Advertisement

Antisense Oligonucleotides as Modulators of Pre-mRNA Splicing

  • Halina Sierakowska
  • Sudhir Agrawal
  • Ryszard Kole
Part of the Methods in Molecular Biology™ book series (MIMB, volume 133)

Abstract

Antisense oligonucleotides have been extensively used as downregulators of gene expression (1), and as such are not only increasingly used in various fields as sequence-specific research tools (2, 3, 4), but are also tested in clinical trials as antiviral and anticancer agents (5, 6, 7, 8).

Keywords

Splice Site Reverse Transcription Polymerase Chain Reaction Antisense Oligonucleotide Hank Balance Salt Solution Ammonium Persulfate 
These keywords were added by machine and not by the authors. This process is experimental and the keywords may be updated as the learning algorithm improves.

References

  1. 1.
    Crooke S. T. and Bennett CF. (1996) Progress in antisense oligonucleotide therapeutics. Ann. Rev. Pharm.Tox. 36, 107–129.CrossRefGoogle Scholar
  2. 2.
    Pasternak G. W. and Standifer K. M. (1995) Mapping of opioid receptors using antisense oligodeoxynucleotides: correlating their molecular biology and pharmacology. Trends Pharmacol. Sci. 16, 344–350.PubMedCrossRefGoogle Scholar
  3. 3.
    Niggli E., Schwaller B., and Lipp P. (1996) Antisense oligodeoxynucleotides directed against the Na–Ca exchanger mRNA. Promising tools for studies on the cellular and molecular level. Ann. NY Acad. Sci. 779, 93–102.PubMedCrossRefGoogle Scholar
  4. 4.
    Ramchandani S., MacLeod R. A., Pinard M., von Hoffe E., and Szyf M. (1997) Inhibition of tumorigenesis by a cytosine-DNA, methyltransferase, antisense oligodeoxynucleotide. Proc. Natl. Acad. Sci. USA 94, 684–689.PubMedCrossRefGoogle Scholar
  5. 5.
    Zhang R., Y. J., Shahinian H., Amin G., Lu Z., Liu T., Saag M.S., Temsamani Y., and Martin R. R. (1995) Pharmacokinetics of an anti-human immunodeficiency virus antisense oligodeoxynucleotide phosphorothioate (GEM 91) in HIVinfected subjects. Clin. Pharm. Ther. 58, 44–53.CrossRefGoogle Scholar
  6. 6.
    Bishop, M. R., Iversen, P. L., Bayever, E., Shar, J. G., Greiner, T. C., Copple, et al. (1995) Phase I trial of an antisense oligonucleotide OL(1)p53 in hematologic malignancies. J. Clin. Oncol. 14, 1320–1326.Google Scholar
  7. 7.
    Agrawal, S (1996) Antisense oligonucleotides: towards clinical trials. Trends Biotech. 14, 376–378.CrossRefGoogle Scholar
  8. 8.
    Tonkinson, J. L. and Stein, C. A. (1996) Antisense oligonucleotides as clinical therapeutic agents. Cancer Invest. 14, 64–65.CrossRefGoogle Scholar
  9. 9.
    Dominski, Z. and Kole, R. (1993) Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides. Proc. Natl. Acad. Sci. USA 90, 8673–8677.PubMedCrossRefGoogle Scholar
  10. 10.
    Sierakowska, H., Sambade, M. J., Agrawal, S., and Kole, R. (1996) Repair of thalassemic human β-globin mRNA in mammalian cells by antisense oligonucleotides. Proc. Natl. Acad. Sci. USA. 93, 12840–12844.PubMedCrossRefGoogle Scholar
  11. 11.
    Weatherall, D. J. (1994) Thalassemias, in The Molecular Basis of Blood Diseases (Stamatoyannopoulos, G., et al., eds.), W.B. Saunders Philadelphia, pp. 157–205.Google Scholar
  12. 12.
    Mirabelli, C. K., and Crooke, S. T. (1993) Antisense oligonucleotides in the context of modern molecular drug discovery and development, in Antisense Research and Applications (Crooke, S. T. and Lebleu, B., eds.), CRC, Boca Raton, FL, pp. 7–35.Google Scholar
  13. 13.
    Furdon, P. F., Dominski, Z., and Kole, R. (1989) RNase H cleavage of RNA hybridized to oligonucleotides containing methylphosphonate, phosphorothioate and phosphodiester bonds. Nucleic Acids Res. 17, 9193–9204.PubMedCrossRefGoogle Scholar
  14. 14.
    Sproat, B. S. and Lamond, A. I. (1993) 2′-O-alkyloligoribonucleotides, in Antisense Research and Applications (Crooke, S. T. and Lebleu, B., eds.), CRC, Boca Raton, FL, pp. 351–3Google Scholar
  15. 15.
    Zhang, R., Lu, Z., Zhao, H., Zhang, X., Diasio, R. B., Habus, I., et al. (1995) In vivo stability, disposition and metabolism of a “hybrid” oligonucleotide phosphorothioate in rats. Biochem. Pharm. 50, 545–556.PubMedCrossRefGoogle Scholar
  16. 16.
    Hawley-Nelson, P., Ciccarone, V., Gebeyehu, G., Jessee, J., and Felgner, P. L. (1993) Lipofectamine reagent: a new, higher efficiency polycationic liposome transfection reagent. Focus 15, 73–79.Google Scholar
  17. 17.
    Schagger, H. and von Jagov, G. (1987) Tricine-sodium dodecyl sulfate-polyacrylamide gel electrophoresis for the separation of proteins in the range from 1 to 100 kDa. Anal. Biochem. 166, 368–379.PubMedCrossRefGoogle Scholar

Copyright information

© Humana Press Inc. 2000

Authors and Affiliations

  • Halina Sierakowska
  • Sudhir Agrawal
  • Ryszard Kole

There are no affiliations available

Personalised recommendations