antisense oligoribonucleotides


Summary: Short fragments of RNA that are used to alter the function of target RNAs or DNAs to which they hybridize.

Top Publications

  1. Rimessi P, Sabatelli P, Fabris M, Braghetta P, Bassi E, Spitali P, et al. Cationic PMMA nanoparticles bind and deliver antisense oligoribonucleotides allowing restoration of dystrophin expression in the mdx mouse. Mol Ther. 2009;17:820-7 pubmed publisher
    ..This nonviral approach may valuably improve the therapeutic usage of AONs in DMD as well as the delivery of RNA molecules with many implications in both basic research and medicine. ..
  2. Kurreck J. Antisense technologies. Improvement through novel chemical modifications. Eur J Biochem. 2003;270:1628-44 pubmed
  3. Chalk A, Sonnhammer E. Computational antisense oligo prediction with a neural network model. Bioinformatics. 2002;18:1567-75 pubmed
    ..Using these thresholds the model predicts on average 12 effective AOs per 1000 base pairs, making it a stringent yet practical method for AO prediction. ..
  4. Diop Frimpong B, Prakash T, Rajeev K, Manoharan M, Egli M. Stabilizing contributions of sulfur-modified nucleotides: crystal structure of a DNA duplex with 2'-O-[2-(methoxy)ethyl]-2-thiothymidines. Nucleic Acids Res. 2005;33:5297-307 pubmed
  5. Schumacher N, Borawski J, Leberfinger C, Gessler M, Kerkhoff E. Overlapping expression pattern of the actin organizers Spir-1 and formin-2 in the developing mouse nervous system and the adult brain. Gene Expr Patterns. 2004;4:249-55 pubmed
    ..In mouse embryos both genes are expressed in the developing nervous system. In the adult brain high expression of the genes was found in the Purkinje cells of the cerebellum and in neuronal cells of the hippocampus and dentate gyrus. ..
  6. Zhu S, Xiang J, Li X, Shen S, Lu H, Zhou J, et al. Poly(L-lysine)-modified silica nanoparticles for the delivery of antisense oligonucleotides. Biotechnol Appl Biochem. 2004;39:179-87 pubmed
    ..These results indicated that PMS-NP was a suitable delivery vector for antisense ODN, although its delivery efficiency decreased in the presence of a serum-containing medium. ..
  7. Michel C, Dunn Meynell A, Levin B. Reduced brain CRH and GR mRNA expression precedes obesity in juvenile rats bred for diet-induced obesity. Behav Brain Res. 2004;154:511-7 pubmed
    ..The reduced expression of brain GR and CRH expression at the end of this period might contribute to the propensity of DIO rats to become obese selectively on HE diet after 5 weeks of age. ..
  8. Byon C, Hardy R, Ren C, Ponnazhagan S, Welch D, McDonald J, et al. Free fatty acids enhance breast cancer cell migration through plasminogen activator inhibitor-1 and SMAD4. Lab Invest. 2009;89:1221-8 pubmed publisher
    ..That linoleic acid induces PAI-1 expression in breast cancer cells through SMAD4 provides a novel insight into understanding the relationships between two migration-associated molecules, FFAs, and PAI-1. ..
  9. Zhao F, Zhang Q, Kang C, Cui X, Wang T, Xu P, et al. Suppression of matrix metalloproteinase-9 expression by RNA interference inhibits SGC7901 gastric adenocarcinoma cell growth and invasion in vitro and in vivo. Med Oncol. 2010;27:774-84 pubmed publisher
    ..MMP-9 is a potential therapeutic target for gastric adenocarcinomas. ..

More Information


  1. Grünweller A, Wyszko E, Bieber B, Jahnel R, Erdmann V, Kurreck J. Comparison of different antisense strategies in mammalian cells using locked nucleic acids, 2'-O-methyl RNA, phosphorothioates and small interfering RNA. Nucleic Acids Res. 2003;31:3185-93 pubmed
    ..The high potency of siRNAs and chimeric LNA-DNA oligonucleotides make them valuable candidates for cell culture and in vivo applications targeting the VR1 mRNA. ..
