Morpholino antisense oligonucleotide induced dystrophin exon 23 skipping in mdx mouse muscle

Human Molecular Genetics

Volumn 12, Issue 15, 2003, Pages 1801-1811

  Gebski, Bianca L ^a   Mann, Chrisopher J ^a   Fletcher, Susan ^a   Wilton, Stephen D ^a  

^a UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

Author keywords

[No Author keywords available]

Indexed keywords

1,2 DIELAIDOYLPHOSPHATIDYLETHANOLAMINE; 1,2-DIELAIDOYLPHOSPHATIDYLETHANOLAMINE; ANTISENSE OLIGODEOXYNUCLEOTIDE; ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; MESSENGER RNA; MORPHOLINE DERIVATIVE; PHOSPHATIDYLETHANOLAMINE;

ALTERNATIVE RNA SPLICING; ANIMAL; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CELL CULTURE; CELLULAR DISTRIBUTION; COMPARATIVE STUDY; CONCENTRATION RESPONSE; CONTROLLED STUDY; DISEASE MODEL; DRUG DESIGN; DRUG EFFICACY; DRUG SAFETY; EXON; GENETIC PROCEDURES; GENETICS; IMMUNOHISTOCHEMISTRY; IN VIVO STUDY; MOLECULAR GENETICS; MOUSE; MOUSE STRAIN; MUSCULAR DYSTROPHY; NONHUMAN; NONVIRAL GENE DELIVERY SYSTEM; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; PROTEIN SYNTHESIS; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; SARCOLEMMA; TREATMENT OUTCOME; WESTERN BLOTTING;

ALTERNATIVE SPLICING; ANIMALS; BASE SEQUENCE; BLOTTING, WESTERN; CELLS, CULTURED; DISEASE MODELS, ANIMAL; DYSTROPHIN; EXONS; IMMUNOHISTOCHEMISTRY; MICE; MICE, INBRED MDX; MOLECULAR SEQUENCE DATA; MUSCULAR DYSTROPHIES; OLIGODEOXYRIBONUCLEOTIDES, ANTISENSE; PHOSPHATIDYLETHANOLAMINES; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION; RNA, MESSENGER;

ANIMALIA;

EID: 0043133425     PISSN: 09646906     EISSN: None     Source Type: Journal    
DOI: 10.1093/hmg/ddg196     Document Type: Article

References (38)

