AON-induced splice-switching and DMPK pre-mRNA degradation as potential therapeutic approaches for Myotonic Dystrophy type 1

Expansion of an unstable CTG repeat in the 3′UTR of the DMPK gene causes Myotonic Dystrophy type 1 (DM1). CUG-expanded DMPK transcripts (CUG(exp)) sequester Muscleblind-like (MBNL) alternative splicing regulators in ribonuclear inclusions (foci), leading to abnormalities in RNA processing and splici...

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Detaylı Bibliyografya
Yayımlandı:Nucleic Acids Res
Asıl Yazarlar: Stepniak-Konieczna, Ewa, Konieczny, Patryk, Cywoniuk, Piotr, Dluzewska, Julia, Sobczak, Krzysztof
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: Oxford University Press 2020
Konular:
Molecular Biology
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC7049696/
https://ncbi.nlm.nih.gov/pubmed/31965181
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkaa007
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