A direct comparison of anti-microRNA oligonucleotide potency

Pharmaceutical Research

Volumn 27, Issue 9, 2010, Pages 1788-1799

  Lennox, Kim A ^a   Behlke, Mark A ^a  

^a INTEGRATED DNA TECHNOLOGIES   (United States)

Author keywords

antagonist; antisense; LNA; miRNA; potency

Indexed keywords

MICRORNA OLIGONUCLEOTIDE ANTIBODY; RNA ANTIBODY; UNCLASSIFIED DRUG;

ANIMAL TISSUE; ARTICLE; BINDING AFFINITY; BINDING SITE; CHEMICAL MODIFICATION; COMPARATIVE STUDY; CONTROLLED STUDY; CROSS LINKING; DOWN REGULATION; DRUG ACTIVITY; DRUG CROSS REACTIVITY; DRUG DESIGN; DRUG POTENCY; DRUG PROTEIN BINDING; DRUG SPECIFICITY; DRUG STABILITY; DRUG SYNTHESIS; ELECTROSPRAY MASS SPECTROMETRY; HUMAN; HUMAN CELL; IN VITRO STUDY; MALE; MOUSE; NONHUMAN; PRIORITY JOURNAL;

ANIMALS; BASE SEQUENCE; BINDING SITES; CELL CULTURE TECHNIQUES; CHROMATOGRAPHY, HIGH PRESSURE LIQUID; ENZYME STABILITY; HELA CELLS; HUMANS; LUCIFERASES, FIREFLY; LUCIFERASES, RENILLA; MALE; MICE; MICRORNAS; MOLECULAR SEQUENCE DATA; OLIGONUCLEOTIDES, ANTISENSE; PLASMIDS; RIBONUCLEASES; SPECTROMETRY, MASS, ELECTROSPRAY IONIZATION; TRANSFECTION;

EID: 77955469975     PISSN: 07248741     EISSN: 1573904X     Source Type: Journal    
DOI: 10.1007/s11095-010-0156-0     Document Type: Article

References (54)

