Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1

Non-invasive monitoring of alternative splicing outcomes to identify candidate therapies for myotonic dystrophy type 1

Myotonic dystrophy type 1 (DM1) is associated with aberrant transcript splicing. Here, the authors develop a transgenic mouse model expressing a bi-chromatic reporter system that allows non-invasive monitoring of splicing of a transcript altered in DM1 in vivo, and show that it allows for evaluation...

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Autores principales: Ningyan Hu, Layal Antoury, Timothy M. Baran, Soumya Mitra, C. Frank Bennett, Frank Rigo, Thomas H. Foster, Thurman M. Wheeler
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2018
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Science
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Acceso en línea:https://doaj.org/article/b1565fb81d1d487aac6b620c6b16e1b3
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