  2. Fernández Fernández J, Andrade Y, Arniges M, Fernandes J, Plata C, Rubio Moscardo F, et al. Functional coupling of TRPV4 cationic channel and large conductance, calcium-dependent potassium channel in human bronchial epithelial cell lines. Pflugers Arch. 2008;457:149-59 pubmed publisher
  3. Keck M, Holsboer F. Hyperactivity of CRH neuronal circuits as a target for therapeutic interventions in affective disorders. Peptides. 2001;22:835-44 pubmed
    ..Recent clinical observations support this possibility. ..
  4. Lee D, Gelein R, Opanashuk L. Heme-oxygenase-1 promotes polychlorinated biphenyl mixture aroclor 1254-induced oxidative stress and dopaminergic cell injury. Toxicol Sci. 2006;90:159-67 pubmed
    ..These observations suggest that enhanced HO-1 catalytic activity and subsequent liberation of Fe participate in neurotoxic DAergic cell injury caused by A1254 exposure in vitro. ..
  5. Ramon A, Bertrand J, Malvy C. Delivery of small interfering RNA. A review and an example of application to a junction oncogene. Tumori. 2008;94:254-63 pubmed
    ..Indeed, many studies have shown that small interfering RNA and antisense oligonucleotides are made efficient by various innovative delivery methods and, under these conditions, offer a powerful new therapeutic tool in cancer treatment. ..
  6. Rossi A, Maione P, Colantuoni G, Guerriero C, Gridelli C. The role of new targeted therapies in small-cell lung cancer. Crit Rev Oncol Hematol. 2004;51:45-53 pubmed
    ..To date, no targeted therapy has been approved for use in the treatment of patients with SCLC. This review will focus on the main novel biologic agents investigated in the treatment of SCLC. ..
  7. Geigenberger P, Regierer B, Nunes Nesi A, Leisse A, Urbanczyk Wochniak E, Springer F, et al. Inhibition of de novo pyrimidine synthesis in growing potato tubers leads to a compensatory stimulation of the pyrimidine salvage pathway and a subsequent increase in biosynthetic performance. Plant Cell. 2005;17:2077-88 pubmed
    ..This results in increased uridine nucleotide pool levels in tubers and improved biosynthetic performance. ..
  8. Park J, Hahn E, Kong J, Cho H, Yoon C, Cheong S, et al. TCDD-induced apoptosis in EL-4 cells deficient of the aryl hydrocarbon receptor and down-regulation of IGFBP-6 prevented the apoptotic cell death. Toxicol Lett. 2003;145:55-68 pubmed
    ..Interestingly, the effects of TCDD were exerted without aromatic hydrocarbon (Ah) receptor (AhR). Taken together, the results have shown that IGFBP-6 mediates the immunotoxic effects of TCDD in EL-4 cells in an AhR-independent pathway. ..
  9. Bogen O, Dina O, Gear R, Levine J. Dependence of monocyte chemoattractant protein 1 induced hyperalgesia on the isolectin B4-binding protein versican. Neuroscience. 2009;159:780-6 pubmed publisher
  10. Yacyshyn B, Bowen Yacyshyn M, Shanahan W. The clinical experience of antisense therapy to ICAM-1 in Crohn's disease. Curr Opin Mol Ther. 1999;1:332-5 pubmed
    ..This 20 base phosphorothioate hybridizes to a sequence in the 3' untranslated region of human ICAM-1 mRNA. This substrate is cleaved by RNase H, resulting in specific message and protein reduction 11,21. ..
  11. Dan Q, Wong R, Chung S, Chung S, Lam K. Interaction between the polyol pathway and non-enzymatic glycation on aortic smooth muscle cell migration and monocyte adhesion. Life Sci. 2004;76:445-59 pubmed
    ..The greater AGE-induced changes in the TG SMCs have provided further support for the dependency of such changes on polyol pathway hyperactivity. ..
  12. Chen J, Lovell J, Lo P, Stefflova K, Niedre M, Wilson B, et al. A tumor mRNA-triggered photodynamic molecular beacon based on oligonucleotide hairpin control of singlet oxygen production. Photochem Photobiol Sci. 2008;7:775-81 pubmed publisher
    ..When incubated with c-raf-1 expressing MDA-MB-231 cancer cells, the PMB displayed efficient cellular uptake and subsequently effective PDT activation in targeted cells. ..