1. RW Carthew EJ Sontheimer 2009 Origins and mechanisms of miRNAs and siRNAs Cell. 136 642 655 10.1016/j.cell.2009.01.035 1:CAS:528:DC%2BD1MXkvFGksb0%3D 19239886
2. H Vaucheret 2006 Post-transcriptional small RNA pathways in plants: mechanisms and regulations Genes Dev. 20 759 771 10.1101/gad.1410506 1:CAS:528:DC%2BD28XjsFGjsr0%3D 16600909
3. BP Lewis CB Burge DP Bartel 2005 Conserved seed pairing, often flanked by adenosines, indicates that thousands of human genes are microRNA targets Cell. 120 15 20 10.1016/j.cell.2004.12.035 1:CAS:528:DC%2BD2MXot1ChsA%3D%3D 15652477 (Pubitemid 40094598)
4. A Krek D Grun MN Poy R Wolf L Rosenberg EJ Epstein, et al. 2005 Combinatorial microRNA target predictions Nat Genet. 37 495 500 10.1038/ng1536 1:CAS:528:DC%2BD2MXjsF2ksrw%3D 15806104 (Pubitemid 40617277)
5. I Alvarez-Garcia EA Miska 2005 MicroRNA functions in animal development and human disease Development. 132 4653 4662 10.1242/dev.02073 1:CAS:528:DC%2BD2MXht12gsbjI 16224045 (Pubitemid 41685276)
6. RJ Johnston O Hobert 2003 A microRNA controlling left/right neuronal asymmetry in Caenorhabditis elegans Nature. 426 845 849 10.1038/nature02255 1:CAS:528:DC%2BD3sXpvVGmtbg%3D 14685240 (Pubitemid 38056870)
7. BR Wilfred WX Wang PT Nelson 2007 Energizing miRNA research: a review of the role of miRNAs in lipid metabolism, with a prediction that miR-103/107 regulates human metabolic pathways Mol Genet Metab. 91 209 217 10.1016/j.ymgme.2007.03.011 1:CAS:528:DC%2BD2sXmtVejsL8%3D 17521938 (Pubitemid 46819124)
8. MA Lindsay 2008 microRNAs and the immune response Trends Immunol. 29 343 351 10.1016/j.it.2008.04.004 1:CAS:528:DC%2BD1cXnvVGlsb0%3D 18515182
9. PT Nelson WX Wang BW Rajeev 2008 MicroRNAs (miRNAs) in neurodegenerative diseases Brain Pathol. 18 130 138 10.1111/j.1750-3639.2007.00120.x 1:CAS:528:DC%2BD1cXjtFKru7c%3D 18226108
10. N Bushati SM Cohen 2008 MicroRNAs in neurodegeneration Curr Opin Neurobiol. 18 292 296 10.1016/j.conb.2008.07.001 1:CAS:528:DC%2BD1cXhtlSgsr%2FK 18662781
11. T Thum D Catalucci J Bauersachs 2008 MicroRNAs: novel regulators in cardiac development and disease Cardiovasc Res. 79 562 570 10.1093/cvr/cvn137 1:CAS:528:DC%2BD1cXhtFChsrzP 18511432
12. C Xiao K Rajewsky 2009 MicroRNA control in the immune system: basic principles Cell. 136 26 36 10.1016/j.cell.2008.12.027 1:CAS:528: DC%2BD1MXhtFOisbw%3D 19135886
13. A Esquela-Kerscher FJ Slack 2006 Oncomirs-microRNAs with a role in cancer Nat Rev Cancer. 6 259 269 10.1038/nrc1840 1:CAS:528:DC%2BD28XivVyqtrs%3D 16557279
14. EA Wiemer 2007 The role of microRNAs in cancer: no small matter Eur J Cancer. 43 1529 1544 10.1016/j.ejca.2007.04.002 1:CAS:528:DC%2BD2sXmsFKnsr0%3D 17531469 (Pubitemid 46901017)
15. CM Croce 2009 Causes and consequences of microRNA dysregulation in cancer Nat Rev Genet. 10 704 714 10.1038/nrg2634 1:CAS:528:DC%2BD1MXhtFCqurjE 19763153
16. Y Lee C Ahn J Han H Choi J Kim J Yim, et al. 2003 The nuclear RNase III Drosha initiates microRNA processing Nature. 425 415 419 10.1038/nature01957 1:CAS:528:DC%2BD3sXnsV2kt7s%3D 14508493 (Pubitemid 37187272)
17. E Lund S Guttinger A Calado JE Dahlberg U Kutay 2004 Nuclear export of microRNA precursors Science. 303 95 98 10.1126/science.1090599 1:CAS:528:DC%2BD3sXhtVWhs73O 14631048 (Pubitemid 38055779)
18. A Schaefer D O'Carroll CL Tan D Hillman M Sugimori R Llinas, et al. 2007 Cerebellar neurodegeneration in the absence of microRNAs J Exp Med. 204 1553 1558 10.1084/jem.20070823 1:CAS:528:DC%2BD2sXnslylsr8%3D 17606634 (Pubitemid 47048023)
19. X Cai CH Hagedorn BR Cullen 2004 Human microRNAs are processed from capped, polyadenylated transcripts that can also function as mRNAs RNA. 10 1957 1966 10.1261/rna.7135204 1:CAS:528:DC%2BD2cXhtVGisLjM 15525708 (Pubitemid 39557833)
20. J Krutzfeldt N Rajewsky R Braich KG Rajeev T Tuschl M Manoharan, et al. 2005 Silencing of microRNAs in vivo with 'antagomirs' Nature. 438 685 689 10.1038/nature04303 16258535 (Pubitemid 41740580)
