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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Detalles Bibliográficos
Autores principales: Wojciechowska, Marzena, Taylor, Katarzyna, Sobczak, Krzysztof, Napierala, Marek, Krzyzosiak, Wlodzimierz J
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Landes Bioscience 2014
Materias:
Research Paper
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4156505/
https://www.ncbi.nlm.nih.gov/pubmed/24824895
http://dx.doi.org/10.4161/rna.28799

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