Modelling Duchenne muscular dystrophy in MYOD1-converted urine-derived cells treated with 3-deazaneplanocin A hydrochloride

Duchenne muscular dystrophy (DMD) is a severe muscle disorder characterised by mutations in the DMD gene. Recently, we have completed a phase I study in Japan based on systemic administration of the morpholino antisense that is amenable to exon-53 skipping, successfully. However, to achieve the effe...

Descripción completa

Detalles Bibliográficos
Autores principales: Takizawa, Hotake, Hara, Yuko, Mizobe, Yoshitaka, Ohno, Taisuke, Suzuki, Sadafumi, Inoue, Ken, Takeshita, Eri, Shimizu-Motohashi, Yuko, Ishiyama, Akihiko, Hoshino, Mikio, Komaki, Hirofumi, Takeda, Shin’ichi, Aoki, Yoshitsugu
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group UK 2019
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6405839/
https://www.ncbi.nlm.nih.gov/pubmed/30846748
http://dx.doi.org/10.1038/s41598-019-40421-z

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6405839/
https://www.ncbi.nlm.nih.gov/pubmed/30846748
http://dx.doi.org/10.1038/s41598-019-40421-z

Ejemplares similares