2′-O-aminopropyl ribonucleotides: A zwitterionic modification that enhances the exonuclease resistance and biological activity of antisense oligonucleotides

Journal of Medicinal Chemistry

Volumn 39, Issue 26, 1996, Pages 5100-5109

  Griffey, Richard H ^a   Monia, Brett P ^a   Cummins, Lendall L ^a   Freier, Susan ^a   Greig, Michael J ^a   Guinosso, Charles J ^a   Lesnik, Elena ^a   Manalili, Sherilynn M ^a   Mohan, Venkatraman ^a   Owens, Steven ^a   Ross, Bruce R ^a   Sasmor, Henri ^a   Wancewicz, Ed ^a   Weiler, Kurt ^a   Wheeler, Patrick D ^a   Cook, P Dan ^a  

^a ISIS PHARMACEUTICALS   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

AMPHOLYTE; ANTISENSE OLIGODEOXYNUCLEOTIDE; EXONUCLEASE; RIBONUCLEOTIDE;

ARTICLE; ELECTROPORATION; ELECTROSTATIC PRECIPITATION; ENZYME DEGRADATION; ENZYME MODIFICATION; GENE TARGETING; GENETIC TRANSFECTION; HUMAN; HUMAN CELL; MOLECULAR DYNAMICS; MOLECULAR INTERACTION; NUCLEOTIDE SEQUENCE; RNA SYNTHESIS;

EXONUCLEASES; MAGNETIC RESONANCE SPECTROSCOPY; MODELS, MOLECULAR; NUCLEIC ACID HYBRIDIZATION; OLIGONUCLEOTIDES, ANTISENSE; PROTEIN-SERINE-THREONINE KINASES; PROTO-ONCOGENE PROTEINS; PROTO-ONCOGENE PROTEINS C-RAF; RIBONUCLEOTIDES;

EID: 0030463954     PISSN: 00222623     EISSN: None     Source Type: Journal    
DOI: 10.1021/jm950937o     Document Type: Article

References (30)

