Systemic administration of the antisense oligonucleotide NS-065/NCNP-01 for skipping of exon 53 in patients with Duchenne muscular dystrophy
Komaki, Hirofumi, Nagata, Tetsuya, Saito, Takashi, Masuda, Satoru, Takeshita, Eri, Sasaki, Masayuki, Tachimori, Hisateru, Nakamura, Harumasa, Aoki, Yoshitsugu, Takeda, Shin’ichiVolume:
10
Language:
english
Journal:
Science Translational Medicine
DOI:
10.1126/scitranslmed.aan0713
Date:
April, 2018
File:
PDF, 2.11 MB
english, 2018