  13. Matter N, König H. Targeted 'knockdown' of spliceosome function in mammalian cells. Nucleic Acids Res. 2005;33:e41 pubmed
    ..The data provide the first evidence for a role of distinct spliceosomes in pre-mRNA splicing from endogenous mammalian genes and establish a tool to define roles for the different splicing machineries in vivo. ..
  14. Sloop K, Showalter A, Cox A, Cao J, Siesky A, Zhang H, et al. Specific reduction of hepatic glucose 6-phosphate transporter-1 ameliorates diabetes while avoiding complications of glycogen storage disease. J Biol Chem. 2007;282:19113-21 pubmed
    ..Together, these results demonstrate that effective glucose lowering by G6PT1 inhibitors can be achieved without adversely affecting carbohydrate and lipid metabolism. ..
  15. Zhou J, Yu Q, Zou T. Alternative splicing of exon 10 in the tau gene as a target for treatment of tauopathies. BMC Neurosci. 2008;9 Suppl 2:S10 pubmed publisher
    ..In this review, we will discuss how realistic, specific biological molecules can be found to regulate exon 10 splicing in the tau gene for potential treatment of tauopathies. ..
  16. Persson A, Bull C, Eriksson P. Requirement for Id1 in opioid-induced oligodendrogenesis in cultured adult rat hippocampal progenitors. Eur J Neurosci. 2006;23:2277-88 pubmed
    ..We suggest a requirement for Id1 in opioid-induced oligodendrogenesis in cultured AHPs possibly acting on opioid-responsive AHPs expressing the proneural transcriptional activator Mash1. ..
  17. Sakai A, Asada M, Seno N, Suzuki H. Involvement of neural cell adhesion molecule signaling in glial cell line-derived neurotrophic factor-induced analgesia in a rat model of neuropathic pain. Pain. 2008;137:378-88 pubmed
    ..These findings suggest that NCAM signaling plays a critical role in the analgesic effect of GDNF and that development of new drugs activating GDNF-NCAM signaling may represent a new strategy for the relief of intractable pain. ..
  18. Nakamura Y, Makabe K, Nishida H. The functional analysis of Type I postplasmic/PEM mRNAs in embryos of the ascidian Halocynthia roretzi. Dev Genes Evol. 2006;216:69-80 pubmed
    ..On the other hand, Hr-PEM3 and Hr-PEN1 MOs seemed to have no effect. We conclude that the functions of at least some localized maternal Type I postplasmic/PEM mRNAs are necessary for early embryonic patterning in ascidians. ..
  19. Wagner N, Wagner K, Schley G, Coupland S, Heimann H, Grantyn R, et al. The Wilms' tumor suppressor Wt1 is associated with the differentiation of retinoblastoma cells. Cell Growth Differ. 2002;13:297-305 pubmed
    ..These findings suggest a role for Wt1 in the differentiation of retinoblastoma cells. Furthermore, Wt1 expression in retinoblastoma may reflect the potential of these tumors to initiate the early steps of neuronal differentiation. ..
  20. Mouchel N, Henstra S, McCarthy V, Williams S, Phylactides M, Harris A. HNF1alpha is involved in tissue-specific regulation of CFTR gene expression. Biochem J. 2004;378:909-18 pubmed
    ..This is the first report of a transcription factor, which confers tissue specificity on the expression of this important disease-associated gene. ..
  21. Bowen Yacyshyn M, Bennett C, Nation N, Rayner D, Yacyshyn B. Amelioration of chronic and spontaneous intestinal inflammation with an antisense oligonucleotide (ISIS 9125) to intracellular adhesion molecule-1 in the HLA-B27/beta2 microglobulin transgenic rat model. J Pharmacol Exp Ther. 2002;302:908-17 pubmed
  22. Hafid Medheb K, Augery Bourget Y, Minatchy M, Hanania N, Robert Lézénès J. Bcl-XL is required for heme synthesis during the chemical induction of erythroid differentiation of murine erythroleukemia cells independently of its antiapoptotic function. Blood. 2003;101:2575-83 pubmed
    ..Finally, Bcl-X(L) localized to mitochondria during MEL erythroid differentiation, suggesting that it may mediate a critical mitochondrial transport function related to heme biosynthesis. ..
