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Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

  • Julia Alter
  • , Fang Lou
  • , Adam Rabinowitz
  • , Hai Fang Yin
  • , Jeffrey Rosenfeld
  • , Steve D. Wilton
  • , Terence A. Partridge
  • , Long Lu Qi

Research output: Contribution to journalArticlepeer-review

Original languageEnglish
Pages (from-to)175-177
Number of pages3
JournalNature Medicine
Volume12
Issue number2
DOIs
StatePublished - Feb 2006
Externally publishedYes

Keywords

  • Animals
  • Base Sequence
  • Drug Administration Schedule
  • Dystrophin/genetics
  • Gene Expression Regulation
  • Genetic Therapy
  • Humans
  • Injections, Intravenous
  • Male
  • Mice
  • Mice, Inbred C57BL
  • Mice, Inbred mdx
  • Muscle, Skeletal
  • Muscular Dystrophy, Animal/genetics
  • Muscular Dystrophy, Duchenne/genetics
  • Oligodeoxyribonucleotides, Antisense/administration & dosage

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