Modification of Alternative Splicing by Antisense Therapeutics

Oligonucleotides

Volumn 14, Issue 1, 2004, Pages 65-74

  Kole, Ryszard ^a,b   Vacek, Marla ^a   Williams, Tiffany ^a  

^a UNIVERSITY OF NORTH CAROLINA   (United States)

^b UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; MESSENGER RNA; MESSENGER RNA PRECURSOR; OLIGONUCLEOTIDE; PROTEIN;

ALTERNATIVE RNA SPLICING; CANCER; CHEMICAL MODIFICATION; DEGENERATIVE DISEASE; DOWN REGULATION; GENETIC TRANSCRIPTION; IN VIVO STUDY; PRIORITY JOURNAL; REVIEW; THALASSEMIA;

ALTERNATIVE SPLICING; RNA PRECURSORS; RNA, MESSENGER;

EID: 1942453441     PISSN: 15454576     EISSN: None     Source Type: Journal    
DOI: 10.1089/154545704322988067     Document Type: Review

References (61)

1. ASTRIAB-FISHER, A., SERGUEEV, D., FISHER, M., SHAW, B.R., and JULIANO, R.L. (2002). Conjugates of antisense oligonucleotides with the Tat and antennapedia cell-penetrating peptides: Effects on cellular uptake, binding to target sequences, and biologic actions. Pharm. Res. 19, 744-754.
2. BLACK, D.L. (2003). Mechanisms of alternative pre-messenger RNA splicing. Annu. Rev. Biochem. 72, 291-336.
3. BLENCOWE, B.J. (2000). Exonic splicing enhancers: Mechanism of action, diversity and role in human genetic diseases. Trends Biochem. Sci. 25, 106-110.
4. BLENCOWE, B.J., BOWMAN, J.A., McCRACKEN, S., and ROSONINA, E. (1999). SR-related proteins and the processing of messenger RNA precursors. Biochem. Cell Biol. 77, 277-291.
5. BOISE, L.H., GONZALEZ-GARCIA, M., POSTEMA. C.E., DING, L., LINDSTEN, T., TURKA, L.A., MAO, X., NUNEZ, G., and THOMPSON, C.B. (1993). bcl-x, a bcl-2-related gene that functions as a dominant regulator of apoptotic cell death. Cell 74, 597-608.
6. BROOME, H.E., YU, A.L., DICCIANNI, M., CAMITTA, B.M., MONIA, B.P., and DEAN, N.M. (2002). Inhibition of bcl-xL expression sensitizes T-cell acute lymphoblastic leukemia cells to chemotherapeutic drugs. Leuk. Res. 26, 311-316.
7. BUDKER, V., ZHANG, G., KNECHTLE, S., and WOLFF, J.A. (1996). Naked DNA delivered intraportally expresses efficiently in hepatocytes. Gene Ther. 3, 593-598.
8. CACERES, J.F., STAMM, S., HELFMAN, D.M., and KRAINER, A.R. (1994). Regulation of alternative splicing in vivo by overexpression of antagonistic splicing factors. Science 265, 1706-1709.
9. CARTEGNI, L., CHEW, S.L., and KRAINER, A.R. (2002). Listening to silence and understanding nonsense: Exonic mutations that affect splicing. Nat. Rev. Genet. 3, 285-298.
10. CARTEGNI, L., and KRAINER, A.R. (2003). Correction of disease-associated exon skipping by synthetic exon-specific activators. Nat. Struct. Biol. 10, 120-125.
11. CHAO, H., MANSFIELD, S.G., BARTEL, R.C., HIRIYANNA, S., MITCHELL, L.G., GARCIA-BLANCO, M.A., and WALSH, C.E. (2003). Phenotype correction of hemophilia A mice by spliceosome-mediated RNA transsplicing. Nat. Med. 9, 1015-1019.
12. CONSORTIUM, I.H.G.S. (2001). Initial sequencing and analysis of the human genome. Nature 409, 860-921.
13. CROOKE, S. (2001). Basic principles of antisense technology. In: Antisense Drug Technology: Principles, Strategies and Applications. S. Crooke, ed. (Marcel Dekker, New York), pp. 1-28.
