Evaluation of multiple-turnover capability of locked nucleic acid antisense oligonucleotides in Cell-Free RNase H-mediated antisense reaction and in mice

Nucleic Acid Therapeutics

Volumn 24, Issue 4, 2014, Pages 283-290

  Yamamoto, Tsuyoshi ^a   Fujii, Naoko ^a   Yasuhara, Hidenori ^a   Wada, Shunsuke ^a   Wada, Fumito ^a   Shigesada, Naoya ^a   Harada Shiba, Mariko ^b   Obika, Satoshi ^a  

^a OSAKA UNIVERSITY   (Japan)

^b NATIONAL CEREBRAL AND CARDIOVASCULAR CENTER   (Japan)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; APOLIPOPROTEIN B; APOLIPOPROTEIN B 10A; APOLIPOPROTEIN B 10B; APOLIPOPROTEIN B 10C; APOLIPOPROTEIN B 10D; APOLIPOPROTEIN B 11A; APOLIPOPROTEIN B 11B; APOLIPOPROTEIN B 12A; APOLIPOPROTEIN B 12B; APOLIPOPROTEIN B 12C; APOLIPOPROTEIN B 13A; APOLIPOPROTEIN B 13C; APOLIPOPROTEIN B 13D; APOLIPOPROTEIN B 13E; APOLIPOPROTEIN B 13G; APOLIPOPROTEIN B 13H; APOLIPOPROTEIN B 13I; APOLIPOPROTEIN B 14A; APOLIPOPROTEIN B 14B; APOLIPOPROTEIN B 16A; APOLIPOPROTEIN B 20A; APOLIPOPROTEIN B 20B; APOLIPOPROTEIN B 20C; CHOLESTEROL; LOCKED NUCLEIC ACID; MESSENGER RIBONUCLEIC ACID 1; MESSENGER RNA; RIBONUCLEASE H; UNCLASSIFIED DRUG; UNINDEXED DRUG;

3' UNTRANSLATED REGION; 5' UNTRANSLATED REGION; ANIMAL EXPERIMENT; ANIMAL TISSUE; ANTISENSE THERAPY; ARTICLE; BINDING AFFINITY; CELL FREE SYSTEM; CELLULAR DISTRIBUTION; CHOLESTEROL BLOOD LEVEL; CONSENSUS SEQUENCE; CONTROLLED STUDY; DNA RNA HYBRIDIZATION; DRUG EFFICACY; DRUG PROTEIN BINDING; ENZYME KINETICS; HYPERCHOLESTEROLEMIA; IN VIVO STUDY; LIMIT OF QUANTITATION; LOW DRUG DOSE; MALE; MELTING POINT; MOUSE; NONHUMAN; PRIORITY JOURNAL; PROTEIN BINDING; QUANTITATIVE ANALYSIS; REACTION ANALYSIS; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; TURNOVER TIME;

ANIMALS; APOLIPOPROTEIN B-100; CELL-FREE SYSTEM; GENE EXPRESSION; HALF-LIFE; INJECTIONS, SUBCUTANEOUS; LIVER; MALE; MICE; MICE, INBRED C57BL; OLIGONUCLEOTIDES; OLIGONUCLEOTIDES, ANTISENSE; RIBONUCLEASE H; RNA STABILITY; RNA, MESSENGER; TRANSITION TEMPERATURE;

EID: 84903956784     PISSN: 21593337     EISSN: 21593345     Source Type: Journal    
DOI: 10.1089/nat.2013.0470     Document Type: Article

References (29)

