Research ArticleMuscular Dystrophy

Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy

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Science Translational Medicine  18 Apr 2018:
Vol. 10, Issue 437, eaan0713
DOI: 10.1126/scitranslmed.aan0713

Article Information

vol. 10 no. 437

PubMed: 
Print ISSN: 
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History: 
  • Received for publication March 1, 2017
  • Accepted for publication November 17, 2017
  • .

Author Information

  1. Hirofumi Komaki1,*,
  2. Tetsuya Nagata2,*,
  3. Takashi Saito2,*,
  4. Satoru Masuda2,
  5. Eri Takeshita1,
  6. Masayuki Sasaki1,
  7. Hisateru Tachimori3,
  8. Harumasa Nakamura4,
  9. Yoshitsugu Aoki2 and
  10. Shin’ichi Takeda2,
  1. 1Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
  2. 2Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
  3. 3Department of Mental Health Administration, National Institute of Mental Health, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
  4. 4Translational Medical Center, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
  1. Corresponding author. Email: takeda{at}ncnp.go.jp
    • * These authors contributed equally to this work.

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