1. Hoffman, E.P., Brown, R.H., Jr and Kunkel, L.M. (1987) Dystrophin: the protein product of the Duchenne muscular dystrophy locus. Cell, 51, 919-928.
2. Emery, A.E. (2002) The muscular dystrophies. Lancet, 359, 687-695.
3. Emery, A.E. (1991) Population frequencies of inherited neuromuscular diseases - a world survey. Neuromusc. Disord., 1, 19-29.
4. Hoffman, E.P., Fischbeck, K.H., Brown, R.H., Johnson, M., Medori, R., Loike, J.D., Harris, J.B., Waterston, R., Brooke, M., Specht, L. et al. (1988) Characterization of dystrophin in muscle-biopsy specimens from patients with Duchenne's or Becker's muscular dystrophy. New Engl. J. Med., 318, 1363-1368.
5. Muntoni, F., Fisher, I., Morgan, J.E. and Abraham, D. (2002) Steroids in Duchenne muscular dystrophy: from clinical trials to genomic research. Neuromusc. Disord., 12, S162-S165.
6. Bach, J.R., Ishikawa, Y. and Kim, H. (1997) Prevention of pulmonary morbidity for patients with Duchenne muscular dystrophy. Chest, 112, 1024-1028.
7. Fletcher, S., Wilton, S.D. and Howell, J.M. (2000) Gene therapy and molecular approaches to the treatment of hereditary muscular disorders. Curr. Opin. Neurol., 13, 553-560.
8. Wilton, S.D., Lloyd, F., Carville, K., Fletcher, S., Honeyman, K., Agrawal, S. and Kole, R. (1999) Specific removal of the nonsense mutation from the mdx dystrophin mRNA using antisense oligonucleotides. Neuromusc. Disord., 9, 330-338.
9. 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.
10. Mann, C.J., Honeyman, K., McClorey, G., Fletcher, S. and Wilton, S.D. (2002) Improved antisense oligionucleotide induced exon skipping in the mdx mouse of muscular dystrophy. J. Gene Med., 4, 1-11.
11. van Deutekom, J.C., Bremmer-Bout, M., Janson, A.A., Ginjaar, I.B., Baas, F., den Dunnen, J.T. and van Ommen, G.J. (2001) Antisense-induced exon skipping restores dystrophin expression in DMD patient derived muscle cells. Hum. Mol. Genet., 10, 1547-1554.
12. Summerton, J. and Weller, D. (1997) Morpholino antisense oligomers: design, preparation, and properties. Antisense Nucl. Acid Drug Dev., 7, 187-195.
13. 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. Nucl. Acids Res., 29, 3965-3974.
14. Suwanmanee, T., Sierakowska, H., Lacerra, G., Svasti, S., Kirby, S., Walsh, C.E., Fucharoen, S. and Kole, R. (2002) Restoration of human beta-globin gene expression in murine and human IVS2-654 thalassemic erythroid cells by free uptake of antisense oligonucleotides. Mol. Pharmac., 62, 545-553.
15. Morcos, P.A. (2001) Achieving efficient delivery of morpholino oligos in cultured cells. Genesis, 30, 94-102.
16. Doyle, D.F., Braasch, D.A., Simmons, C.G., Janowski, B.A. and Corey, D.R. (2001) Inhibition of gene expression inside cells by peptide nucleic acids: effect of mRNA target sequence, mismatched bases, and PNA length. Biochemistry, 40, 53-64.
17. Takeshima, Y., Wada, H., Yagi, M., Ishikawa, Y., Ishikawa, Y., Minami, R., Nakamura, H. and Matsuo, M. (2001) Oligonucleotides against a splicing enhancer sequence led to dystrophin production in muscle cells from a Duchenne muscular dystrophy patient. Brain Dev., 23, 788-790.
18. Errington, S.J., Mann, C.J., Fletcher, S. and Wilton, S.D. (2003) Target Selection for Antisense Oligonucleotide Induced Exon Skipping in the Dystrophin Gene. J. Gene Med., 5, 518-527.
19. Takeshima, Y., Nishio, H., Sakamoto, H., Nakamura, H. and Matsuo, M. (1995) Modulation of in vitro splicing of the upstream intron by modifying an intra-exon sequence which is deleted from the dystrophin gene in dystrophin Kobe. J. Clin. Invest., 95, 515-520.
20. Hudziak, R.M., Barofsky, E., Barofsky, D.F., Weller, D.L., Huang, S.B. and Weller, D.D. (1996) Resistance of morpholino phosphorodiamidate oligomers to enzymatic degradation. Antisense Nucl. Acid Drug Dev., 6, 267-272.