  23. Du Q, Wang X, Chen X, Zheng W, Wang S. Antitumor mechanism of antisense cantide targeting human telomerase reverse transcriptase. World J Gastroenterol. 2003;9:2030-5 pubmed
    ..The rapid inhibitory effect of cantide on tumor growth demonstrates its feasibility in cancer treatment. ..
  24. Wada S, Yamada L, Kobayashi K, Shoguchi E, Satoh N. [Morpholino oligonucleotide-based screen of novel genes with developmental function]. Seikagaku. 2003;75:617-20 pubmed
  25. Maggiolini M, Vivacqua A, Fasanella G, Recchia A, Sisci D, Pezzi V, et al. The G protein-coupled receptor GPR30 mediates c-fos up-regulation by 17beta-estradiol and phytoestrogens in breast cancer cells. J Biol Chem. 2004;279:27008-16 pubmed
    ..The ability of E2 and phytoestrogens to regulate the expression of growth-related genes such as c-fos even in the absence of ER has interesting implications for understanding breast cancer progression. ..
  26. Pavelic K, Spaventi S, Gluncic V, Matejcic A, Pavicic D, Karapandza N, et al. The expression and role of insulin-like growth factor II in malignant hemangiopericytomas. J Mol Med (Berl). 1999;77:865-9 pubmed
    ..Our data suggest that tumor cells produce IGF II, and that this in turn stimulates their proliferation by autocrine mechanisms. ..
  27. Werling U, Siehler S, Litfin M, Nau H, Gottlicher M. Induction of differentiation in F9 cells and activation of peroxisome proliferator-activated receptor delta by valproic acid and its teratogenic derivatives. Mol Pharmacol. 2001;59:1269-76 pubmed
    ..In conclusion, our data show that VPA and its teratogenic derivatives induce a specific type of F9 cell differentiation and that PPARdelta is a limiting factor in the control of differentiation. ..
  28. Gonzalo T, Clemente M, Chonco L, Weber N, Diaz L, Serramía M, et al. Gene therapy in HIV-infected cells to decrease viral impact by using an alternative delivery method. ChemMedChem. 2010;5:921-9 pubmed publisher
    ..These results suggest that NN16 dendrimers have the potential to be used as a versatile non-viral vector for gene therapy against HIV infection. ..
  29. Liang C, Chen J, Hsu Y, Ou J, Chang Y. Epstein-Barr virus BZLF1 gene is activated by transforming growth factor-beta through cooperativity of Smads and c-Jun/c-Fos proteins. J Biol Chem. 2002;277:23345-57 pubmed
    ..Those findings also suggest a role of TGF-beta in EBV-associated diseases. ..
  30. Henson E, Hu X, Gibson S. Herceptin sensitizes ErbB2-overexpressing cells to apoptosis by reducing antiapoptotic Mcl-1 expression. Clin Cancer Res. 2006;12:845-53 pubmed
    ..Taken together, these results suggest that herceptin sensitizes ErbB2-overexpressing cells to apoptosis by reducing antiapoptotic Mcl-1 protein levels. ..
  31. Cho Y, Cho Chung Y. Antisense protein kinase A RIalpha acts synergistically with hydroxycamptothecin to inhibit growth and induce apoptosis in human cancer cells: molecular basis for combinatorial therapy. Clin Cancer Res. 2003;9:1171-8 pubmed
    ..These results demonstrated the molecular basis for the use of Gem231 and HCPT as combinatorial therapy to treat human cancer. ..
  32. Nicolson G, Nawa A, Toh Y, Taniguchi S, Nishimori K, Moustafa A. Tumor metastasis-associated human MTA1 gene and its MTA1 protein product: role in epithelial cancer cell invasion, proliferation and nuclear regulation. Clin Exp Metastasis. 2003;20:19-24 pubmed
    ..The results suggest that the MTA1 protein may serve multiple functions in cellular signaling, chromosome remodeling and transcription processes that are important in the progression, invasion and growth of metastatic epithelial cells. ..