14. DEAN, N.M, and BENNETT, C.F. (2003). Antisense oligonucleotide-based therapeutics for cancer. Oncogene 22, 9087-9096.
15. DE ANGELIS, F.G., STHANDIER, O., BERARDUCCI, B., TOSO, S., GALLUZZI, G., RICCI, E., COSSU, G., and BOZZONI, I. (2002). Chimeric snRNA molecules carrying antisense sequences against the splice junctions of exon 51 of the dystrophin pre-mRNA induce exon skipping and restoration of a dystrophin synthesis in delta 48-50 DMD cells. Proc. Natl. Acad. Sci. USA 99, 9456-9461.
16. DEININGER, M.H., WELLER, M., STREFFER, J., and MEYERMANN, R. (1999). Antiapoptotic Bcl-2 family protein expression increases with progression of oligodendroglioma. Cancer 86, 1832-1839.
17. 3 on the increased expression of bcl-xL in psoriasis. Arch. Dermatol. Res. 293, 620-625.
18. GAUTSCHI, O., TSCHOPP, S., OLIE, R.A., LEECH, S.H., SIMOES-WUST, A.P., ZIEGLER, A., BAUMANN, B., ODERMATT, B., HALL, J., STAHEL, R.A., and ZANGEMEISTER-WITTKE, U. (2001). Activity of a novel bcl-2/bcl-xL-bispecific antisense oligonucleotide against tumors of diverse histologic origins. J. Natl. Cancer Inst. 93, 463-471.
19. GEBSKI, B.L., MANN, C.J., FLETCHER, S., and WILTON, S.D. (2003). Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle. Hum. Mol. Genet. 12, 1801-1811.
20. GEMIGNANI, F., SAZANI, P., MORCOS, P., and KOLE, R. (2002). Temperature-dependent splicing of beta-globin pre-mRNA. Nucleic Acids Res. 30, 4592-4598.
21. HASTINGS, M.L., and KRAINER, A.R. (2001). Pre-mRNA splicing in the new millennium. Curr. Opin. Cell Biol. 13, 302-309.
22. HEERE-RESS, E., THALLINGER, C., LUCAS, T., SCHLAGBAUER-WADL, H., WACHECK, V., MONIA, B.P., WOLFF, K., PEHAMBERGER, H., and JANSEN, B. (2002). Bcl-X(L) is a chemoresistance factor in human melanoma cells that can be inhibited by antisense therapy. Int. J. Cancer 99, 29-34.
23. HUISMAN, T.H. (1990). Frequencies of common beta-thalassaemia alleles among different populations: Variability in clinical severity. Br. J. Haematol. 75, 454-457.
24. KALBFUSS, B., MABON, S.A., and MISTELI, T. (2001). Correction of alternative splicing of tau in frontotemporal dementia and parkinsonism linked to chromosome 17. J. Biol. Chem. 276, 42986-42993.
25. KOLE, R., and SAZANI, P. (2001). Antisense effects in the cell nucleus: Modification of splicing. Curr. Opin. Mol. Ther. 3, 229-234.
26. KRAJEWSKA, M., MOSS, S.F., KRAJEWSKI, S., SONG, K., HOLT, P.R., and REED, J.C. (1996). Elevated expression of bcl-X and reduced bak in primary colorectal adenocarcinomas. Cancer Res. 56, 2422-2427.
27. KURRECK, J. (2003). Antisense technologies. Eur. J. Biochem. 270, 1628-1644.
28. LACERRA, G., SIERAKOWSKA, H., CARESTIA, C., FUCHAROEN, S., SUMMERTON, J., WELLER, D., and KOLE, R. (2000). Restoration of hemoglobin A synthesis in erythroid cells from peripheral blood of thalassemic patients. Proc. Natl. Acad. Sci. USA 97, 9591-9596.
29. LIM, S.R., and HERTEL, K.J. (2001). Modulation of survival motor neuron pre-mRNA splicing by inhibition of alternative 3′ splice site pairing. J. Biol. Chem. 276, 45476-45483.
30. LIU, X., JIANG, Q., MANSFIELD, S.G., PUTTARAJU, M., ZHANG, Y., ZHOU, W., COHN, J.A., GARCIA-BLANCO, M.A., MITCHELL, L.G., and ENGELHARDT, J.F. (2002). Partial correction of endogenous deltaF508 CFTR in human cystic fibrosis airway epithelia by spliceosome-mediated RNA trans-splicing. Nat. Biotechnol. 20, 47-52.
