- J.B. Opalinska and A.M. Gewirtz, Nucleic-acid therapeutics: basic principles and recent applications.
Nat. Rev. Drug Discovery, 2002, 1 , 503–514. - L.J. Scherer and J.J. Rossi, Approaches for the sequence-specific knockdown of mRNA. Nat.
Biotechnol., 2003, 21 , 1457–1465. - M. Faria, C.D. Wood, L. Perrouault, J.S. Nelson, A. Winter, M.R.H. White, C. Hélène and
C. Giovannangeli, Targeted inhibition of transcription elongation in cells mediated by triplex-forming
oligonucleotides. Proc. Natl. Acad. Sci. USA, 2000, 97 , 3862–3867. - P.C. Zamecnik and M.L. Stephenson, Inhibition of Rous sarcoma virus replication and transformation
by a specific oligodeoxynucleotide. Proc. Natl. Acad. Sci. USA, 1978, 75 , 280–284. - P. Sazani, M.M. Vacek and R. Kole, Short-term and long-term modulation of gene expression by anti-
sense therapeutics. Curr. Opin. Biotech., 2002, 13 , 468–472. - J. Kurreck, Antisense technologies. Improvement through novel chemical modifications. Eur. J.
Biochem., 2003, 270 , 1628–1644. - B.F. Baker, S.S. Lot, T.P. Condon, S. Cheng-Flourney, E.A. Lesnik, H.M. Sasmor and C.F. Bennett,
2 -O-(2-methoxy)ethyl-modified anti-intercellular adhesion molecule 1 (ICAM-1) oligonucleotides
selectively increase the ICAM-1 mRNA level and inhibit formation of the ICAM-1 translation initiation
complex in human umbilical vein endothelial cells. J. Biol. Chem., 1997, 272 , 11994–12000. - M. Faria, D.G. Spiller, C. Dubertret, J.S. Nelson, M.R.H. White, D. Scherman, C. Hélène and
C. Giovannangeli, Phosphoramidate oligonucleotides as potent antisense molecules in cells in vivo.
Nature Biotech., 2001, 19 , 40–44. - D.R. Mercatante and R. Kole, Control of alternative splicing by antisense oligonucleotides as a poten-
tial chemotherapy: effects on gene expression. Biochim. Biophys. Acta, 2002, 1587 , 126–132. - T.A. Vickers, J.R. Wyatt, T. Burckin, C.F. Bennett and S.M. Freier, Fully modified 2-MOE
oligonucleotides redirect polyadenylation. Nucl. Acids Res., 2001, 29 , 1293–1299. - J.J. Toulmé, C. Boiziau, B. Larrouy, P. Frank, S. Albert and R. Ahmadi, in DNA and RNA Cleavers
and Chemotherapy of Cancer and Viral Diseases, B. Meunier (ed). Kluwer Academic Publishers, The
Netherlands, 1996, 271–288. - R.A. McKay, L.J. Miraglia, L.L. Cummins, S.R. Owens, H. Sasmor and N.M. Dean, Characterization
of a potent and specific class of antisense oligonucleotide inhibitor of human protein kinase C-
expression. J. Biol. Chem., 1999, 274 , 1715–1722. - A. Asai, Y. Oshima, Y. Yamamoto, T. Uochi, H. Kusaka, S. Akinaga, Y. Yamashita, K. Pongracz,
R. Pruzan, E. Wunderet al., A novel telomerase template antagonist (GRN163) as a potential
anticancer agent. Cancer Res., 2003, 63 , 3931–3939. - S. Agrawal and E.R. Kandimella, Medicinal chemistry and therapeutic potential of CpG DNA. Trends
Mol. Med., 2002, 8 , 114–121. - S. Agrawal and E.R. Kandimella, Antisense and siRNA as agonists of Toll-like receptors. Nat.
Biotech., 2004, 22 , 1533–1537. - E.S. Gragoudas, A.P. Adamis, E.T. Cunningham, M. Feinsod and D.R. Guyer, Pegaptanib for
neovascular age-related macular degeneration. New Engl. J. Med., 2004, 351 , 2805–2816. - A.J. Hamilton and D.C. Baulcombe, A species of small antisense RNA in post-transcriptional gene
silencing in plants. Science, 1999, 286 , 950–952. - A. Fire, S. Xu, M.K. Montgomery, S.A. Kostas, S.E. Driver and C.C. Mello, Potent and specific
genetic interference by double-stranded RNA in Caenorhabditis elegans. Nature, 1998, 391 , 806–811. - G. Hannon and J.J. Rossi, Unlocking the potential of the human genome with RNA interference.
Nature, 2004, 431 , 371–378. - O.A. Kent and A.M. MacMillan, RNAi: running interference for the cell. Org. Biomol. Chem.,
2004, 2 , 1957–1961. - S.W. Jones, D.S. P.M. and M.A. Lindsay, siRNA for gene silencing: a route to drug target discovery.
Curr. Opin. Pharmacol., 2004, 4 , 522–527. - M. Tijsterman and R.H.A. Plasterk, Dicers at RISC: the mechanism, of RNAi. Cell, 2004, 117 , 1–4.
206 Chapter 5