  33. McNulty S, Fonfria E. The role of TRPM channels in cell death. Pflugers Arch. 2005;451:235-42 pubmed
    ..Finally, we discuss the possibility that TRPM channels may provide therapeutic targets for degenerative diseases involving oxidative stress-related pathologies including diabetes and Alzheimer's disease. ..
  34. Zhang R, Xiao M, Zhao S. [Effects of dentin sialophosphoprotein antisense oligodeoxynucleotide on ultrastructure of mouse tooth germ]. Hua Xi Kou Qiang Yi Xue Za Zhi. 2003;21:281-3 pubmed
    ..These findings indicated that DSPP played an important role in keeping tooth normal development, as well as in dentin mineralization by maintaining odontoblasts' secreting ability and controlling fiber structure and orientation. ..
  35. Miyasaka Y, Enomoto N, Kurosaki M, Sakamoto N, Kanazawa N, Kohashi T, et al. Hepatitis C virus nonstructural protein 5A inhibits tumor necrosis factor-alpha-mediated apoptosis in Huh7 cells. J Infect Dis. 2003;188:1537-44 pubmed
    ..These findings suggest that the NS5A protein inhibits the apoptotic effect of TNF-alpha upstream of caspase-8 in the apoptosis cascade. ..
  36. Duboc V, Lapraz F, Saudemont A, Bessodes N, Mekpoh F, Haillot E, et al. Nodal and BMP2/4 pattern the mesoderm and endoderm during development of the sea urchin embryo. Development. 2010;137:223-35 pubmed publisher
  37. Hattori T, von der Mark K, Kawaki H, Yutani Y, Kubota S, Nakanishi T, et al. Downregulation of rheumatoid arthritis-related antigen RA-A47 (HSP47/colligin-2) in chondrocytic cell lines induces apoptosis and cell-surface expression of RA-A47 in association with CD9. J Cell Physiol. 2005;202:191-204 pubmed
    ..The surface-exposed RA-A47 may induce autoantibodies and inflammatory reactions in autoimmune disease situations such as rheumatoid arthritis. ..
  38. Akakura S, Singh S, Spataro M, Akakura R, Kim J, Albert M, et al. The opsonin MFG-E8 is a ligand for the alphavbeta5 integrin and triggers DOCK180-dependent Rac1 activation for the phagocytosis of apoptotic cells. Exp Cell Res. 2004;292:403-16 pubmed
  39. Xu B, Bhattacharjee A, Roy B, Feldman G, Cathcart M. Role of protein kinase C isoforms in the regulation of interleukin-13-induced 15-lipoxygenase gene expression in human monocytes. J Biol Chem. 2004;279:15954-60 pubmed
    ..Our findings indicate that PKCdelta plays an important role in regulating IL-13-induced 15-LO expression in human monocytes and subsequently modulates the inflammatory responses mediated by 15-LO products. ..
  40. Mann C, Honeyman K, Cheng A, Ly T, Lloyd F, Fletcher S, et al. Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc Natl Acad Sci U S A. 2001;98:42-7 pubmed
    ..We report an alternative approach where 2'-O-methyl antisense oligoribonucleotides have been used to modify processing of the dystrophin pre-mRNA in the mdx mouse model of DMD...
  41. Helderman van den Enden A, Straathof C, Aartsma Rus A, den Dunnen J, Verbist B, Bakker E, et al. Becker muscular dystrophy patients with deletions around exon 51; a promising outlook for exon skipping therapy in Duchenne patients. Neuromuscul Disord. 2010;20:251-4 pubmed publisher
    ..The mild phenotype encourages further development of exon 51 skipping therapy. ..
  42. Prater C, Saleh A, Wear M, Miller P. Allosteric inhibition of the HIV-1 Rev/RRE interaction by a 3'-methylphosphonate modified antisense oligo-2'-O-methylribonucleotide. Oligonucleotides. 2007;17:275-90 pubmed
    ..These results are consistent with a mechanism by which 2-1mp blocks access of Rev to the RRE/CAT transcript thus preventing nuclear export and subsequent translation. ..
