From dynamic combinatorial ‘hit’ to lead: in vitro and in vivo activity of compounds targeting the pathogenic RNAs that cause myotonic dystrophy

The myotonic dystrophies (DM) are human diseases in which the accumulation of toxic RNA (CUG or CCUG) repeats in the cell causes sequestration of splicing factors, including MBNL1, leading to clinical symptoms such as muscle wasting and myotonia. We previously used Dynamic Combinatorial Chemistry to...

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Detalles Bibliográficos
Autores principales: Ofori, Leslie O., Hoskins, Jason, Nakamori, Masayuki, Thornton, Charles A., Miller, Benjamin L.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2012
Materias:
Synthetic Biology and Chemistry
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3401475/
https://www.ncbi.nlm.nih.gov/pubmed/22492623
http://dx.doi.org/10.1093/nar/gks298

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3401475/
https://www.ncbi.nlm.nih.gov/pubmed/22492623
http://dx.doi.org/10.1093/nar/gks298

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