21. Kang, S.H., Cho, M.J. and Kole, R. (1998) Up-regulation of luciferase gene expression with antisense oligonucleotides: implications and applications in functional assay development. Biochemistry, 37, 6235-6239.
22. Agrawal, S., Jiang, Z., Zhao, Q., Shaw, D., Cai, Q., Roskey, A., Channavajjala, L., Saxinger, C. and Zhang, R. (1997) Mixed-backbone oligonucleotides as second generation antisense oligonucleotides: in vitro and in vivo studies. Proc. Natl Acad. Sci. USA, 94, 2620-2625.
23. Stein, C.A., Subasinghe, C., Shinozuka, K. and Cohen, J.S. (1988) Physicochemical properties of phosphorothioate oligodeoxynucleotides. Nucl. Acids Res., 16, 3209-3221.
24. Cummins, L.L., Owens, S.R., Risen, L.M., Lesnik, E.A., Freier, S.M., McGee, D., Guinosso, C.J. and Cook, P.D. (1995) Characterization of fully 2′-modified oligoribonucleotide hetero- and homoduplex hybridization and nuclease sensitivity. Nucl. Acids Res., 23, 2019-2024.
25. Seeberger, P.H. and Caruthers, M.H. (1998) Modified oligonucleotides as antisense therapeutics. In Stein, C.A. and Krieg, A.M. (eds), Applied Antisense Oligonucleotide Technology. Wiley, New York, pp. 51-71.
26. Crooke, R.M., Graham, M.J., Cooke, M.E. and Crooke, S.T. (1995) In vitro pharmacokinetics of phosphorothioate antisense oligonucleotides. J. Pharmac. Exp. Ther., 275, 462-473.
27. Agrawal, S., Temsamani, J. and Tang, J.Y. (1991) Pharmacokinetics, biodistribution, and stability of oligodeoxynucleotide phosphorothioates in mice. Proc. Natl Acad. Sci. USA, 88, 7595-7599.
28. Hoffman, E.P., Morgan, J.E., Watkins, S.C. and Partridge, T.A. (1990) Somatic reversion/suppression of the mouse mdx phenotype in vivo. J. Neurol. Sci., 99, 9-25.
29. Lu, Q.L., Morris, G.E., Wilton, S.D., Ly, T., Artem'yeva, O.V., Strong, P. and Partridge, T.A. (2000) Massive idiosyncratic exon skipping corrects the nonsense mutation in dystrophic mouse muscle and produces functional revertant fibers by clonal expansion. J. Cell Biol., 148, 985-996.
30. Nicholson, L.V., Bushby, K.M., Johnson, M.A., den Dunnen, J.T., Ginjaar, I.B. and van Ommen, G.J. (1992) Predicted and observed sizes of dystrophin in some patients with gene deletions that disrupt the open reading frame. J. Med. Genet., 29, 892-896.
31. Fanin, M., Danieli, G.A., Cadaldini, M., Miorin, M., Vitiello, L. and Angelini, C. (1995) Dystrophin-positive fibers in Duchenne dystrophy: origin and correlation to clinical course. Muscle Nerve, 18, 1115-1120.
32. Cox, G.A., Sunada, Y., Campbell, K.P. and Chamberlain, J.S. (1994) Dp71 can restore the dystrophin-associated glycoprotein complex in muscle but fails to prevent dystrophy. Nat. Genet. 8, 333-339.
33. Wells, K.E., Torelli, S., Lu, Q., Brown, S.C., Partridge, T., Muntoni, F. and Wells, D.J. (2003) Relocalization of neuronal nitric oxide synthase (nNOS) as a marker for complete restoration of the dystrophin associated protein complex in skeletal muscle. Neuromusc. Disord., 13, 21-31.
34. Squire, S., Raymackers, J.M., Vandebrouck, C., Potter, A., Tinsley, J., Fisher, R., Gillis, J.M. and Davies, K.E. (2002) Prevention of pathology in mdx mice by expression of utrophin: analysis using an inducible transgenic expression system. Hum. Mol. Genet., 11, 3333-3344.
35. 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.
36. Litzinger, D.C., Brown, J.M., Wala, I., Kaufman, S.A., Van, G.Y., Farrell, C.L. and Collins, D. (1996) Fate of cationic liposomes and their complex with oligonucleotide in vivo. Biochim. Biophys. Acta, 1281, 139-149.
37. Allen, T.M., Murray, L., MacKeigan, S. and Shah, M. (1984) Chronic liposome administration in mice: effects on reticuloendothelial function and tissue distribution. J. Pharmac. Exp. Ther., 229, 267-275.
38. Braasch, D.A. and Corey, D.R. (2001) Synthesis, analysis, purification, and intracellular delivery of peptide nucleic acids. Methods, 23, 97-107.