Modifications to toxic CUG RNAs induce structural stability, rescue mis-splicing in a myotonic dystrophy cell model and reduce toxicity in a myotonic dystrophy zebrafish model

CUG repeat expansions in the 3′ UTR of dystrophia myotonica protein kinase (DMPK) cause myotonic dystrophy type 1 (DM1). As RNA, these repeats elicit toxicity by sequestering splicing proteins, such as MBNL1, into protein–RNA aggregates. Structural studies demonstrate that CUG repeats can form A-for...

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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: deLorimier, Elaine, Coonrod, Leslie A., Copperman, Jeremy, Taber, Alex, Reister, Emily E., Sharma, Kush, Todd, Peter K., Guenza, Marina G., Berglund, J. Andrew
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2014
Assuntos:
RNA
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4227782/
https://ncbi.nlm.nih.gov/pubmed/25303993
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gku941
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