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"

Neuromuscular Disorders

Volumn 18, Issue 3, 2008, Pages 268-275

  Muntoni, Francesco ^a   Bushby, Kate D ^b   van Ommen, Gertjan ^c  

^a GREAT ORMOND STREET HOSPITAL   (United Kingdom)

^b INSTITUTE OF HUMAN GENETICS   (United Kingdom)

^c LEIDEN UNIVERSITY MEDICAL CENTER   (Netherlands)

Author keywords

Antisense oligonucleotides; Duchenne muscular dystrophy; Exon skipping; Therapeutic trials

Indexed keywords

ANTISENSE OLIGONUCLEOTIDE; BIOLOGICAL MARKER;

ARTICLE; CLINICAL STUDY; COST; DUCHENNE MUSCULAR DYSTROPHY; ETHICS; EXON; OUTCOME ASSESSMENT; PRIORITY JOURNAL; RATING SCALE;

CLINICAL TRIALS, PHASE I AS TOPIC; CLINICAL TRIALS, PHASE II AS TOPIC; GENE THERAPY; HUMANS; MUSCULAR DYSTROPHY, DUCHENNE; OLIGONUCLEOTIDES, ANTISENSE;

EID: 41549104205     PISSN: 09608966     EISSN: None     Source Type: Journal    
DOI: 10.1016/j.nmd.2007.11.010     Document Type: Article

References (20)

1. Wilton S.D., and Fletcher S. Modification of pre-mRNA processing: application to dystrophin expression. Curr Opin Mol Ther 8 2 (2006) 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 1082 (2006) 74-76
3. Alter J., et al. Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology. Nat Med 12 2 (2006) 175-177
4. Lu Q.L., et al. Systemic delivery of antisense oligoribonucleotide restores dystrophin expression in body-wide skeletal muscles. Proc Natl Acad Sci USA 102 1 (2005) 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 8 2 (2006) 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 27 7 (2005) 488-493
7. Van Deutekom J., Janson A.A., Gingjaar I.B., Frankhuizen W.S., Aartsma-Rus A., Bremmer_Bout M., et al. Local Dystrophin Restoration with Antisense Oligonucleotide PRO051. N Engl J Med 357 (2007) 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 10 2 (2004) 232-240
9. Adams A.M., et al. Antisense oligonucleotide induced exon skipping and the dystrophin gene transcript: cocktails and chemistries. BMC Mol Biol 8 (2007) 57
10. Henry S.P., et al. Drug properties of second-generation antisense oligonucleotides: how do they measure up to their predecessors?. Curr Opin Invest Drugs 2 10 (2001) 1444-1449
11. Ekker S.C. Morphants: a new systematic vertebrate functional genomics approach. Yeast 17 4 (2000) 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 Chapter 13 (2007) 365-400
14. Goyenvalle A., et al. Rescue of dystrophic muscle through U7 snRNA-mediated exon skipping. Science 306 5702 (2004) 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 28 2 (2007) 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 14 3 (2006) 401-407
17. Harding P.L., et al. The influence of antisense oligonucleotide length on dystrophin exon skipping. Mol Ther 15 1 (2007) 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 18 9 (2007) 798-810
19. Scott E., and Mawson S.J. Measurement in Duchenne muscular dystrophy: considerations in the development of a neuromuscular assessment tool. Dev Med Child Neurol 48 6 (2006) 540-544
20. Hoen P.A., et al. Gene expression profiling to monitor therapeutic and adverse effects of antisense therapies for Duchenne muscular dystrophy. Pharmacogenomics 7 3 (2006) 281-297