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Stabilization of Expanded (CTG)•(CAG) Repeats by Antisense Oligonucleotides
Myotonic dystrophy type 1 (DM1) is caused by expansion of a CTG repeat in the gene DMPK. The expansion is highly unstable in somatic cells, a feature that may contribute to disease progression. The RNA expressed from the mutant allele exerts a toxic gain of function, due to the presence of an expand...
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| Format: | Artigo |
| Sprache: | Inglês |
| Veröffentlicht: |
Nature Publishing Group
2011
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| Schlagworte: | |
| Online Zugang: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3242663/ https://ncbi.nlm.nih.gov/pubmed/21971425 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2011.191 |
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