Short antisense-locked nucleic acids (all-LNAs) correct alternative splicing abnormalities in myotonic dystrophy

Myotonic dystrophy type 1 (DM1) is an autosomal dominant multisystemic disorder caused by expansion of CTG triplet repeats in 3′-untranslated region of DMPK gene. The pathomechanism of DM1 is driven by accumulation of toxic transcripts containing expanded CUG repeats (CUG(exp)) in nuclear foci which...

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Detalles Bibliográficos
Autores principales: Wojtkowiak-Szlachcic, Agnieszka, Taylor, Katarzyna, Stepniak-Konieczna, Ewa, Sznajder, Lukasz J., Mykowska, Agnieszka, Sroka, Joanna, Thornton, Charles A., Sobczak, Krzysztof
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2015
Materias:
RNA
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4381072/
https://www.ncbi.nlm.nih.gov/pubmed/25753670
http://dx.doi.org/10.1093/nar/gkv163

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4381072/
https://www.ncbi.nlm.nih.gov/pubmed/25753670
http://dx.doi.org/10.1093/nar/gkv163

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