Chemical modification of gene silencing oligonucleotides for drug discovery and development

Drug Discovery Today

Volumn 10, Issue 8, 2005, Pages 587-593

  Chen, Xiaolan ^a   Dudgeon, Nancy ^a   Shen, Long ^a   Wang, Jui H ^a  

^a UNIVERSITY AT BUFFALO   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGODEOXYNUCLEOTIDE; GENE EXPRESSION MODULATOR 231; OLIGODEOXYNUCLEOTIDE PHOSPHOROTHIOATE; OLIGONUCLEOTIDE; OLIGORIBONUCLEOTIDE;

BINDING AFFINITY; CANCER INHIBITION; CANCER MORTALITY; CHEMICAL MODIFICATION; CLINICAL TRIAL; DRUG DELIVERY SYSTEM; DRUG EFFICACY; DRUG SAFETY; DRUG STABILITY; DRUG SYNTHESIS; EXPRESSION VECTOR; GENE EXPRESSION; GENE SEQUENCE; GENE SILENCING; GENE TARGETING; GENOME ANALYSIS; HEPATITIS; HUMAN; IC 50; LEUKEMIA; NONHUMAN; PLASMID; REVIEW; RNA INTERFERENCE; SOLID TUMOR;

ANIMALS; CHEMISTRY, PHARMACEUTICAL; GENE SILENCING; HUMANS; OLIGODEOXYRIBONUCLEOTIDES, ANTISENSE; RNA, SMALL INTERFERING; TECHNOLOGY, PHARMACEUTICAL;

EID: 17444404949     PISSN: 13596446     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1359-6446(05)03426-4     Document Type: Review

References (45)

