Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

Sebahattin Cirak, Virginia Arechavala-Gomeza, Michela Guglieri, Lucy Feng, Silvia Torelli, Karen Anthony, Stephen Abbs, Maria Elena Garralda, John Bourke, Dominic J. Wells, George Dickson, Matthew J. A. Wood, Steve D. Wilton, Volker Straub, Ryszard Kole, Stephen B. Shrewsbury, Caroline Sewry, Jennifer E. Morgan, Kate Bushby, Francesco Muntoni

Research output: Contribution to journalArticlepeer-review

Original languageEnglish
Pages (from-to)595-605
Number of pages11
JournalThe Lancet
Volume378
Issue number9791
Early online date25 Jul 2011
DOIs
Publication statusPublished - 13 Aug 2011

Keywords

  • EXPRESSION
  • PROTEIN
  • SARCOLEMMA
  • EXERCISE
  • MUSCLE
  • ABNORMALITIES
  • PHENOTYPE
  • PRO051
  • MOUSE
  • NNOS

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