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Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1
During drug development, tissue samples serve as indicators of disease activity and pharmacodynamic responses. Reliable non-invasive measures of drug target engagement will facilitate identification of promising new treatments. Here we develop and validate a novel bi-transgenic mouse model of myoton...
Autores principales: | Hu, Ningyan, Antoury, Layal, Baran, Timothy M., Mitra, Soumya, Bennett, C. Frank, Rigo, Frank, Foster, Thomas H., Wheeler, Thurman M. |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Nature Publishing Group UK
2018
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6286378/ https://www.ncbi.nlm.nih.gov/pubmed/30531949 http://dx.doi.org/10.1038/s41467-018-07517-y |
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