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...

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Vydáno v:Sci Rep
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: Nature Publishing Group UK 2019
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Article
On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC6405839/
https://ncbi.nlm.nih.gov/pubmed/30846748
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41598-019-40421-z
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