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

Maria Kinali, Virginia Arechavala-Gomeza, Lucy Feng, Sebahattin Cirak, David Hunt, Carl Adkin, Michela Guglieri, Emma Ashton, Stephen Abbs, Petros Nihoyannopoulos, Maria Elena Garralda, Mary Rutherford, Caroline McCulley, Linda Popplewell, Ian R Graham, George Dickson, Matthew J A Wood, Dominic J Wells, Steve D Wilton, Ryszard KoleVolker Straub, Kate Bushby, Caroline Sewry, Jennifer E Morgan, Francesco Muntoni

Research output: Contribution to journalArticlepeer-review

Original languageEnglish
Pages (from-to)918-928
Number of pages11
JournalLancet Neurology
Issue number10
Early online date26 Aug 2009
Publication statusPublished - Oct 2009


  • Adolescent
  • Child
  • Dose-Response Relationship, Drug
  • Dystrophin
  • Gene Therapy
  • Humans
  • Immunohistochemistry
  • Male
  • Muscle, Skeletal
  • Muscular Dystrophy, Duchenne
  • Oligonucleotides
  • Reverse Transcriptase Polymerase Chain Reaction
  • Single-Blind Method

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