NS-065/NCNP-01: An Antisense Oligonucleotide for Potential Treatment of Exon 53 Skipping in Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD), the most common lethal heritable childhood disease, is caused by mutations in the DMD gene that result in the absence of functional dystrophin protein. Exon skipping mediated by antisense oligonucleotides has recently emerged as an effective approach for the restor...

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Detalles Bibliográficos
Autores principales: Watanabe, Naoki, Nagata, Tetsuya, Satou, Youhei, Masuda, Satoru, Saito, Takashi, Kitagawa, Hidetoshi, Komaki, Hirofumi, Takagaki, Kazuchika, Takeda, Shin’ichi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2018
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6202794/
https://www.ncbi.nlm.nih.gov/pubmed/30388618
http://dx.doi.org/10.1016/j.omtn.2018.09.017

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6202794/
https://www.ncbi.nlm.nih.gov/pubmed/30388618
http://dx.doi.org/10.1016/j.omtn.2018.09.017

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