21. S Davis B Lollo S Freier C Esau 2006 Improved targeting of miRNA with antisense oligonucleotides Nucleic Acids Res. 34 2294 2304 10.1093/nar/gkl183 1:CAS:528:DC%2BD28Xks1Smt7o%3D 16690972 (Pubitemid 43985667)
22. KA Lennox JL Sabel MJ Johnson BG Moreira CA Fletcher SD Rose, et al. 2006 Characterization of modified antisense oligonucleotides in Xenopus laevis embryos Oligonucleotides. 16 26 42 10.1089/oli.2006.16.26 1:CAS:528: DC%2BD28XjtVOltbo%3D 16584293
23. DA Brown SH Kang SM Gryaznov L DeDionisio O Heidenreich S Sullivan, et al. 1994 Effect of phosphorothioate modification of oligodeoxynucleotides on specific protein binding J Biol Chem. 269 26801 26805 1:CAS:528: DyaK2cXmslClsb0%3D 7929417
24. RS Geary TA Watanabe L Truong S Freier EA Lesnik NB Sioufi, et al. 2001 Pharmacokinetic properties of 2′-O-(2-methoxyethyl)-modified oligonucleotide analogs in rats J Pharmacol Exp Ther. 296 890 897 1:CAS:528:DC%2BD3MXhtlOksrY%3D 11181921
25. SM Freier KH Altmann 1997 The ups and downs of nucleic acid duplex stability: structure-stability studies on chemically-modified DNA:RNA duplexes Nucleic Acids Res. 25 4429 4443 10.1093/nar/25.22.4429 1:CAS:528: DyaK1cXhsVCnsQ%3D%3D 9358149
26. M Petersen K Bondensgaard J Wengel JP Jacobsen 2002 Locked nucleic acid (LNA) recognition of RNA: NMR solution structures of LNA:RNA hybrids J Am Chem Soc. 124 5974 5982 10.1021/ja012288d 1:CAS:528:DC%2BD38Xjtlaksbg%3D 12022830 (Pubitemid 34555398)
27. J Kurreck E Wyszko C Gillen VA Erdmann 2002 Design of antisense oligonucleotides stabilized by locked nucleic acids Nucleic Acids Res. 30 1911 1918 10.1093/nar/30.9.1911 1:CAS:528:DC%2BD38XktFGhtLY%3D 11972327
28. R Crinelli M Bianchi L Gentilini M Magnani 2002 Design and characterization of decoy oligonucleotides containing locked nucleic acids Nucleic Acids Res. 30 2435 2443 10.1093/nar/30.11.2435 1:CAS:528: DC%2BD38XkvV2hs74%3D 12034831
29. M Frieden HF Hansen T Koch 2003 Nuclease stability of LNA oligonucleotides and LNA-DNA chimeras Nucleos Nucleot Nucleic Acids. 22 1041 1043 10.1081/NCN-120022731 1:CAS:528:DC%2BD3sXntFCgsbc%3D (Pubitemid 37139318)
30. M Petersen J Wengel 2003 LNA: a versatile tool for therapeutics and genomics Trends Biotechnol. 21 74 81 10.1016/S0167-7799(02)00038-0 1:CAS:528:DC%2BD3sXptlynsw%3D%3D 12573856 (Pubitemid 36165007)
31. AM Cheng MW Byrom J Shelton LP Ford 2005 Antisense inhibition of human miRNAs and indications for an involvement of miRNA in cell growth and apoptosis Nucleic Acids Res. 33 1290 1297 10.1093/nar/gki200 1:CAS:528: DC%2BD2MXit1Knu7s%3D 15741182 (Pubitemid 41439922)
32. G Hutvagner MJ Simard CC Mello PD Zamore 2004 Sequence-specific inhibition of small RNA function PLoS Biol. 2 E98 10.1371/journal.pbio.0020098 15024405 (Pubitemid 39234504)
33. G Meister M Landthaler Y Dorsett T Tuschl 2004 Sequence-specific inhibition of microRNA- and siRNA-induced RNA silencing Rna. 10 544 550 10.1261/rna.5235104 1:CAS:528:DC%2BD2cXhslOlsrw%3D 14970398 (Pubitemid 38241013)
34. UA Orom S Kauppinen AH Lund 2006 LNA-modified oligonucleotides mediate specific inhibition of microRNA function Gene. 372 137 141 10.1016/j.gene.2005. 12.031 1:CAS:528:DC%2BD28XjslOqtrs%3D 16503100
35. A Vermeulen B Robertson AB Dalby WS Marshall J Karpilow D Leake, et al. 2007 Double-stranded regions are essential design components of potent inhibitors of RISC function Rna. 13 723 730 10.1261/rna.448107 1:CAS:528:DC%2BD2sXltVakurc%3D 17400817 (Pubitemid 46641531)
36. C Esau S Davis SF Murray XX Yu SK Pandey M Pear, et al. 2006 miR-122 regulation of lipid metabolism revealed by in vivo antisense targeting Cell Metab. 3 87 98 10.1016/j.cmet.2006.01.005 1:CAS:528:DC%2BD28XhvFyjt7c%3D 16459310
37. WP Kloosterman AK Lagendijk RF Ketting JD Moulton RH Plasterk 2007 Targeted inhibition of miRNA maturation with morpholinos reveals a role for miR-375 in pancreatic islet development PLoS Biol. 5 e203 10.1371/journal.pbio. 0050203 17676975 (Pubitemid 47328277)
