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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...
Autores principales: | , , , , , , , , |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
Oxford University Press
2017
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Materias: | |
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 |