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Lomofungin and dilomofungin: inhibitors of MBNL1-CUG RNA binding with distinct cellular effects
Myotonic dystrophy type 1 (DM1) is a dominantly inherited neuromuscular disorder resulting from expression of RNA containing an expanded CUG repeat (CUG(exp)). The pathogenic RNA is retained in nuclear foci. Poly-(CUG) binding proteins in the Muscleblind-like (MBNL) family are sequestered in foci, c...
Autores principales: | Hoskins, Jason W., Ofori, Leslie O., Chen, Catherine Z., Kumar, Amit, Sobczak, Krzysztof, Nakamori, Masayuki, Southall, Noel, Patnaik, Samarjit, Marugan, Juan J., Zheng, Wei, Austin, Christopher P., Disney, Matthew D., Miller, Benjamin L., Thornton, Charles A. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2014
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4041448/ https://www.ncbi.nlm.nih.gov/pubmed/24799433 http://dx.doi.org/10.1093/nar/gku275 |
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