Mismatched single stranded antisense oligonucleotides can induce efficient dystrophin splice switching

BMC Medical Genetics

Volumn 12, Issue , 2011, Pages

  Fragall, Clayton T ^a   Adams, Abbie M ^a   Johnsen, Russell D ^a   Kole, Ryszard ^b   Fletcher, Sue ^a   Wilton, Steve D ^a  

^a UNIVERSITY OF WESTERN AUSTRALIA   (Australia)

^b AVI BioPharma   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; DYSTROPHIN; MESSENGER RNA; OLIGOMER;

ARTICLE; CHEMICAL BINDING; CONTROLLED STUDY; DYSTROPHY; EXON; GENE INSERTION; GENE MUTATION; GENE SEQUENCE; GENE SWITCHING; GENETIC TRANSCRIPTION; HUMAN; HUMAN CELL; NORMAL HUMAN; RNA SPLICING; CELL CULTURE; DUCHENNE MUSCULAR DYSTROPHY; GENE EXPRESSION REGULATION; GENETICS; MISMATCH REPAIR; NUCLEOTIDE SEQUENCE;

CELLS, CULTURED; DNA MISMATCH REPAIR; DYSTROPHIN; EXONS; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; MUTAGENESIS, INSERTIONAL; OLIGONUCLEOTIDES, ANTISENSE; READING FRAMES; RNA SPLICING;

EID: 80054680717     PISSN: None     EISSN: 14712350     Source Type: Journal    
DOI: 10.1186/1471-2350-12-141     Document Type: Article

References (23)

