Development of an orally available inhibitor of CLK1 for skipping a mutated dystrophin exon in Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a fatal progressive muscle-wasting disease. Various attempts are underway to convert severe DMD to a milder phenotype by modulating the splicing of the dystrophin gene and restoring its expression. In our previous study, we reported TG003, an inhibitor of CDC2-li...

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發表在:Sci Rep
Main Authors: Sako, Yukiya, Ninomiya, Kensuke, Okuno, Yukiko, Toyomoto, Masayasu, Nishida, Atsushi, Koike, Yuka, Ohe, Kenji, Kii, Isao, Yoshida, Suguru, Hashimoto, Naohiro, Hosoya, Takamitsu, Matsuo, Masafumi, Hagiwara, Masatoshi
格式: Artigo
語言:Inglês
出版: Nature Publishing Group 2017
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Article
在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC5448077/
https://ncbi.nlm.nih.gov/pubmed/28555643
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/srep46126
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