Regulation of pre-mRNA splicing by antisense oligonucleotides

Current Opinion in Drug Discovery and Development

Volumn 2, Issue 2, 1999, Pages 147-151

  Taylor, Jennifer K ^a   Dean, Nicholas M ^a  

^a ISIS PHARMACEUTICALS   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; BETA GLOBIN; COLLAGEN TYPE 1; RIBONUCLEASE H;

ARTICLE; BETA THALASSEMIA; CHEMICAL MODIFICATION; GENE MUTATION; GENE TARGETING; GENE THERAPY; HUMAN; OSTEOGENESIS IMPERFECTA; RNA BINDING; RNA DEGRADATION; RNA SPLICING;

EID: 0032892641     PISSN: 13676733     EISSN: None     Source Type: Journal    
DOI: None     Document Type: Article

References (26)

1. Crooke ST: Therapeutic applications of oligonucleotides. Annu Rev Pharmacol Toxicol (1992) 32:329-376.
2. Crooke ST, Bennett CF: Progress in antisense oligonucleotide therapeutics. Annu Rev Pharmacol Toxicol (1996)36:107-129. An excellent review describing the use of oligonucleotides to treat disease.
3. Stein CA, Cheng YC: Antisense oligonucleotides as therapeutic agents - is the bullet really magical? Science (1993)261:1004-1012.
4. Bennett CF, Dean NM, Ecker DJ, Monia BP: Pharmacological activity of phosphorothioate antisense oligonucleotides: HI vivo studies. In: Methods in Molecular Medicine: Antisense Therapeutics. Agrawal S (Ed). Humana Press, Totowa, NJ, USA(1995):13-46.
5. Monia BP, Dean NM: Pharmacological activity of antisense oligonucleotides in animal models of disease. In: Handbook of Experimental Pharmacology. Vol 131, Antisense Research and Application. Crooke ST (Ed). Springer Press, Heidelberg, Germany (1998):427-443. A comprehensive review of the pharmacological activity of antisense oligonucleotides in vivo.
6. Monia BP: First- and second-generation antisense inhibitors targeted to human c-raf kinase: in vitro and in vivo studies. Anticancer Drug Des (1997) 12:327-339.
7. Orr RM, Monia BP: Antisense therapy for cancer. Curr Res Mol T/;er(1998) 1:102-108.
8. Dean NM, Griffey RH: Identification and characterization of second-generation antisense oligonucleotides. Antisense Nucleic Acid Drug Development (1997) 7:229-233. A thorough review of chemical modifications of antisense oligonucleotides.
9. Cook PD: Medicinal chemistry strategies for antisense research. In: Antisense Research and Applications. Crooke ST, Lebleu B (Eds). CRC Press, Boca Raton, FL, USA (1993):149-187.
10. Sanghvi YS, Cook PD: Towards second-generation synthetic backbones for antisense oligonucleosides. In: Nucleosides, Nucleotides as Antiviral and Antitumor Agents. Chu CK, Baker DC (Eds). Plenum Press, New York (1993):309-322.
11. Kawasaki AM, Casper MD, Freier SM, Lesnik EA, Zounes MC, Cummins LL, Gonzalez C, Cook PD: Uniformly modified 2'deoxy-2'-fluoro phosphorothioate oligonucleotides as nuclease-resistant antisense compounds with high affinity and specificity for RNA targets. J Med Chem (1993) 35:831841.
12. McKay RM, Cummins LL, Graham MJ, Lesnick EA, Owens SR, Winniman M, Dean NM: Enhanced activity of an antisense pligonucleotide targeting murine PKC-alpha by the incorporation of 2'-O-propyl modifications. Nucleic Acids Res (1996) 24:411-417.
13. Iribarren AM, Sproat BS, Neuner P, Sulston I, Ryder U, Lamond AI: 2'-O-alkyl oligoribonucleotides as antisense probes. Proc NatlAcadSci USA (1990) 87:7747-7751.
