Neuromuscular Disorders
Volume 18, Issue 3 , Pages 268-275 , March 2008

149th ENMC International Workshop and 1st TREAT-NMD Workshop on: “Planning Phase I/II Clinical trials using Systemically Delivered Antisense Oligonucleotides in Duchenne Muscular Dystrophy”

  • Francesco Muntoni

      Affiliations

    • Dubowitz Neuromuscular Centre, Institute of Child Health & Great Ormond Street Hospital, 30 Guilford Street, London WC1N 1EH, UK
    • Corresponding Author InformationCorresponding author. Tel.: +44 208 383 3295; fax: +44 208 7462187.
    • ,
  • Kate D. Bushby

      Affiliations

    • Institute of Human Genetics, International Centre for Life, Newcastle upon Tyne, UK
    • ,
  • Gertjan van Ommen

      Affiliations

    • Leiden University Medical Center, Leiden, The Netherlands

Received 15 November 2007

References 

  1. Wilton SD, Fletcher S. Modification of pre-mRNA processing: application to dystrophin expression. Curr Opin Mol Ther. 2006;8(2):130–135
  2. Aartsma-Rus A, et al. Therapeutic modulation of DMD splicing by blocking exonic splicing enhancer sites with antisense oligonucleotides. Ann N Y Acad Sci. 2006;1082:74–76
  3. Alter J, et al. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med. 2006;12(2):175–177
  4. Lu QL, et al. Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci USA. 2005;102(1):198–203
  5. Fletcher S, et al. Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide. J Gene Med. 2006;8(2):207–216
  6. Takeshima Y, et al. Intraperitoneal administration of phosphorothioate antisense oligodeoxynucleotide against splicing enhancer sequence induced exon skipping in dystrophin mRNA expressed in mdx skeletal muscle. Brain Dev. 2005;27(7):488–493
  7. Van Deutekom J, Janson AA, Gingjaar IB, Frankhuizen WS, Aartsma-Rus A, Bremmer_Bout M, et al. Local Dystrophin Restoration with Antisense Oligonucleotide PRO051. N Engl J Med. 2007;357:2677–2686
  8. Bremmer-Bout M, et al. Targeted exon skipping in transgenic hDMD mice: a model for direct preclinical screening of human-specific antisense oligonucleotides. Mol Ther. 2004;10(2):232–240
  9. Adams AM, et al. Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries. BMC Mol Biol. 2007;8:57
  10. Henry SP, et al. Drug properties of second-generation antisense oligonucleotides: how do they measure up to their predecessors?. Curr Opin Invest Drugs. 2001;2(10):1444–1449
  11. Ekker SC. Morphants: a new systematic vertebrate functional genomics approach. Yeast. 2000;17(4):302–306
  12. S.P. Henry, et al., in: S.T. Crooke(Eds.), Antisense Drug Technology, Chapter 12: 2007, pp. 327–364.
  13. Kwoh J. An overview of the clinical safety experience of first- and second-generation antisense oligonucleotides. Antis Drug Technol. 2007;Chapter 13:365–400
  14. Goyenvalle A, et al. Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science. 2004;306(5702):1796–1799
  15. Beroud C, et al. Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy. Hum Mutat. 2007;28(2):196–202
  16. Aartsma-Rus A, et al. Exploring the frontiers of therapeutic exon skipping for Duchenne muscular dystrophy by double targeting within one or multiple exons. Mol Ther. 2006;14(3):401–407
  17. Harding PL, et al. The influence of antisense oligonucleotide length on dystrophin exon skipping. Mol Ther. 2007;15(1):157–166
  18. Arechavala-Gomeza V, et al. Comparative analysis of antisense oligonucleotide sequences for targeted skipping of exon 51 during dystrophin pre-mRNA splicing in human muscle. Hum Gene Ther. 2007;18(9):798–810
  19. Scott E, Mawson SJ. Measurement in Duchenne muscular dystrophy: considerations in the development of a neuromuscular assessment tool. Dev Med Child Neurol. 2006;48(6):540–544
  20. Hoen PA, et al. Gene expression profiling to monitor therapeutic and adverse effects of antisense therapies for Duchenne muscular dystrophy. Pharmacogenomics. 2006;7(3):281–297

PII: S0960-8966(07)00768-7

doi: 10.1016/j.nmd.2007.11.010

Neuromuscular Disorders
Volume 18, Issue 3 , Pages 268-275 , March 2008

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