Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy-Reference-Cited by-同舟云学术

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

Published:2018-04-18 Issue:437 Volume:10 Page:
ISSN:1946-6234
Container-title:Science Translational Medicine
language:en
Short-container-title:Sci. Transl. Med.

Author:

Komaki Hirofumi¹^ORCID,Nagata Tetsuya²^ORCID,Saito Takashi²^ORCID,Masuda Satoru²,Takeshita Eri¹,Sasaki Masayuki¹^ORCID,Tachimori Hisateru³,Nakamura Harumasa⁴,Aoki Yoshitsugu²,Takeda Shin’ichi²^ORCID

Affiliation:

1. Department of Child Neurology, National Center Hospital, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.

2. Department of Molecular Therapy, National Institute of Neuroscience, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.

3. Department of Mental Health Administration, National Institute of Mental Health, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.

4. Translational Medical Center, National Center of Neurology and Psychiatry, 4-1-1 Ogawa-Higashi, Kodaira, Tokyo, Japan.

Abstract

NS-065/NCNP-01, an antisense oligonucleotide that enables exon 53 skipping in the dystrophin gene, showed a favorable safety profile and promising pharmacokinetics in 10 patients with Duchenne muscular dystrophy.

Publisher

American Association for the Advancement of Science (AAAS)

Subject

General Medicine

Reference36 articles.

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4. Dystrophin: The protein product of the duchenne muscular dystrophy locus

5. Functional amounts of dystrophin produced by skipping the mutated exon in the mdx dystrophic mouse

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