38. D Leaman PY Chen J Fak A Yalcin M Pearce U Unnerstall, et al. 2005 Antisense-mediated depletion reveals essential and specific functions of microRNAs in Drosophila development Cell. 121 1097 1108 10.1016/j.cell.2005.04. 016 1:CAS:528:DC%2BD2MXmtF2ntr0%3D 15989958 (Pubitemid 40884399)
39. J Elmen M Lindow A Silahtaroglu M Bak M Christensen A Lind-Thomsen, 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 10.1093/nar/gkm1113 1:CAS:528:DC%2BD1cXislKiu7k%3D 18158304 (Pubitemid 351330933)
40. J Elmen M Lindow S Schutz M Lawrence A Petri S Obad, et al. 2008 LNA-mediated microRNA silencing in non-human primates Nature. 452 896 899 10.1038/nature06783 1:CAS:528:DC%2BD1cXkvVSis7w%3D 18368051 (Pubitemid 351550859)
41. S Davis S Propp SM Freier LE Jones MJ Serra G Kinberger, et al. 2009 Potent inhibition of microRNA in vivo without degradation Nucleic Acids Res. 37 70 77 10.1093/nar/gkn904 1:CAS:528:DC%2BD1MXktlentg%3D%3D 19015151
42. J Kurreck 2003 Antisense technologies. Improvement through novel chemical modifications Eur J Biochem. 270 1628 1644 10.1046/j.1432-1033.2003.03555.x 1:CAS:528:DC%2BD3sXjtl2jtbc%3D 12694176 (Pubitemid 36532522)
43. PS Eder RJ DeVine JM Dagle JA Walder 1991 Substrate specificity and kinetics of degradation of antisense oligonucleotides by a 3′ exonuclease in plasma Antisense Res Dev. 1 141 151 1:CAS:528:DyaK3sXitVyhtbY%3D 1841656
44. JM Dagle DL Weeks JA Walder 1991 Pathways of degradation and mechanism of action of antisense oligonucleotides in Xenopus laevis embryos Antisense Res Dev. 1 11 20 1:CAS:528:DyaK3MXltFWjsr0%3D 1668307
45. M Koziolkiewicz M Wojcik A Kobylanska B Karwowski B Rebowska P Guga, et al. 1997 Stability of stereoregular oligo(nucleoside phosphorothioate)s in human plasma: diastereoselectivity of plasma 3′-exonuclease Antisense Nucleic Acid Drug Dev. 7 43 48 1:CAS:528:DyaK2sXhsVKitL0%3D 9055038 (Pubitemid 27132658)
46. M Gilar A Belenky Y Budman DL Smisek AS Cohen 1998 Impact of 3′-exonuclease stereoselectivity on the kinetics of phosphorothioate oligonucleotide metabolism Antisense Nucleic Acid Drug Dev. 8 35 42 1:CAS:528:DyaK1cXhsFymtbc%3D 9512094
47. J Krutzfeldt S Kuwajima R Braich KG Rajeev J Pena T Tuschl, et al. 2007 Specificity, duplex degradation and subcellular localization of antagomirs Nucleic Acids Res. 35 2885 2892 10.1093/nar/gkm024 1:CAS:528: DC%2BD2sXmvF2ntrg%3D 17439965 (Pubitemid 47073627)
48. JY Tang J Temsamani S Agrawal 1993 Self-stabilized antisense oligodeoxynucleotide phosphorothioates: properties and anti-HIV activity Nucleic Acids Res. 21 2729 2735 10.1093/nar/21.11.2729 1:CAS:528:DyaK3sXmsVSqsbk%3D 8392706
49. K Hosono T Kuwasaki S Tsukahara K Takai H Takaku 1995 Properties of base-pairing in the stem region of hairpin antisense oligonucleotides containing 2′-methoxynucleosides Biochim Biophys Acta. 1244 339 344 1:STN:280:DyaK2Mzit1Ciug%3D%3D 7599153
50. RE Lanford ES Hildebrandt-Eriksen A Petri R Persson M Lindow ME Munk, et al. 2010 Therapeutic silencing of microRNA-122 in primates with chronic hepatitis C virus infection Science. 327 198 201 10.1126/science.1178178 1:CAS:528:DC%2BC3cXht1egsA%3D%3D 19965718
51. JA Chan AM Krichevsky KS Kosik 2005 MicroRNA-21 is an antiapoptotic factor in human glioblastoma cells Cancer Res. 65 6029 6033 10.1158/0008-5472. CAN-05-0137 1:CAS:528:DC%2BD2MXmt1eht7c%3D 16024602 (Pubitemid 40994386)
52. J Weiler J Hunziker J Hall 2006 Anti-miRNA oligonucleotides (AMOs): ammunition to target miRNAs implicated in human disease? Gene Ther. 13 496 502 10.1038/sj.gt.3302654 1:CAS:528:DC%2BD28XitVGnurg%3D 16195701
53. Y You BG Moreira MA Behlke R Owczarzy 2006 Design of LNA probes that improve mismatch discrimination Nucleic Acids Res. 34 e60 10.1093/nar/gkl175 16670427 (Pubitemid 43985690)
54. DP Bartel 2009 MicroRNAs: target recognition and regulatory functions Cell. 136 215 233 10.1016/j.cell.2009.01.002 1:CAS:528:DC%2BD1MXhs1Kiuro%3D 19167326