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A flow cytometry-based screen identifies MBNL1 modulators that rescue splicing defects in myotonic dystrophy type I

Myotonic dystrophy Type 1 (DM1) is a rare genetic disease caused by the expansion of CTG trinucleotide repeats ((CTG)(exp)) in the 3' untranslated region of the DMPK gene. The repeat transcripts sequester the RNA binding protein Muscleblind-like protein 1 (MBNL1) and hamper its normal function...

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Detalles Bibliográficos
Autores principales:	Zhang, Fan, Bodycombe, Nicole E., Haskell, Keith M., Sun, Yumei L., Wang, Eric T., Morris, Carl A., Jones, Lyn H., Wood, Lauren D., Pletcher, Mathew T.
Formato:	Online Artículo Texto
Lenguaje:	English
Publicado:	Oxford University Press 2017
Materias:	Articles
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5886090/ https://www.ncbi.nlm.nih.gov/pubmed/28535287 http://dx.doi.org/10.1093/hmg/ddx190

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