Intracellular mRNA cleavage induced through activation of RNase P by nuclease-resistant external guide sequences

Nature Biotechnology

Volumn 18, Issue 1, 2000, Pages 58-61

  Ma, Michael ^a   Benimetskaya, Lyuba ^b   Lebedeva, Irina ^b   Dignam, Jeanne ^a   Takle, Garry ^a   Stein, C A ^b  

^a VimRx Pharmaceuticals   (United States)

^b Och Spine at New York Presbyterian Hospitals   (United States)

Author keywords

External guide sequences; RNase P

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; MESSENGER RNA; NUCLEASE; OLIGORIBONUCLEOTIDE; PROTEIN BCL X; PROTEIN KINASE C; RIBONUCLEASE H; RIBONUCLEASE P; RIBOZYME; TRANSFER RNA;

ARTICLE; BLADDER CARCINOMA; CONTROLLED STUDY; DOWN REGULATION; GENE EXPRESSION REGULATION; GENE SEQUENCE; HUMAN; HUMAN CELL; PRIORITY JOURNAL; PROTEIN EXPRESSION; RNA CLEAVAGE;

3' UNTRANSLATED REGIONS; BCL-X PROTEIN; BLOTTING, WESTERN; DOWN-REGULATION; ENDORIBONUCLEASES; ENZYME ACTIVATION; HUMANS; ISOENZYMES; NUCLEIC ACID CONFORMATION; OLIGORIBONUCLEOTIDES; PHOSPHATIDYLETHANOLAMINES; PROTEIN KINASE C; PROTEIN KINASE C-ALPHA; PROTO-ONCOGENE PROTEINS C-BCL-2; RIBONUCLEASE H, CALF THYMUS; RIBONUCLEASE P; RNA PROCESSING, POST-TRANSCRIPTIONAL; RNA, ANTISENSE; RNA, CATALYTIC; RNA, GUIDE; RNA, MESSENGER; RNA, TRANSFER; SUBSTRATE SPECIFICITY; TRANSFECTION; TUMOR CELLS, CULTURED; URINARY BLADDER NEOPLASMS;

EID: 0033986726     PISSN: 10870156     EISSN: None     Source Type: Journal    
DOI: 10.1038/71924     Document Type: Article

References (26)

