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Manumycin A corrects aberrant splicing of Clcn1 in myotonic dystrophy type 1 (DM1) mice
Myotonic dystrophy type 1 (DM1) is the most common muscular dystrophy in adults and as yet no cure for DM1. Here, we report the potential of manumycin A for a novel DM1 therapeutic reagent. DM1 is caused by expansion of CTG repeat. Mutant transcripts containing expanded CUG repeats lead to aberrant...
Autores principales: | Oana, Kosuke, Oma, Yoko, Suo, Satoshi, Takahashi, Masanori P., Nishino, Ichizo, Takeda, Shin'ichi, Ishiura, Shoichi |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group
2013
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3701899/ https://www.ncbi.nlm.nih.gov/pubmed/23828222 http://dx.doi.org/10.1038/srep02142 |
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