Potent inhibition of microRNA in vivo without degradation

Nucleic Acids Research

Volumn 37, Issue 1, 2009, Pages 70-77

  Davis, Scott ^a   Propp, Stephanie ^b   Freier, Susan M ^b   Jones, Laura E ^c   Serra, Martin J ^c   Kinberger, Garth ^b   Bhat, Balkrishen ^a   Swayze, Eric E ^b   Bennett, C Frank ^b   Esau, Christine ^a  

^a REGULUS THERAPEUTICS   (United States)

^b ISIS PHARMACEUTICALS   (United States)

^c ALLEGHENY COLLEGE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

2' FLUORO/2' O METHOXYETHYL ANTISENSE OLIGONUCLEOTIDE; 2' O METHOXYETHYL ANTISENSE OLIGONUCLEOTIDE; 2' O METHOXYETHYL LOCKED NUCLEIC ACID; 2' O METHYL ANTISENSE OLIGONUCLEOTIDE; ALANINE AMINOTRANSFERASE; ANTISENSE OLIGONUCLEOTIDE; CHOLESTEROL; MICRORNA; OLIGONUCLEOTIDE; SPC 3649; UNCLASSIFIED DRUG;

ALANINE AMINOTRANSFERASE BLOOD LEVEL; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; CHEMICAL MODIFICATION; CHOLESTEROL BLOOD LEVEL; CONTROLLED STUDY; DOSE RESPONSE; DRUG DOSE COMPARISON; DRUG MEGADOSE; DRUG POTENCY; DRUG SCREENING; DRUG SYNTHESIS; GENE EXPRESSION; IN VIVO STUDY; LIVER; MALE; MOUSE; NONHUMAN; NUCLEIC ACID ANALYSIS; PRIORITY JOURNAL; SPLEEN WEIGHT;

ANIMALS; GENE EXPRESSION REGULATION; LIVER; MALE; MICE; MICE, INBRED C57BL; MICRORNAS; OLIGONUCLEOTIDES, ANTISENSE; PEPTIDE NUCLEIC ACIDS;

EID: 58549109007     PISSN: 03051048     EISSN: 13624962     Source Type: Journal    
DOI: 10.1093/nar/gkn904     Document Type: Article

References (23)

1. Bartel,D.P. (2004) MicroRNAs: Genomics, biogenesis, mechanism, and function. Cell, 116, 281-297.
2. Du,T. and Zamore,P.D. (2005) microPrimer: The biogenesis and function of microRNA. Development, 132, 4645-4652.
3. Ambros,V. (2004) The functions of animal microRNAs. Nature, 431 350-355.
4. Kloosterman,W.P. and Plasterk,R.H. (2006) The diverse functions of microRNAs in animal development and disease. Dev. Cell, 11, 441-450.
5. Davis,S., Lollo,B., Freier,S. and Esau,C. (2006) Improved targeting of miRNA with antisense oligonucleotides. Nucleic Acids Res., 34 2294-2304.
6. Hutvagner,G., Simard,M.J., Mello,C.C. and Zamore,P.D. (2004) Sequence-specific inhibition of small RNA function. PLoS Biol., 2, E98.
7. Meister,G., Landthater,M., Dorsett,Y. and Tuschl,T. (2004) Sequence-specific inhibition of microRNA- and siRNA-induced RNA silencing. RNA, 10, 544-550.
8. Orom,U.A., Kauppinen,S. and Lund,A.H. (2006) LNA-modified oligonucleotides mediate specific inhibition of microRNA function. Gene, 372, 137-141.
9. Vermeulen,A., Robertson,B., Dalby,A.B., Marshall,W.S., Karpilow,J., Leake,D., Khvorova,A. and Baskerville,S. (2007) Double-stranded regions are essential design components of potent inhibitors of RISC function. RNA, 13, 723-730.
10. Elmen,J., Lindow,M., Schutz,S., Lawrence,M., Petri,A., Obad,S., Lindholm,M., Hedtjam,M., Hansen,H.F., Berger,U. et al. (2008) LNA-mediated microRNA silencing in non-human primates. Nature, 452, 896-899.
11. Elmen,J., Lindow,M., Silahtaroglu,A., Bak,M., Christensen,M., Lind-Thomsen,A., Hedtjam,M., Hansen,J.B., Hansen,H.F., Straarup,E.M. et al. (2008) Antagonism of microRNA-122 in mice by systemically administered LNA-antimiR leads to up-regulation of a large set of predicted target mRNAs in the liver. Nucleic Acids Res., 36, 1153-1162.
12. Esau,C., Davis,S., Murray,S.F., Yu,X.X., Pandey,S.K., Pear,M., Watts,L., Booten,S.L., Graham,M., McKay,R. et al. (2006) miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting. Cell Metab., 3, 87-98.
13. Krutzfeldt,J., Rajewsky,N., Braich,R., Rajeev,K.G., Tuschl,T., Manoharan,M. and Stoffel,M. (2005) Silencing of microRNAs in vivo with 'antagomirs'. Nature, 438, 685-689.
14. Martin,P. (1995) Ein neuer Zugang zu 2-O-Alkylribonucleosiden und Eigenschaften deren Oligonuclecotide. Helv. Chim. Acta, 78, 486-504.
15. Ross,B. and Song,Q. (2004) US patent no. 20040082775.
16. Ross,B.S., Springer,R.H., Sprankle,K.G. and Vasquez,G. (1997) An efficient and scalable synthesis of arabinosylguanine and 2′-deoxy-2′-fluoroguanosine. Nucleosides Nucleotides, 16 1645-1647.
17. 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.
18. Swayze,E.E., Siwkowski,A.M., Wancewicz,E.V., Migawa,M.T., Wyrzykiewicz,T.K., Hung,G., Monia,B.P. and Bennett,C.F. (2007) Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals. Nucleic Acids Res., 35, 687-700.
19. Puglisi,J.D. and Tinoco,I. Jr. (1989) Absorbance melting curves of RNA. Methods Enzymiol., 180, 304-325.
20. McDowell,J.A. and Turner,D.H. (1996) Investigation of the structural basis for thermodynamic stabilities of tandem GU mismatches: Solution structure of (rGAGGUCUC)2 by two-dimensional NMR and simulated annealing. Biochemistry, 35, 14077-14089.
21. Geary,R.S., Watanabe,T.A., Truong,L., Freier,S., Lesnik,E.A., Sioufi,N.B., Sasmor,H., Manoharan,M. and Levin,A.A. (2001) Pharmacokinetic properties of 2′-O-(2-methoxyethyl)-modified oligonucleotide analogs in rats. J. Pharmacol. Exp. Ther., 296 890-897.
22. Henry,S., Stecker,K., Brooks,D., Monteith,D., Conklin,B. and Bennett,C.F. (2000) Chemically modified oligonucleotides exhibit decreased immune stimulation in mice. J. Pharmacol. Exp. Ther., 292, 468-479.
23. Fabani,M.M. and Gait,M.J. (2008) miR-122 targeting with LNA, 2′-O-methyl oligonucleotide mixmers, peptide nucleic acids (PNA), and PNA-peptide conjugates. RNA, 14, 336-346.