1. Stein, C.A. & Cheng, Y.-C. Antisense oligonucleotides as therapeutic agents - Is the bullet really magic? Science 261, 1004-1002 (1993).
2. Wagner, R. Gene inhibition using antisense oligodeoxynucleotides. Nature 372, 333-335 (1994).
3. Stein, C.A. & Krieg, A. eds. Applied antisense oligonucleotide technology. (Wiley-Liss, New York; 1998).
4. Mulamba, G., Hu, A., Azad, R., Anderson, K. & Coen, I. Human cytomegalovirus mutant with sequence-dependent resistance to the phosphorothioate oligonucleotide fomivirsen (Isis 2922). Antimicrob. Agents Chemother. 42, 971-973 (1998).
5. Walder, R.Y. & Walder, J.A. Role of RNase H in hybrid-arrested translation by antisense oligonucleotides. Proc Natl. Acad. Sci. USA 85, 5011-5015 (1988).
6. Minshull, J. & Hunt, T. The use of single-stranded DNA and RNase-H to promote quantitative "hybrid arrest of translation" of mRNA-DNA hybrids in reticulocyte lysate cell-free translations. Nucleic Acids Res. 14, 6433-6451 (1986).
7. Hausen, P & Stein, H. Ribonuclease H. An enzyme degrading the RNA moiety of DNA-RNA hybrids. Eur. J. Biochem. 14, 278-283 (1970).
8. Stein, C.A., Subasinghe, C., Shinozuka, K. & Cohen, J. Physicochemical properties of phosphorothioate oligodeoxyribonucleotides. Nucleic Acids Res. 16, 3209-3221 (1988).
9. Giles, R. & Tidd, D. Increased specificity for antisense oligodeoxynucleotide targeting of RNA cleavage by RNase H using chimeric methylphosphonodiester/phosphodiester structures. Nucleic Acids Res. 20, 763-770 (1992).
10. Giles, R., Spiller, D. & Tidd, D. Chimeric oligodeoxynucleotide analogues: enhanced cell uptake of structures that direct ribonuclease H with high specificity. Anti-Cancer Drug Res. 8, 33-51 (1993).
11. Giles, R., Ruddell, C., Spiller, D., Green, J. & Tidd, D. Single base discrimination for ribonuclease H dependent antisense effects within intact human leukaemia cells. Nucleic Acids Res. 23, 954-961 (1995).
12. Monia, B. et al. Evaluation of 2'-modified oligonucleotides containing 2'-deoxy gaps as antisense inhibitors of gene expression. J. Biol. Chem. 268, 14154-14522 (1993).
13. Benimetskaya, L. et al. Cationic porphyrins: novel delivery vehicles for antisense oligonucleotides. Nucleic Acids Res. 26, 5310-5317 (1998).
14. Forster, A. & Altman, S. External guide sequences for an RNA enzyme. Science 249, 783-786 (1990).
15. Altman, S. RNA enyme-directed gene therapy. Proc. Natl. Acad. Sci. USA 90, 10898-10900 (1993).
16. Ma, M. et al. Nuclease-resistant external guide sequence-induced cleavage of target RNA by human ribonuclease P. Antisense Nucleic Acid Drug Devel. 8, 415-426 (1998).
17. Heidenreich, O., Benseler, F., Fahrenholz, A. & Eckstein, F. High activity and stability of hammerhead ribozymes containing 2'-modified pyrimidine nucleosides and phosphorothioates. J. Biol. Chem. 269, 2131-2138 (1994).
18. Yuan, Y., Hwang, E. & Altman, S. Targeted cleavage of mRNA by human RNase P. Proc. Natl. Acad. Sci. USA 89, 8006-8010 (1992).
19. Dean, N., McKay, R., Condon, T., & Bennett, F. Inhibition of protein kinase C-α expression in human A549 cells by antisense oligonucleotides inhibits induction of intercellular adhesion molecule 1 (ICAM-1) mRNA by phorbol esters. J. Biol. Chem. 269, 16416-16424 (1994).
20. Dean, N. & McKay, R. Inhition of protein kinase C-α expression in mice after systemic administration of phosphorothioate antisense oligodeoxynucleotides. Proc. Natl. Acad. Sci. USA 91, 11762-11766 (1994).
21. Loria, A. & Pan, T. Recognition of the T stem-loop of a pre-tRNA substrate by the ribozyme from Bacillus subtilis ribonuclease P. Biochemistry 36, 6317-6325 (1997).
22. Kahle, D., Wehmeyer, U. & Krupp, G. Substrate recognition by RNase P and by the catalytic M1 RNA: identification of possible contact points in pre-tRNAs. EMBO J. 9, 1929-1937 (1990).
23. Werner, M., Rose, E., Al Emran, O., Goldberg, A. & George, S. Targeted cleavage of RNA molecules by human RNase P using minimized external guide sequences. Antisense Nucleic Acid Drug Devel. 9, 81-98 (1999).
24. Werner, M., Rosa, E., Nordstrom, J., Goldberg, A. & George, S. Short oligonucleotides as external guide sequences for site-specific cleavage of RNA molecules with human RNase P. RNA 4, 847-855 (1998).
25. Ortigao, J. et al. Antisense effect of oligodeoxynucleotides with inverted terminal internucleotidic linkages: a minimal modification protecting against nucleolytic degradation. Antisense Res. Devel. 2, 129-146 (1992).
26. Sinha, N. et al. Labile exocyclic amine protection of nucleosides in DNA, RNA and oligonucleotide analog synthesis facilitating N-deacylation, minimizing depurination and chain degradation. Biochimie 75, 13-23 (1993).