Dosing regimen has a significant impact on the efficiency of morpholino oligomer-induced exon skipping in mdx mice

Alberto Malerba, Francesca C Thorogood, George Dickson, Ian R Graham

Research output: Contribution to journalArticlepeer-review

Original languageEnglish
Pages (from-to)955-65
Number of pages11
JournalHuman Gene Therapy
Issue number9
Publication statusPublished - 2009


  • Animals
  • Disease Models, Animal
  • Dose-Response Relationship, Drug
  • Dystrophin
  • Dystrophin-Associated Protein Complex
  • Exons
  • Gene Therapy
  • Humans
  • Injections, Intramuscular
  • Injections, Intravenous
  • Mice
  • Mice, Inbred mdx
  • Morpholines
  • Muscle, Skeletal
  • Muscular Dystrophy, Animal
  • Muscular Dystrophy, Duchenne
  • Oligonucleotides, Antisense
  • Treatment Outcome

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