1. Crooke, S.T., Lemonidis, K.M., Neilson, L., Griffey, R., Lesnik, E.A., aND Monia, B.P. (1995). Kinetic characteristics of Escherichia coli RNase H1: Cleavage of various antisense oligonucleotide-RNA duplexes. Biochem. J. 312, 599-608 (Pubitemid 26002231)
2. Crooke, T.S. (2007). Antisense Drug Technologies: Principles, Strategies, and Applications. 2nd ed. (CRC Press Taylor & Francis Group, Boca Raton, FL
3. Gao, W.Y., Han, F.S., Storm, C., Egan, W., aND Cheng, Y.C. (1992). Phosphorothioate oligonucleotides are inhibitors of human DNA polymerases and RNase H: Implications for antisense technology. Mol. Pharmacol. 41, 223-229
4. Hari, Y., Obika, S., Ohnishi, R., Eguchi, K., Osaki, T., Ohishi, H., aND Imanishi, T. (2006). Synthesis and properties of 2¢-O, 4-C- methyleneoxymethylene bridged nucleic acid. Bioorgan. Med. Chem. 14, 1029-1038 (Pubitemid 43059794)
5. Hutvagner, G., aND Zamore, P.D. (2002). A microRNA in a multiple-Turnover RNAi enzyme complex. Science 297, 2056-2060
6. 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
7. Kurreck, J., Wyszko, E., Gillen, C., aND Erdmann, V.A. (2002). Design of antisense oligonucleotides stabilized by locked nucleic acids. Nucleic Acids Res. 30, 1911-1918 (Pubitemid 34537585)
8. Lima, W.F., aND Crooke, S.T. (1997). Binding affinity and specificity of Escherichia coli RNase H1: Impact on the kinetics of catalysis of antisense oligonucleotide-RNA hybrids. Biochemistry 36, 390-398 (Pubitemid 27043475)
9. Lima, W.F., Rose, J.B., Nichols, J.G., Wu, H.J., Migawa, M.T., Wyrzykiewicz, T.K., Siwkowski, A.M., aND Crooke, S.T. (2007a). Human RNase H1 discriminates between subtle variations in the structure of the heteroduplex substrate. Mol. Pharmacol. 71, 83-91 (Pubitemid 46020979)
10. Lima, W.F., Rose, J.B., Nichols, J.G., Wu, H.J., Migawa, M.T., Wyrzykiewicz, T.K., Vasquez, G., Swayze, E.E., aND Crooke, S.T. (2007b). The positional influence of the helical geometry of the heteroduplex substrate on human RNase H1 catalysis. Mol. Pharmacol. 71, 73-82 (Pubitemid 46020978)
11. Miyashita, K., Rahman, S.M.A., Seki, S., Obika, S., aND Imanishi, T. (2007). N-Methyl substituted 2 ¢, 4 ¢-BNANC: A highly nuclease-resistant nucleic acid analogue with highaffinity RNA selective hybridization. Chem. Commun. 2007, 3765-3767 (Pubitemid 47404177)
12. Monia, B.P., Lesnik, E.A., Gonzalez, C., Lima, W.F., Mcgee, D., Guinosso, C.J., Kawasaki, A.M., Cook, P.D., aND Freier, S.M. (1993). Evaluation of 2¢-modified oligonucleotides containing 2¢-deoxy gaps as antisense inhibitors of gene expression. J. Biol. Chem. 268, 14514-14522 (Pubitemid 23195583)
13. 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 (Pubitemid 47599996)
14. 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 (Pubitemid 28295121)
15. Obika, S., Nanbu, D., Hari, Y., Morio, K., In, Y., Ishida, T., aND Imanishi, T. (1997). Synthesis of 2¢-O, 4¢-C-methyleneuridine and-cytidine. Novel bicyclic nucleosides having a fixed C-3,-endo sugar puckering. Tetrahedron Lett. 38, 8735-8738 (Pubitemid 27504436)
16. 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
17. 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
18. Singh, S.K., Nielsen, P., Koshkin, A.A., aND Wengel, J. (1998). LNA (locked nucleic acids): Synthesis and highaffinity nucleic acid recognition. Chem. Commun. 1998, 455-456 (Pubitemid 128675297)
19. Sohlenius-Sternbeck, A.K. (2006) Determination of the hepatocellularity number for human, dog, rabbit, rat and mouse livers from protein concentration measurements. Toxicol. In Vitro 20, 1582-1586
20. Stanton, R., Sciabola, S., Salatto, C., Weng, Y., Moshinsky, D., Little, J., Walters, E., Kreeger, J., et al. (2012). Chemical modification study of antisense gapmers. Nucleic Acid Ther. 22, 344-359
21. Stein, C.A., Hansen, J.B., Lai, J., Wu, S., Voskresenskiy, A., Hog, A., Worm, J., Hedtjarn, M., Souleimanian, N., Miller, P., et al. (2010). Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents. Nucleic Acids Res. 38, e3
22. Straarup, E.M., Fisker, N., Hedtjarn, M., Lindholm, M.W., Rosenbohm, C., Aarup, V., Hansen, H.F., Orum, H., Hansen, J.B., aND 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, 7100-7111
23. Vester, B., Boel, A.M., Lobedanz, S., Babu, B.R., Raunkjaer, M., Lindegaard, D., Raunak, HRDLICKA, P.J., Hojland, T., et al. (2008). Chemically modified oligonucleotides with efficient RNase H response. Bioorg. Med. Chem. Lett. 18, 2296-2300 (Pubitemid 351442904)
24. Watanabe, T.A., Geary, R.S., aND Levin, A.A. (2006). Plasma protein binding of an antisense oligonucleotide targeting human ICAM-1 (ISIS 2302). Oligonucleotides 16, 169-180
25. Yahara, A., Shrestha, A.R., Yamamoto, T., Hari, Y., Osawa, T., Yamaguchi, M., Nishida, M., Kodama, T., aND Obika, S. (2012). Amido-bridged nucleic acids (AmNAs): Synthesis, duplex stability, nuclease resistance, and in vitro antisense potency. Chembiochem 13, 2513-2516
26. Yamamoto, T., Harada-Shiba, M., Nakatani, M., Wada, S., Yasuhara, H., Narukawa, K., Sasaki, K., Shibata, M.A., Torigoe, H., et al. (2012a). Cholesterol-lowering action of BNA-based antisense oligonucleotides targeting PCSK9 in atherogenic diet-induced hypercholesterolemic mice. Mol. Ther. Nucleic Acids 1, e22
27. Yamamoto, T., Nakatani, M., Narukawa, K., aND Obika, S. (2011). Antisense drug discovery and development. Future Med. Chem. 3, 339-365
28. Yamamoto, T., Yasuhara, H., Wada, F., Haradashiba, M., Imanishi, T., aND Obika, S. (2012b). Superior silencing by 22, 42-BNANC-Based short antisense oligonucleotides compared to 22, 42-BNA/LNA-based apolipoprotein B antisense inhibitors. J. Nucleic Acids, Article ID 707323
29. Zhang, Y., Qu, Z., Kim, S., Shi, V., Liao, B., Kraft, P., Bandaru, R., Wu, Y., Greenberger, L.M., aND Horak, I.D. (2011). Down-modulation of cancer targets using locked nucleic acid (LNA)-based antisense oligonucleotides without transfection. Gene Ther, 18, 326-333.