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...

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Detalles Bibliográficos
Autores principales: Oana, Kosuke, Oma, Yoko, Suo, Satoshi, Takahashi, Masanori P., Nishino, Ichizo, Takeda, Shin'ichi, Ishiura, Shoichi
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Nature Publishing Group 2013
Materias:
Article
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

Internet

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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