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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: | , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Landes Bioscience
2014
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| Assuntos: | |
| Acesso em linha: | 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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