Triplet-repeat oligonucleotide-mediated reversal of RNA toxicity in myotonic dystrophy

Myotonic dystrophy type 1 (DM1) is caused by toxicity of an expanded, noncoding (CUG)n tract in DM protein kinase (DMPK) transcripts. According to current evidence the long (CUG)n segment is involved in entrapment of muscleblind (Mbnl) proteins in ribonuclear aggregates and stabilized expression of...

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Detalhes bibliográficos
Main Authors: Mulders, Susan A. M., van den Broek, Walther J. A. A., Wheeler, Thurman M., Croes, Huib J. E., van Kuik-Romeijn, Petra, de Kimpe, Sjef J., Furling, Denis, Platenburg, Gerard J., Gourdon, Geneviève, Thornton, Charles A., Wieringa, Bé, Wansink, Derick G.
Formato: Artigo
Idioma:Inglês
Publicado em: National Academy of Sciences 2009
Assuntos:
Biological Sciences
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2728995/
https://ncbi.nlm.nih.gov/pubmed/19667189
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1073/pnas.0905780106
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