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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...

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Publicat a:Nat Commun
Autors principals: Hu, Ningyan, Antoury, Layal, Baran, Timothy M., Mitra, Soumya, Bennett, C. Frank, Rigo, Frank, Foster, Thomas H., Wheeler, Thurman M.
Format: Artigo
Idioma:Inglês
Publicat: Nature Publishing Group UK 2018
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Accés en línia:https://ncbi.nlm.nih.gov/pmc/articles/PMC6286378/
https://ncbi.nlm.nih.gov/pubmed/30531949
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41467-018-07517-y
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