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Research ArticleMuscular Dystrophy
Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy
- View ORCID ProfileHirofumi Komaki1,*,
- View ORCID ProfileTetsuya Nagata2,*,
- View ORCID ProfileTakashi Saito2,*,
- Satoru Masuda2,
- Eri Takeshita1,
- View ORCID ProfileMasayuki Sasaki1,
- Hisateru Tachimori3,
- Harumasa Nakamura4,
- Yoshitsugu Aoki2 and
- View ORCID ProfileShin’ichi Takeda2,†
- 1Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
- 2Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
- 3Department of Mental Health Administration, National Institute of Mental Health, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
- 4Translational Medical Center, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
- ↵†Corresponding author. Email: takeda{at}ncnp.go.jp
↵* These authors contributed equally to this work.
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Science Translational Medicine 18 Apr 2018:
Vol. 10, Issue 437, eaan0713
DOI: 10.1126/scitranslmed.aan0713
Vol. 10, Issue 437, eaan0713
DOI: 10.1126/scitranslmed.aan0713
Hirofumi Komaki
1Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
Tetsuya Nagata
2Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
Takashi Saito
2Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
Satoru Masuda
2Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
Eri Takeshita
1Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
Masayuki Sasaki
1Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
Hisateru Tachimori
3Department of Mental Health Administration, National Institute of Mental Health, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
Harumasa Nakamura
4Translational Medical Center, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
Yoshitsugu Aoki
2Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
Shin’ichi Takeda
2Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.
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