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...

Descripción completa

Detalles Bibliográficos
Autores principales: Stepniak-Konieczna, Ewa, Konieczny, Patryk, Cywoniuk, Piotr, Dluzewska, Julia, Sobczak, Krzysztof
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Oxford University Press 2020
Materias:
Molecular Biology
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7049696/
https://www.ncbi.nlm.nih.gov/pubmed/31965181
http://dx.doi.org/10.1093/nar/gkaa007

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7049696/
https://www.ncbi.nlm.nih.gov/pubmed/31965181
http://dx.doi.org/10.1093/nar/gkaa007

Ejemplares similares