  43. Liu N, Ding H, Vanderheyden J, Zhu Z, Zhang Y. Radiolabeling small RNA with technetium-99m for visualizing cellular delivery and mouse biodistribution. Nucl Med Biol. 2007;34:399-404 pubmed
    ..The results of this study could open up a new approach for measuring the in vivo delivery of small RNAs that might further facilitate the development of siRNAs as targeted therapies. ..
  44. Wu M, Sherwin T, Brown W, Stockley P. Delivery of antisense oligonucleotides to leukemia cells by RNA bacteriophage capsids. Nanomedicine. 2005;1:67-76 pubmed
    ..The findings suggest that RNA phage VLPs may be useful as a new nanomaterial for targeted delivery of antisense ODNs, or other macromolecular drug candidates. ..
  45. Bo X, Lou S, Sun D, Shu W, Yang J, Wang S. Selection of antisense oligonucleotides based on multiple predicted target mRNA structures. BMC Bioinformatics. 2006;7:122 pubmed
    ..The results of this paper imply that some features characterizing multiple predicted target site structures can be used to predict ODN efficacy. ..
  46. Lin R, Tuo C, Lu Q, Zhang W, Wang S. Inhibition of tumor growth and metastasis with antisense oligonucleotides (Cantide) targeting hTERT in an in situ human hepatocellular carcinoma model. Acta Pharmacol Sin. 2005;26:762-8 pubmed
    ..These results demonstrated that Cantide was an effective antitumor antisense oligonucleotide in vivo and has the potential to be developed into a clinical anti-cancer drug. ..
  47. Svensson C, Fitzsimmons B, Azizi S, Powell H, Hua X, Yaksh T. Spinal p38beta isoform mediates tissue injury-induced hyperalgesia and spinal sensitization. J Neurochem. 2005;92:1508-20 pubmed
    ..Thus, spinal p38beta, probably in microglia, plays a significant role in spinal nociceptive processing and represents a potential target for pain therapy. ..
  48. Crouch S, Spidel C, Lindsey J. HGF and ligation of alphavbeta5 integrin induce a novel, cancer cell-specific gene expression required for cell scattering. Exp Cell Res. 2004;292:274-87 pubmed
    ..These results are the first to demonstrate that Mig-7 expression may be used as a cancer cell-specific target to inhibit cell scattering. ..
  49. McCracken M, Miraglia L, McKay R, Strobl J. Protein kinase C delta is a prosurvival factor in human breast tumor cell lines. Mol Cancer Ther. 2003;2:273-81 pubmed
    ..5 Gy radiation-treated cells. We conclude that PKC delta acts as a prosurvival factor in human breast tumor cells in vitro. ..
  50. Darken R, Scola A, Rakeman A, Das G, Mlodzik M, Wilson P. The planar polarity gene strabismus regulates convergent extension movements in Xenopus. EMBO J. 2002;21:976-85 pubmed
    ..Thus our results strongly support the emerging hypothesis that a vertebrate analog of the planar polarity pathway governs convergent extension movements. ..
  51. Stierle V, Laigle A, Jolles B. Modulation of the efficiency of a siRNA directed against MDR1 expression in MCF7-R cells when combined with a second siRNA. Biochimie. 2007;89:1033-6 pubmed
    ..It is suggested that designing efficient siRNAs from thermodynamic characteristics is favored when siRNAs are incorporated into the RISC Loading Complex (RLC) rather than directly loaded into RISC. ..
  52. Ding J, Li D, Wang X, Wang C, Wu T. Fibronectin promotes invasiveness and focal adhesion kinase tyrosine phosphorylation of human colon cancer cell. Hepatogastroenterology. 2008;55:2072-6 pubmed
    ..Fibronectin can promote invasion of Colo320 cells, FAK tyrosine phosphorylation plays a key role in this process. Inhibition of FAK expression can inhibit colon cancer cell invasion. ..
  53. Radecke F, Radecke S, Schwarz K. Unmodified oligodeoxynucleotides require single-strandedness to induce targeted repair of a chromosomal EGFP gene. J Gene Med. 2004;6:1257-71 pubmed
    ..In concert with the gene copy number correlation, functional interaction between the repair molecule and the target site seems to be one bottleneck in targeted gene repair. ..