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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Detalhes bibliográficos
Publicado no:Nucleic Acids Res
Main Authors: Wojtkowiak-Szlachcic, Agnieszka, Taylor, Katarzyna, Stepniak-Konieczna, Ewa, Sznajder, Lukasz J., Mykowska, Agnieszka, Sroka, Joanna, Thornton, Charles A., Sobczak, Krzysztof
Formato: Artigo
Idioma:Inglês
Publicado em: Oxford University Press 2015
Assuntos:
RNA
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC4381072/
https://ncbi.nlm.nih.gov/pubmed/25753670
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkv163
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