31. LU, Q.L., MANN, C.J., LOU, F., BOU-GHARIOS, G., MORRIS, G.E., XUE, S.A., FLETCHER, S., PARTRIDGE, T.A., and WILTON, S.D. (2003). Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse. Nat. Med. 9, 1009-1014.
32. MANN, C.J., HONEYMAN, K., CHENG, A.J., LY, T., LLOYD, F., FLETCHER, S., MORGAN, J.E., PARTRIDGE, T.A., and WILTON, S.D. (2001). Antisense-induced exon skipping and synthesis of dystrophin in the mdx mouse. Proc. Natl. Acad. Sci. USA 98, 42-47.
33. MAURER, C.A., FRIESS, H., BUHLER, S.S., WAHL, B.R., GRABER, H., ZIMMERMANN, A., and BUCHLER, M.W. (1998). Apoptosis inhibiting factor bcl-xL might be the crucial member of the bcl-2 gene family in colorectal cancer. Dig. Dis. Sci. 43, 2641-2648.
34. MERCATANTE, D.R., MOHLER, J.L., and KOLE, R. (2002). Cellular response to an antisense-mediated shift of bcl-x pre-mRNA splicing and antineoplastic agents. J. Biol. Chem. 277, 49374-49382.
35. MERCATANTE, D.R., SAZANI, P., and KOLE, R. (2001). Modification of alternative splicing by antisense oligonucleotides as a potential chemotherapy for cancer and other diseases. Curr. Cancer Drug Targets 1, 211-230.
36. MIYAJIMA, H., MIYASO, H., OKUMURA, M., KURISU, J., and IMAIZUMI, K. (2002). Identification of a cis-acting element for the regulation of SMN exon 7 splicing. J. Biol. Chem. 277, 23271-23277.
37. MIYASO, H., OKUMURA, M., KONDO, S., HIGASHIDE, S., MIYAJIMA, H., and IMAIZUMI, K. (2003). An intronic splicing enhancer element in survival motor neuron (SMN) pre-mRNA. J. Biol. Chem. 278, 15825-15831.
38. MORCOS, P.A. (2001). Achieving efficient delivery of morpholino oligos in cultured cells. Genesis 30, 94-102.
39. NITA, M.E., ONO-NITA, S.K., TSUNO, N., TOMINAGA, O., TAKENOUE, T., SUNAMI, E., KITAYAMA, J., NAKAMURA, Y., and NAGAWA, H. (2000). bcl-X(L) antisense sensitizes human colon cancer cell line to 5-fluorouracil. Jpn. J. Cancer Res. 91, 825-832.
40. REED, J.C. (1998). Bcl-2 family proteins. Oncogene 17, 3225-3236.
41. ROBERTS, G.C., and SMITH, C.W. (2002). Alternative splicing: Combinatorial output from the genome. Curr. Opin. Chem. Biol. 6, 375-383.
42. SAZANI, P., ASTRIAB-FISCHER, A., and KOLE, R. (2003). Effects of base modifications on antisense properties of 2′-O-methoxyethyl and PNA oligonucleotides. Antisense Nucleic Acid Drug Dev. 13, 119-128.
43. SAZANI, P., GEMIGNANI, F., KANG, S.H., MAIER, M.A., MANOHARAN, M., PERSMARK, M., BORTNER, D., and KOLE, R. (2002). Systemically delivered antisense oligomers upregulate gene expression in mouse tissues. Nat. Biotechnol. 20, 1228-1233.
44. SAZANI, P., KANG, S.H., MAIER, M.A., WEI, C., DILLMAN, J., SUMMERTON, J., MANOHARAN, M., and KOLE, R. (2001). Nuclear antisense effects of neutral, anionic and cationic oligonucleotide analogs. Nucleic Acids Res. 29, 3965-3974.
45. SAZANI, P., and KOLE, R. (2003a). Modulation of alternative splicing by antisense oligonucleotides. Prog. Mol. Subcell. Biol. 31, 217-239.
46. SAZANI, P., and KOLE, R. (2003b). Therapeutic potential of antisense oligonucleotides as modulators of alternative splicing. J. Clin. Invest. 112, 481-486.
