W. J., Kuhn, M., Treadway, J. L., et al. (2011). Modeling the mechanism of action of a DGAT1 inhibitor using a causal reasoning platform. PloS ONE 6, e27009.
8. Enayetallah, A. E., Puppala, D., Ziemek, D., Fischer, J. E., Kantesaria, S., and Pletcher, M. T. (2013). Assessing the translatability of in vivo cardiotoxicity mechanisms to in vitro models using causal reasoning. BMC Pharmacol. Toxicol. 14, 46.
9. Fernandez, D. J., McVicker, B. L., Tuma, D. J., and Tuma, P. L. (2009). Ethanol selectively impairs clathrin-mediated internalization in polarized hepatic cells. Biochem. Pharmacol. 78, 648-655.
10. Grünweller, A., and Hartmann, R. K. (2007). Locked nucleic acid oligonucleotides: The next generation of antisense agents? BioDrugs 21, 235-243.
11. Hsiang, Y. H., and Liu, L. F. (1988). Identification of mammalian DNA topoisomerase I as an intracellular target of the anticancer drug camptothecin. Cancer Res. 48, 1722-1726.
12. Jepsen, J. S., Sørensen, M. D., and Wengel, J. (2004). Locked nucleic acid: A potent nucleic acid analog in therapeutics and biotechnology. Oligonucleotides 14, 130-146.
13. Jones, D. (2011). The long march of antisense. Nat. Rev. Drug Discov. 10, 401-402
14. Juliano, R. L., Dixit, V. R., Kang, H., Kim, T. Y., Miyamoto, Y., and Xu, D. (2005). Epigenetic manipulation of gene expression: A toolkit for cell biologists. J. Cell Biol. 169, 847-857.
15. 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.
16. Kollmannsberger, C., Mross, K., Jakob, A., Kanz, L., and Bokemeyer, C. (1999). Topotecan-A novel topoisomerase I inhibitor: Pharmacology and clinical experience. Oncology 56, 1-12.
17. Krieg, A. M. (2012). Targeting LDL cholesterol with LNA. Mol. Ther. Nucleic Acids 1, 1-2.
18. Laifenfeld, D., Gilchrist, A., Drubin, D., Jorge, M., Eddy, S. F., Frushour, B. P., Ladd, B., Obert, L. A., Gosink, M. M., Cook, J. C., et al. (2010). The role of hypoxia in 2-butoxyethanol-induced hemangiosarcoma. Toxicol. Sci. 113, 254-266.
19. Le Roy, C., and Wrana, J. L. (2005). Clathrin- and non-clathrin-mediated endocytic regulation of cell signalling. Nat. Rev. Mol. Cell Biol. 6, 112-126.
20. McMahon, H. T., and Boucrot, E. (2011). Molecular mechanism and physiological functions of clathrin-mediated endocytosis. Nat. Rev. Mol. Cell Biol. 12, 517-533.
21. Mundy, D. I., Machleidt, T., Ying, Y. S., Anderson, R. G., and Bloom, G. S. (2002). Dual control of caveolar membrane traffic by microtubules and the actin cytoskeleton. J. Cell Sci. 115(Pt 22), 4327-4339.
22. Murrow, L., Debnath, J. (2013). Autophagy as a stress-response and qualitycontrol mechanism: Implications for cell injury and human disease. Annu. Rev. Pathol. 24, 105-137.
23. Pommier, Y. (2006). Topoisomerase I inhibitors: camptothecins and beyond. Nat. Rev. Cancer 6, 789-802.
24. Pommier, Y., Barcelo, J. M., Rao, V. A., Sordet, O., Jobson, A. G., Thibaut, L., Miao, Z. H., Seiler, J. A., Zhang, H., Marchand, C., et al. (2006). Repair of topoisomerase I-mediated DNA damage. Prog. Nucleic Acid Res. Mol. Biol. 81, 179-229.
25. Straarup, E. M., Fisker, N., Hedtjärn, M., Lindholm, M. W., Rosenbohm, C., Aarup, V., Hansen, H. F., Ørum, H., Hansen, J. B., Koch, T. (2010). Short locked nucleic acid antisense oligonucleotides potently reduce apolipoprotein B mRNA and serum cholesterol in mice and non-human primates. Nucleic Acids Res. 38, 7100-7011.
26. Stanton, R., Sciabola, S., Salatto, C., Weng, Y., Moshinsky, D., Little, J., Walters, E., Kreeger, J., DiMattia, D., Chen, T., et al. (2012). Chemical modification study of antisense gapmers. Nucleic Acid Ther. 22, 344-359.
27. Swayze, E. E., Siwkowski, A. M., Wancewicz, E. V., Migawa, M. T., Wyrzykiewicz, T. K., Hung, G., Monia, B. P., Bennett, C. F., et al. (2007). Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals. Nucleic Acids Res. 35, 687-700.
28. Ungewickell, E. J., and Hinrichsen, L. (2007). Endocytosis: Clathrin-mediated membrane budding. Curr. Opin. Cell Biol. 19, 417-425.