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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Autores principales: Nakamori, Masayuki, Gourdon, Geneviève, Thornton, Charles A
Formato: Artigo
Lenguaje:Inglês
Publicado: Nature Publishing Group 2011
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Original Article
Acceso en línea: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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