Small molecule kinase inhibitors alleviate different molecular features of myotonic dystrophy type 1

Expandable (CTG)n repeats in the 3′ UTR of the DMPK gene are a cause of myotonic dystrophy type 1 (DM1), which leads to a toxic RNA gain-of-function disease. Mutant RNAs with expanded CUG repeats are retained in the nucleus and aggregate in discrete inclusions. These foci sequester splicing factors...

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Main Authors: Wojciechowska, Marzena, Taylor, Katarzyna, Sobczak, Krzysztof, Napierala, Marek, Krzyzosiak, Wlodzimierz J
Format: Artigo
Sprog:Inglês
Udgivet: Landes Bioscience 2014
Fag:
Research Paper
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC4156505/
https://ncbi.nlm.nih.gov/pubmed/24824895
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.4161/rna.28799
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