Applications of antisense and siRNAs during preclinical drug development

Drug Discovery Today

Volumn 7, Issue 17, 2002, Pages 912-917

  Thompson, James D ^a,b  

^a Impact Biosciences   (United States)

^b Sirna Therapeutics Inc   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ACETYLSALICYLIC ACID; ANTISENSE OLIGONUCLEOTIDE; IBUPROFEN; NONSTEROID ANTIINFLAMMATORY AGENT; RNA;

BIOTECHNOLOGY; DRUG MECHANISM; DRUG RESEARCH; DRUG SENSITIVITY; DRUG SPECIFICITY; DRUG TARGETING; NUCLEOTIDE SEQUENCE; REVIEW;

DRUG EVALUATION, PRECLINICAL; HUMANS; OLIGONUCLEOTIDE ARRAY SEQUENCE ANALYSIS; RNA, ANTISENSE; RNA, SMALL INTERFERING; TECHNOLOGY, PHARMACEUTICAL;

EID: 0036742715     PISSN: 13596446     EISSN: None     Source Type: Journal    
DOI: 10.1016/S1359-6446(02)02410-8     Document Type: Review

References (49)

1. Drews J. Drug Industry: a historical perspective. Science. 287:2000;1960-1964.
2. 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.
3. Stein C.A. Does antisense exist? Nat. Med. 1:1995;1119-1121.
4. Wagner R.W. The state of the art in antisense research. Nat. Med. 1:1995;1116-1118.
5. Hogrefe R.I. An antisense oligonucleotide primer. Antisense Nucleic Acid Drug Dev. 9:1999;351-357.
6. Bennett C.F., Cowsert L.M. Application of antisense oligonucleotides for gene functionalization and target validation. Curr. Opin. Mol. Ther. 1:1999;359-371.
7. Bennett C.F. Antisense oligonucleotides: is the glass half full or half empty? Biochem. Pharmacol. 55:1998;9-19.
8. Baker B.F., et al. Discovery and analysis of antisense oligonucleotide activity in cell culture. Methods. 23:2001;191-198.
9. Agrawal S. Importance of nucleotide sequence and chemical modifications of antisense oligonucleotides. Biochim. Biophys. Acta. 1489:1999;53-68.
10. Adah S.A., et al. Chemistry and biochemistry of 2′,5′-oligoadenylate-based antisense strategy. Curr. Med. Chem. 8:2001;1189-1212.
11. Nielsen P.E., et al. Sequence-selective recognition of DNA by strand displacement with a thymine-substituted polyamide. Cancer Res. 15:1991;5468-5473.
12. Iversen P.L. Phosphorodiamidate morpholino oligomers: favorable properties for sequence-specific gene inactivation. Curr. Opin. Mol. Ther. 3:2001;235-238.
13. Orum H., Wengel J. Locked nucleic acids: a promising molecular family for gene-function analysis and antisense drug development. Curr. Opin. Mol. Ther. 3:2001;239-243.
14. Fire A., et al. Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature. 391:1998;806-811.
15. Sharp P.A. RNA interference 2001. Genes Dev. 15:2001;485-490.
16. Plasterk R.H.A. RNA silencing: the genome's immune system. Science. 296:2002;1263-1265.
17. Zamore P.D. Ancient pathways programmed by small RNAs. Science. 296:2002;1265-1269.
18. Hamilton A.J., Baulcombe D.C. A species of small antisense RNA in posttranscriptional gene silencing in plants. Science. 286:1999;950-952.
19. Elbashir S.M. Functional anatomy of siRNAs for mediating efficient RNAi in Drosophila melanogaster embryo lysate. EMBO J. 20:2001;6877-6888.
20. Kumar M., et al. Antisense RNA: function and fate of duplex RNA in cells of higher eukaryotes. Microbiol. Mol. Biol. Rev. 62:1998;1415-1434.
21. Elbashir S.M., et al. Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells. Nature. 411:2001;494-498.
22. Elbashir S.M., et al. Analysis of gene function in somatic mammalian cells using small interfering RNAs. Methods. 26:2002;199-213.
23. Hughes M.D., et al. The cellular delivery of antisense oligonucleotides and ribozymes. Drug Discov. Today. 6:2001;303-315.
24. Lebedeva I., et al. Cellular delivery of antisense oligonucleotides. Eur. J. Pharm. Biopharm. 50:2000;101-119.