1. Zamecnik P.C., Stephenson M.L. Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide. Proc. Natl. Acad. Sci. U. S. A. 75:1978;280-284
2. Kurreck J. Antisense technologies. Improvement through novel chemical modifications. Eur. J. Biochem. 270:2003;1628-1644
3. Cho-Chung Y.S., et al. Antisense-protein kinase A: a single-gene-based therapeutic approach. Antisense Nucleic Acid Drug Dev. 7:1997;217-223
4. Srivastava R.K., et al. Growth arrest and induction of apoptosis in breast cancer cells by antisense depletion of protein kinase A-RI alpha subunit: p53-independent mechanism of action. Mol. Cell. Biochem. 195:1999;25-36
5. Wang H., et al. Antitumor activity and pharmacokinetics of a mixed-backbone antisense oligonucleotide targeted to the RIalpha subunit of protein kinase A after oral administration. Proc. Natl. Acad. Sci. U. S. A. 96:1999;13989-13994
6. Summerton J. Morpholino antisense oligomers: the case for an RNase H-independent structural type. Biochim. Biophys. Acta. 1489:1999;141-158
7. Elbashir S.M., et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature. 411:2001;494-498
8. Fire A., et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature. 391:1998;806-811
9. Hamilton A.J., Baulcombe D.C. A species of small antisense RNA in posttranscriptional gene silencing in plants. Science. 286:1999;950-952
10. Miyagishi M., et al. Comparison of the suppressive effects of antisense oligonucleotides and siRNAs directed against the same targets in mammalian cells. Antisense Nucleic Acid Drug Dev. 13:2003;1-7
11. Martinez J., et al. Single-stranded antisense siRNAs guide target RNA cleavage in RNAi. Cell. 110:2002;563-574
12. Stevenson M. N. Engl. J. Med. 351:2004;1772-1777
13. Wagner R.W. Gene inhibition using antisense oligodeoxynucleotides. Nature. 372:1994;333-335
14. Crooke S.T. Progress in antisense technology: the end of the beginning. Methods Enzymol. 313:2000;3-45
15. Hogrefe R.I. An antisense oligonucleotide primer. Antisense Nucleic Acid Drug Dev. 9:1999;351-357
16. Levin A.A. A review of the issues in the pharmacokinetics and toxicology of phosphorothioate antisense oligonucleotides. Biochim. Biophys. Acta. 1489:1999;69-84
17. Agrawal S., Zhao Q. Mixed backbone oligonucleotides: improvement in oligonucleotide-induced toxicity in vivo. Antisense Nucleic Acid Drug Dev. 8:1998;135-139
18. Shen L.X., et al. Impact of mixed-backbone oligonucleotides on target binding affinity and target cleaving specificity and selectivity by Escherichia coli RNase H. Bioorg. Med. Chem. 6:1998;1695-1705
19. Goel S., et al. A safety study of a mixed-backbone oligonucleotide (GEM231) targeting the type I regulatory subunit alpha of protein kinase A using a continuous infusion schedule in patients with refractory solid tumors. Clin. Cancer Res. 9:2003;4069-4076
20. Nielsen P.E., Egholm M. An introduction to peptide nucleic acid. Curr. Issues Mol. Biol. 1:1999;89-104
21. Braasch D.A., Corey D.R. Novel antisense and peptide nucleic acid strategies for controlling gene expression. Biochemistry. 41:2002;4503-4510
22. Braasch D.A., Corey D.R. Locked nucleic acid (LNA): fine-tuning the recognition of DNA and RNA. Chem. Biol. 8:2001;1-7
23. Wahlestedt C., et al. Potent and nontoxic antisense oligonucleotides containing locked nucleic acids. Proc. Natl. Acad. Sci. U. S. A. 97:2000;5633-5638
24. Fluiter K., et al. In vivo tumor growth inhibition and biodistribution studies of locked nucleic acid (LNA) antisense oligonucleotides. Nucleic Acids Res. 31:2003;953-962
25. Hammond S.M., et al. Argonaute2, a link between genetic and biochemical analyses of RNAi. Science. 293:2001;1146-1150
26. Hannon G.J. RNA interference. Nature. 418:2002;244-251
27. Zamore P.D., et al. RNAi: double-stranded RNA directs the ATP-dependent cleavage of mRNA at 21 to 23 nucleotide intervals. Cell. 101:2000;25-33
28. Rahman M.H., et al. Selective removal of ribonucleases from solution with covalently anchored macromolecular inhibitor. Anal. Chem. 68:1996;134-138
29. Chen X., et al. Poly-2'-DNP-RNAs with enhanced efficacy for inhibiting cancer cell growth. Oligonuclotides. 14:2004;90-99
30. Ru K., et al. Growth inhibition and antimetastatic effect of antisense poly-DNP-RNA on human breast cancer cells. Oncol. Res. 11:1999;505-512
31. Shen L., et al. A high-efficacy antisense RIalpha poly-DNP 21-nt RNA. Antisense Nucleic Acid Drug Dev. 13:2003;67-74
32. Ru K., et al. Specific inhibition of breast cancer cells by antisense poly-DNP-oligoribonucleotides and targeted apoptosis. Oncol. Res. 10:1998;389-397
33. Wang A., Wang J.H. Effective treatment of murine leukemia with antisense poly-2'O-(2,4-dinitrophenyl)-oligoribonucleotides. Antisense Nucleic Acid Drug Dev. 9:1999;43-51
34. Xin W., Wang J.H. Treatment of duck hepatitis B virus by antisense poly-2'-O-(2,4-dinitrophenyl)-oligoribonucleotides. Antisense Nucleic Acid Drug Dev. 8:1998;459-468
35. Shen, L. et al. Silencing of human c-myc oncogene expression by poly-DNP-RNA. Oligonucleotides (in press)
36. Ashun M.A., et al. Inhibition of murine leukemia virus with poly-2'-O-(2,4-dinitrophenyl)poly. Antimicrob. Agents Chemother. 40:1996;2311-2317
37. Bradbury A.W., et al. Protein kinase A (PK-A) regulatory subunit expression in colorectal cancer and related mucosa. Br. J. Cancer. 69:1994;738-742
38. Srivastava R.K., et al. Antisense depletion of RIalpha subunit of protein kinase A induces apoptosis and growth arrest in human breast cancer cells. Breast Cancer Res. Treat. 49:1998;97-107
39. Offensperger W.B., et al. In vivo inhibition of duck hepatitis B virus replication and gene expression by phosphorothioate modified antisense oligodeoxynucleotides. EMBO J. 12:1993;1257-1262
40. Czauderna F., et al. Structural variations and stabilising modifications of synthetic siRNAs in mammalian cells. Nucleic Acids Res. 31:2003;2705-2716
41. Amarzguioui M., et al. Tolerance for mutations and chemical modifications in a siRNA. Nucleic Acids Res. 31:2003;589-595
42. Harborth J., et al. Sequence, chemical, and structural variation of small interfering RNAs and short hairpin RNAs and the effect on mammalian gene silencing. Antisense Nucleic Acid Drug Dev. 13:2003;83-105
43. Holen T., et al. Positional effects of short interfering RNAs targeting the human coagulation trigger Tissue Factor. Nucleic Acids Res. 30:2002;1757-1766
44. Pusch O., et al. Nucleotide sequence homology requirements of HIV-1-specific short hairpin RNA. Nucleic Acids Res. 31:2003;6444-6449
45. Holen T., et al. Similar behaviour of single-strand and double-strand siRNAs suggests they act through a common RNAi pathway. Nucleic Acids Res. 31:2003;2401-2407