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Exon 44 skipping in Duchenne muscular dystrophy: NS-089/NCNP-02, a dual-targeting antisense oligonucleotide

Exon-skipping therapy mediated by antisense oligonucleotides is expected to provide a therapeutic option for Duchenne muscular dystrophy. Antisense oligonucleotides for exon skipping reported so far target a single continuous sequence in or around the target exon. In the present study, we investigat...

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Detalles Bibliográficos
Autores principales:	Watanabe, Naoki, Tone, Yuichiro, Nagata, Tetsuya, Masuda, Satoru, Saito, Takashi, Motohashi, Norio, Takagaki, Kazuchika, Aoki, Yoshitsugu, Takeda, Shin’ichi
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2023
Materias:	Original Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10579524/ https://www.ncbi.nlm.nih.gov/pubmed/37854955 http://dx.doi.org/10.1016/j.omtn.2023.102034

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