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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...

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Detalles Bibliográficos
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.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2014
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