1. Crooke, S. T. Oligonucleotide therapeutics. In Burger's Medicinal Chemistry and Drug Discovery, Vol. 1: Principles and Practice; Wolff, M. E., Ed.; John Wiley & Sons, Inc.: New York, 1995; pp 863-900.
2. Crooke, S. T. Oligonucleotide therapy. Curr. Opin. Biotechnol. 1992, 3, 656-661.
3. Uhlmann, E.; Peyman, A. Antisense oligonucleotides: a new therapeutic principle. Chem. Rev. 1990, 90, 544-579.
4. Sanghvi, Y. S.; Cook, P. D. Towards second-generation synthetic backbones for antisense oligonucleosides. In Nucleosides and Nucleotides as Antitumor and Antiviral Agents; Chu, C.K., Baker, D., Eds.; Plenum Press: New York, 1993; pp 311-324.
5. DeMesmaeker, A.; Waldner, A.; Lebreton, J.; Hoffmann, P.; Fritsch, V.; Wolf, R. M.; Freier, S. M. Amides as a new type of backbone modification in oligonucleotides. Angew. Chem., Int. Ed. Engl. 1994, 33, 226-229.
6. Nielsen, P. E.; Eghom, M.; Berg, R. H.; Buchardt, O. Peptide Nucleic Acids (PNA). Potential anti-sense and anti-gene agents. Anticancer Drug Des. 1993, 8(1), 53-63.
7. Browne, K. A.; Dempcy, R. O.; Bruice, T. C. Binding studies of cation thymidyl deoxyribonucleic guanidine to RNA homopolynucleotides. Proc. Natl. Acad. Sci. U.S.A. 1995, 92, 7051-7055.
8. Egholm, M.; Buchardt, O.; Christensen, L.; Behrens, C.; Freier, S. M.; Driver, D. A.; Berg, R. H.; Kim, S. K.; Norden, B.; Nielsen, P. E. PNA hybridizes to complementary oligonucleotides obeying the Watson-Crick hydrogen bonding rules. Nature 1993, 365, 566-568.
9. Sanghvi, Y. S.; Cook, P. D. Carbohydrates: Synthetic methods and applications in antisense therapeutics: An overview. In Carbohydrate Modifications in Antisense Research; Sanghvi, Y. S., Cook, P. D., Eds.; ACS Symposium Series No. 580; American Chemical Society: Washington, DC, 1994; pp 1-22.
10. Lesnik, E. A.; Guinosso, C. J.; Kawasaki, A. M.; Sasmor, H.; Zounes, M.; Cummins, L. L.; Ecker, D. J.; Cook, P. D.; Freier, S. M. Oligodeoxynucleotides containing 2′-O-modified adenosine: Synthesis and effects on stability of DNA:RNA duplexes. Biochemistry 1993, 32 (30), 7832-7838. Sanghvi, Y. S.; Bellon, L.; Morvan, F.; Hoshiko, T.; Swayze, E.; Cummins, L.; Freier, S.; Dean, N.; Monia, B.; Griffey, R. H.; Cook, P. D. Synthesis, biophysical and biological evaluations of novel antisense oligonucleosides containing dephosphono-internucleosidic linkages. Nucleosides Nucleotides 1995, 14, 1087-1090.
11. Lesnik, E. A.; Guinosso, C. J.; Kawasaki, A. M.; Sasmor, H.; Zounes, M.; Cummins, L. L.; Ecker, D. J.; Cook, P. D.; Freier, S. M. Oligodeoxynucleotides containing 2′-O-modified adenosine: Synthesis and effects on stability of DNA:RNA duplexes. Biochemistry 1993, 32 (30), 7832-7838. Sanghvi, Y. S.; Bellon, L.; Morvan, F.; Hoshiko, T.; Swayze, E.; Cummins, L.; Freier, S.; Dean, N.; Monia, B.; Griffey, R. H.; Cook, P. D. Synthesis, biophysical and biological evaluations of novel antisense oligonucleosides containing dephosphono-internucleosidic linkages. Nucleosides Nucleotides 1995, 14, 1087-1090.
12. 4-(3-acetamidopropyl)cytidine. J. Org. Chem. 1993, 58, 5048-5049.
13. 4-(3-acetamidopropyl)cytidine. J. Org. Chem. 1993, 58, 5048-5049.
14. Hashimoto, H.; Nelson, M. G.; Switzer, C. Zitterionic DNA. J. Am. Chem. Soc. 1993, 115, 7128-7134.
15. Wagner, D.; Verheyden, J. P. H.; Moffat, J. G. Preparation and synthetic utility of some organotin derivatives of nucleosides. J. Org. Chem. 1974, 39, 24-30.
16. McGee, D. P.; Cook, P. D.; Guinosso, C. J. Novel 2′-O-alkyl-nucleosides and phosphroamidites: processes for the preparation and uses thereof. PCT WO 94/02501, 1994.
17. Ti, G. S.; Gaffney, B. L.; Jones, R. A. Transient protection: efficient one-flask syntheses of protected deoxynucleosides. J. Am. Chem. Soc. 1982, 104, 1316-1319.
18. Wolfe, S.; Hasan, S. K. Five-membered rings. II. Inter and intramolecular reactions of simple amines with N-substituted phthalimides. Methylamine as a reagent for removal of a phthaloyl group from nitrogen. Can. J. Chem. 1970, 48, 3572-3579.
19. Reddy, M. P.; Hanna, N. B.; Farooqui, F. Fast cleavage and deprotection of oligonucleotides. Tetrahedron Lett. 1994, 35, 4311-4314.
20. Mohan, V.; Griffey, R. H.; Davis, D. R. Structure and dynmaics of MMI linked nucleotides. Tetrahedron 1995, 51, 6855-6868.
21. De Leeuw, F. A. A. M.; Altona, C. Computer-assisted pseudorotation analysis of five-membered rings by means of proton spin-spin coupling constants: program PSEUDOROT. J. Comput. Chem. 1983, 4, 428-437.
22. Cummins, L. L.; Owens, S. R.; Risen, L. M.; Lesnik, E. A.; Freier, S. M.; McGee, D.; Guinosso, C. J.; Cook, P. D. Characterization of fully 2′-modified oligoribonucleotide hetero- and homoduplex hybridization and nuclease sensitivity. Nucleic Acid Res. 1995, 23, 2019-2024.
23. Kiely, J. S. Recent advances in antisense technology. Annu. Rep. Med. Chem. 1994, 29, 297-306.
24. Crooke, S. T.; Leminidis, K. M.; Neilson, L.; Griffey, R. H.; Lesnik, E. A.; Monia, B. P. Kinetic characteristics of Escherichia coli Rnase H1: cleavage of various antisense oligonucleotide-RNA duplexes. Biochem. J. 1995, 312, in press.
25. C. Guinosso, private communication.
26. Nogle, J. H.; Schirmer, R. E. The Nuclear Overhauser Effect; Academic Press: New York, 1971.
27. Iyer, P. R.; Phillips, L. R.; Egan, W.; Regan, J. B.; Beaucage, S. The automated synthesis of sulfur-containing oligodeoxyribonucleotides using 3H-1,2-benzodithiol-3-one 1,1-dioxide as a sulfur-transfer reagent. J. Org. Chem. 1990, 55, 4693-4698.
28. Greig, M.; Griffey, R. H. Utility of organic bases for improved electrospray mass spectrometry of oligonucleotides. Rapid Commun. Mass Spectrom. 1995, 5, 97-102.
29. Monia, B. P.; Johnston, J. F.; Geiger, T.; Muller, M.; Fabbro, D. Antitumor activity of a phsophorothioate antisense oligonucleotide targeted against C-raf kinase. Nature Med. 1996, 2, 668-675.
30. Ausubel, F. M., Brent, R., Kingston, R. E., Moore, D. D., Seidman, J. G., Smith, J. A., Struhl, K., Eds. Introduction of DNA into mammalian cells. In Current protocols in molecular biology; Green Publishing Associates and Wiley Interscience: New York, 1987.