25. Fraser G.L., et al. Antisense inhibition of delta-opioid receptor gene function in vivo by peptide nucleic acids. Mol. Pharmacol. 57:2000;725-731.
26. Henry S.P., et al. Toxicological and pharmacokinetic properties of chemically modified antisense oligonucleotide inhibitors of PKC-alpha and C-raf kinase. Anticancer Drug Des. 12:1997;409-420.
27. Stein C.A. The experimental use of antisense oligonucleotides: a guide for the perplexed. J. Clin. Invest. 108:2001;641-644.
28. Fisher T.L., et al. Intracellular disposition and metabolism of fluorescently-labeled unmodified and modified oligonucleotides microinjected into mammalian cells. Nucleic Acids Res. 21:1993;3857-3865.
29. Sierakowska H., et al. Antisense oligonucleotides and RNAs as modulators of pre-mRNA splicing. Methods Enzymol. 313:2000;506-521.
30. Kole R., Sazani P. Antisense effects in the cell nucleus: modification of splicing. Curr. Opin. Mol. Ther. 3:2001;229-234.
31. Billy E., et al. Specific interference with gene expression by long, double-stranded RNA in mouse embryonal teratocarcinoma cell lines. Proc. Natl. Acad. Sci. U. S. A. 98:2001;14428-14433.
32. Venter J.C., et al. The sequence of the human genome. Science. 291:2001;1304-1351.
33. Lander E.S., et al. Initial sequencing and analysis of the human genome. Nature. 409:2001;860-921.
34. Pfeffer U., et al. Alternative splicing of the estrogen receptor primary transcript normally occurs in estrogen receptor positive tissues and cell lines. J. Steroid Biochem. Mol. Biol. 56:1996;99-105.
35. Miyagishi M., Taira K. U6 promoter-driven siRNAs with four uridine 3′ overhangs efficiently suppress targeted gene expression in mammalian cells. Nat. Biotechnol. 20:2002;497-500.
36. Lee N.S., et al. Expression of small interfering RNAs targeted against HIV-1 rev transcripts in human cells. Nat. Biotechnol. 20:2002;500-505.
37. Paul C.P., et al. Effective expression of small interfering RNA in human cells. Nat. Biotechnol. 20:2002;505-508.
38. Simon L.S. COX-2 inhibitors. Are there nonsteroidal anti-inflammatory drugs with a better safety profile? Gastroenterol. Clin. North Am. 30:2001;1011-1025.
39. Giles R.V., et al. Single base discrimination for ribonuclease H-dependent antisense effects within intact human leukaemia cells. Nucleic Acids Res. 23:1995;954-961.
40. Yamada R., et al. Selective inhibition of cyclooxygenase-2 with antisense oligodeoxynucleotide restricts induction of rat adjuvant-induced arthritis. Biochem. Biophys. Res. Commun. 269:2000;415-421.
41. Issaq H.J. The SELDI-TOF MS approach to proteomics: protein profiling and biomarker identification. Biochem. Biophys. Res. Commun. 292:2002;587-592.
42. van Veer L.J., et al. Gene expression profiling predicts clinical outcome of breast cancer. Nature. 415:2002;530-536.
43. Hughes T.R., et al. Functional discovery via a compendium of expression profiles. Cell. 7:2000;109-126.
44. Marton M.J., et al. Drug target validation and identification of secondary drug target effects using DNA microarrays. Nat. Med. 4:1998;1293-1301.
45. Brown P.O., Botstein D. Exploring the new world of the genome with DNA microarrays. Nat. Genet. 21:1999;33-37.
46. Brody E.N., Gold L. Aptamers as therapeutic and diagnostic agents. J. Biotechnol. 74:2000;5-13.
47. Myers E.W., Miller W. Optimal alignments in linear space. Comput. Appl. Biosci. 4:1988;11-17.
48. Pearson W.R., Lipman D.J. Improved tools for biological sequence comparison. Proc. Natl. Acad. Sci. U. S. A. 85:1988;2444-2448.
49. Pearson W.R. Rapid and sensitive sequence comparison with FASTP and FASTA. Methods Enzymol. 183:1990;63-98.