14. Altmann KH, Dean NM, Fabbro D, Freier SM, Geiger T, Haner R, Husken D, Martin P, Monia BP, Muller M, Natt F, Nickiin P, Phillips J, Pieles U, Sasmor H, Moser HE: Second generation of antisense oligonucleotides: From nuclease resistance to biological efficacy in animals. C/?/m/a(1996) 50:168-176. A thorough review on the biochemical and physiological properties of second generation antisense oligonucleotides.
15. Chabot B: Directing alternative splicing: cast and scenarios. Trends Genet (1996) 12:472-478. A comprehensive overview summarizing what is known about alternative splicing in Drosophila and mammalian cells.
16. Black DL: Finding splice sites within a wilderness of RNA. RNA (1995) 1:763-771. A thorough review on the selection of splice sites in higher eukaryotic mRNA.
17. Kole R: Modification of alternative splicing of pre-mRNA by antisense oligonucleotides. In: Applied Antisense Oligonucleotide Technology. Stein CA, Krieg AM (Eds), WileyLiss Inc, New York, USA (1998) 451-469. An excellent, comprehensive review of the use of antisense oligonucleotides to regulate splicing.
18. Marcusson EG, Bhat B, Manoharan M, Bennett CF, Dean NM: Phosphorothioate oligodeoxyribonucleotides dissociate from cationic lipids before entering the nucleus. Nucleic Acids Res (1998) 26:2016-23.
19. Dominski Z, Kole R: Restoration of correct splicing in thalassemic pre-mRNA by antisense oligonucleotides. Proc Natl Acad Sci USA (1993) 90:8673-8677. First demonstration that antisense oligonucleotides can be used to correct aberrant splicing in vitro.
20. Sierakowska H, Sambade MJ, Agrawal S, Kole R: Repair of thalassemic human -globin mRNA in mammalian cells by antisense oligonucleotides. Proc Natl Acad Sci USA (1996) 93:12840-12844. This is an important paper that demonstrates that it is possible to correct mutant -g!obin gene using chemically-modified oligonucleotides in mammalian cells.
21. Dominski Z, Kole R: Identification and characterization by antisense oligonucleotides of exon and intron sequences required for splicing. Mol Ce//B/o/(1994) 14:7445-7454. This is an interesting paper that describes how important sequences required for RNA splicing were identified using oligonucleotides.
22. Condon TP, Bennett CF: Altered mRNA splicing and inhibition of human E-selectin expression by an antisense oligonucleolide in human umbilical vein endothelial cells. J Biol Chem (1996) 271:30398-30403. This is a convincing report that demonstrates that oligonucleotides target pre-mRNA and can modify RNA splicing.
23. Hodges D, Crooke ST: Inhibition of splicing of wild-type and mutated luciferase adenovirus pre-mRNAs by antisense oligonucleotides. Mo/P/7am7aco/(1995) 45:905-918. Using a luciferase reporter assay, the data demonstrate that the 5', 3' and branchpoint sequences each contribute to splicing efficiency.
24. Kang SH, Cho MJ, Kole R: Dp-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development. Biochemistry (1998) 37:6235-6239. This paper describes a modified luciferase reporter assay and discusses the possible application for general analysis of antisense research.
25. Wang Q, Marini JC: Antisense oligodeoxynucleotides selectively suppress expression of the mutant o2(l) collagen allele in type IV osteogenesis imperfects fibroblasts. A molecular approach to therapeutics of dominant negative disorders. J Clin Invest (1996) 97:448-454. This paper demonstrates that oligonucleotides can be used to selectively suppress mutant mRNA transcripts.
26. Balvay L, Libri D, Fiszman MY: Pre-mRNA secondary structure and the regulation of splicing. B/oEssays(1993) 15:165-169. A review of how secondary structure of pre-mRNA can affect splice site selection.