Comparative analysis of antisense oligonucleotide sequences for targeted skipping of Exon 51 during dystrophin Pre-mRNA splicing in human muscle

V. Arechavala-Gomeza, I. R. Graham, L. J. Popplewell, A. M. Adams, A. Aartsma-Rus, M. Kinali, J. E. Morgan, J. C. Van Deutekom, S. D. Wilton, G. Dickson, F. Muntoni

Research output: Contribution to journalArticlepeer-review

Original languageEnglish
Pages (from-to)798-810
Number of pages13
JournalHuman Gene Therapy
Volume18
Issue number9
DOIs
Publication statusPublished - Sept 2007

Keywords

  • DUCHENNE MUSCULAR-DYSTROPHY
  • MDX MOUSE MUSCLE
  • NONSENSE MUTATION
  • SOMATIC REVERSION
  • SYSTEMIC DELIVERY
  • REVERTANT FIBERS
  • GENE TRANSCRIPT
  • POSITIVE FIBERS
  • IN-VITRO
  • EXPRESSION

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