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Oligonucleotides against a splicing enhancer sequence led to dystrophin production in muscle cells from a Duchenne muscular dystrophy patient.

AuthorsY Takeshima, H Wada, M Yagi, Y Ishikawa, Y Ishikawa, R Minami, H Nakamura, M Matsuo
JournalBrain & development (Brain Dev) Vol. 23 Issue 8 Pg. 788-90 (Dec 2001) ISSN: 0387-7604 [Print] Netherlands
PMID11720794 (Publication Type: Journal Article, Research Support, Non-U.S. Gov't)
Chemical References
  • DNA, Recombinant
  • Dystrophin
  • Oligonucleotides, Antisense
  • RNA, Messenger
Topics
  • Cells, Cultured
  • Child
  • DNA, Recombinant (genetics)
  • Dystrophin (biosynthesis, genetics)
  • Enhancer Elements, Genetic (genetics)
  • Exons (genetics)
  • Frameshift Mutation (genetics)
  • Genetic Therapy (methods)
  • Humans
  • Male
  • Muscle, Skeletal (metabolism, physiopathology)
  • Muscular Dystrophy, Duchenne (genetics, metabolism, therapy)
  • Oligonucleotides, Antisense (therapeutic use)
  • RNA, Messenger (genetics)

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