1. van Deutekom JC, Janson AA, Ginjaar IB, Frankhuizen WS, Aartsma-Rus A, Bremmer-Bout M, den Dunnen JT, Koop K, van der Kooi AJ, Goemans NM, et al. Local dystrophin restoration with antisense oligonucleotide PRO051. N Engl J Med 2007, 357:2677-2686. 10.1056/NEJMoa073108, 18160687.
2. Kinali M, Arechavala-Gomeza V, Feng L, Cirak S, Hunt D, Adkin C, Guglieri M, Ashton E, Abbs S, Nihoyannopoulos P, et al. Local restoration of dystrophin expression with the morpholino oligomer AVI-4658 in Duchenne muscular dystrophy: a single-blind, placebo-controlled, dose-escalation, proof-of-concept study. Lancet Neurol 2009, 8:918-928. 10.1016/S1474-4422(09)70211-X, 2755039, 19713152.
3. Goemans NM, Tulinius M, van den Akker JT, Burm BE, Ekhart PF, Heuvelmans N, Holling T, Janson AA, Platenburg GJ, Sipkens JA, et al. Systemic Administration of PRO051 in Duchenne's Muscular Dystrophy. N Engl J Med 2011,
4. Cirak S, Arechavala-Gomeza V, Guglieri M, Feng L, Torelli S, Anthony K, Abbs S, Garralda ME, Bourke J, Wells DJ, et al. Exon skipping and dystrophin restoration in Duchenne Muscular Dystrophy patients after systemic phosphorodiamidate morpholino oligomer treatment. Lancet
5. Wilton SD, Fall AM, Harding PL, McClorey G, Coleman C, Fletcher S. Antisense oligonucleotide-induced exon skipping across the human dystrophin gene transcript. Mol Ther 2007, 15:1288-1296. 10.1038/sj.mt.6300095, 17285139.
6. Adams AM, Harding PL, Iversen PL, Coleman C, Fletcher S, Wilton SD. Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries. BMC Mol Biol 2007, 8:57. 10.1186/1471-2199-8-57, 1933433, 17601349.
7. Mann CJ, Honeyman K, McClorey G, Fletcher S, Wilton SD. Improved antisense oligonucleotide induced exon skipping in the mdx mouse model of muscular dystrophy. J Gene Med 2002, 4:644-654. 10.1002/jgm.295, 12439856.
8. Jearawiriyapaisarn N, Moulton HM, Buckley B, Roberts J, Sazani P, Fucharoen S, Iversen PL, Kole R. Sustained dystrophin expression induced by peptide-conjugated morpholino oligomers in the muscles of mdx mice. Mol Ther 2008, 16:1624-1629. 10.1038/mt.2008.120, 2671676, 18545222.
9. Moulton HM, Fletcher S, Neuman BW, McClorey G, Stein DA, Abes S, Wilton SD, Buchmeier MJ, Lebleu B, Iversen PL. Cell-penetrating peptide-morpholino conjugates alter pre-mRNA splicing of DMD (Duchenne muscular dystrophy) and inhibit murine coronavirus replication in vivo. Biochem Soc Trans 2007, 35:826-828. 10.1042/BST0350826, 17635157.
10. Rando TA, Blau HM. Primary mouse myoblast purification, characterization, and transplantation for cell-mediated gene therapy. J Cell Biol 1994, 125:1275-1287. 10.1083/jcb.125.6.1275, 2290930, 8207057.
11. Lattanzi L, Salvatori G, Coletta M, Sonnino C, Cusella De Angelis MG, Gioglio L, Murry CE, Kelly R, Ferrari G, Molinaro M, et al. High efficiency myogenic conversion of human fibroblasts by adenoviral vector-mediated MyoD gene transfer. An alternative strategy for ex vivo gene therapy of primary myopathies. J Clin Invest 1998, 101:2119-2128. 10.1172/JCI1505, 508800, 9593768.
12. Harding PL, Fall AM, Honeyman K, Fletcher S, Wilton SD. The influence of antisense oligonucleotide length on dystrophin exon skipping. Mol Ther 2006, 15:157-166.
13. Wilton SD, Lim L, Dye D, Laing N. Bandstab: a PCR-based alternative to cloning PCR products. Biotechniques 1997, 22:642-645.
14. Cooper ST, Lo HP, North KN. Single section Western blot: improving the molecular diagnosis of the muscular dystrophies. Neurology 2003, 61:93-97.
15. Nicholson LV, Davison K, Falkous G, Harwood C, O'Donnell E, Slater CR, Harris JB. Dystrophin in skeletal muscle. I. Western blot analysis using a monoclonal antibody. J Neurol Sci 1989, 94:125-136. 10.1016/0022-510X(89)90223-2, 2693617.
16. Cartegni L, Wang J, Zhu Z, Zhang MQ, Krainer AR. ESEfinder: A web resource to identify exonic splicing enhancers. Nucleic Acids Res 2003, 31:3568-3571. 10.1093/nar/gkg616, 169022, 12824367.
17. Fairbrother WG, Yeo GW, Yeh R, Goldstein P, Mawson M, Sharp PA, Burge CB. RESCUE-ESE identifies candidate exonic splicing enhancers in vertebrate exons. Nucleic Acids Res 2004, 32:W187-190. 10.1093/nar/gkh393, 441531, 15215377.
18. Harding PL, Fall AM, Honeyman K, Fletcher S, Wilton SD. The influence of antisense oligonucleotide length on dystrophin exon skipping. Mol Ther 2007, 15:157-166. 10.1038/sj.mt.6300006, 17164787.
19. Forrest S, Meloni PL, Muntoni F, Kim J, Fletcher S, Wilton SD. Personalized exon skipping strategies to address clustered non-deletion dystrophin mutations. Neuromuscul Disord 2010, 20:810-816. 10.1016/j.nmd.2010.07.276, 20817455.
20. Errington SJ, Mann CJ, Fletcher S, Wilton SD. Target selection for antisense oligonucleotide induced exon skipping in the dystrophin gene. J Gene Med 2003, 5:518-527. 10.1002/jgm.361, 12797117.
21. Aartsma-Rus A, Kaman WE, Bremmer-Bout M, Janson AA, den Dunnen JT, van Ommen GJ, van Deutekom JC. Comparative analysis of antisense oligonucleotide analogs for targeted DMD exon 46 skipping in muscle cells. Gene Ther 2004, 11:1391-1398. 10.1038/sj.gt.3302313, 15229633.
22. Heemskerk HA, de Winter CL, de Kimpe SJ, van Kuik-Romeijn P, Heuvelmans N, Platenburg GJ, van Ommen GJ, van Deutekom JC, Aartsma-Rus A. In vivo comparison of 2'-O-methyl phosphorothioate and morpholino antisense oligonucleotides for Duchenne muscular dystrophy exon skipping. J Gene Med 2009, 11:257-266. 10.1002/jgm.1288, 19140108.
23. Filipowicz W, Bhattacharyya SN, Sonenberg N. Mechanisms of post-transcriptional regulation by microRNAs: are the answers in sight?. Nat Rev Genet 2008, 9:102-114.