47. SHEN, L., SIWKOWSKI, A., WANCEWICZ, E.V., LESNIK, E., BUTLER, M., WITCHELL, D., VASQUEZ, G., ROSS, B., ACEVEDO, O., INAMATI, G., SASMOR, H., MANOHARAN, M., and MONIA, B.P. (2003). Evaluation of C-5 propynyl pyrimidine-containing oligonucleotides in vitro and in vivo. Antisense Nucleic Acid Drug Dev. 13, 129-142.
48. SKORDIS, L.A., DUNCKLEY, M.G., YUE, B., EPERON, I.C., and MUNTONI, F. (2003). Bifunctional antisense oligonucleotides provide a trans-acting splicing enhancer that stimulates SMN2 gene expression in patient fibroblasts. Proc. Natl. Acad. Sci. USA 100, 4114-4119.
49. SMITH, C.W., and VALCARCEL, J. (2000). Alternative pre-mRNA splicing: The logic of combinatorial control. Trends Biochem. Sci. 25, 381-388.
50. SUWANMANEE, T., SIERAKOWSKA, H., FUCHAROEN, S., and KOLE, R. (2002a). Repair of a splicing defect in erythroid cells from patients with beta-thalassemia/HbE disorder. Mol. Ther. 6, 718-726.
51. SUWANMANEE, T., SIERAKOWSKA, H., LACERRA, G., SVASTI, S., KIRBY, S., WALSH, C., FUCHAROEN, S., and KOLE, R. (2002b). Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides. Mol. Pharmacol. 62, 545-553.
52. TAYLOR, J.K., ZHANG, Q.Q., MONIA, B.P., MARCUSSON, E.G., and DEAN, N.M. (1999a). Inhibition of bclx(l) expression sensitizes normal human keratinocytes and epithelial cells to apoptotic stimuli. Oncogene 18, 4495-4504.
53. TAYLOR, J.K., ZHANG, Q.Q., WYATT, J.R., and DEAN, N.M. (1999b). Induction of endogenous Bcl-xS through the control of bcl-x pre-mRNA splicing by antisense oligonucleotides. Nat. Biotechnol. 17, 1097-1100.
54. VACEK, M.M., MA, H., GEMIGNANI, F., LACERRA, G., KAFRI, T., and KOLE, R. (2003a). High-level expression of hemoglobin A in human thalassemic erythroid progenitor cells following lentiviral vector delivery of an antisense snRNA. Blood 101, 104-111.
55. VACEK, M.M., SAZANI, P., and KOLE, R. (2003b). Antisense-mediated redirection of mRNA splicing. Cell. Mol. Life Sci. 60, 825-833.
56. VILLEMAIRE, J., DION, I., ELELA, S.A., and CHABOT, B. (2003). Reprogramming alternative pre-messenger RNA splicing through the use of protein-binding antisense oligonucleotides. J. Biol. Chem. 278, 50031-50039.
57. WACHECK, V., SELZER, E., GUNSBERG, P., LUCAS, T., MEYER, H., THALLINGER, C., MONIA, B.P., and JANSEN, B. (2003). Bcl-x(L) antisense oligonucleotides radiosensitise colon cancer cells. Br. J. Cancer 89, 1352-1357.
58. WATANABE, J., KUSHIHATA, F., HONDA, K., MOMINOKI, K., MATSUDA, S., and KOBAYASHI, N. (2002). Bcl-xL overexpression in human hepatocellular carcinoma. Int. J. Oncol. 21, 515-519.
59. WOERFEL, G., and BINDEREIF, A. (2001). In vitro selection of exonic splicing enhancer sequences: Identification of novel CD44 enhancers. Nucleic Acids Res. 29, 3204-3211.
60. XERRI, L., PARC, P., BROUSSET, P., SCHLAIFER, D., HASSOUN, J., REED, J.C., KRAJEWSKI, S., and BIRNBAUM, D. (1996). Predominant expression of the long isoform of bcl-x (bcl-xL) in human lymphomas. Br. J. Haematol. 92, 900-906.
61. XU, Z., FRIESS, H., SOLIOZ, M., AEBI, S., KORC, M., KLEEFF, J., and BUCHLER, M.W. (2001). bcl-x(L) antisense oligonucleotides induce apoptosis and increase sensitivity of pancreatic cancer cells to gemcitabine. Int. J. Cancer 94, 268-274.