Ontology highlight
ABSTRACT:
SUBMITTER: Wojtkowiak-Szlachcic A
PROVIDER: S-EPMC4381072 | biostudies-literature | 2015 Mar
REPOSITORIES: biostudies-literature
Wojtkowiak-Szlachcic Agnieszka A Taylor Katarzyna K Stepniak-Konieczna Ewa E Sznajder Lukasz J LJ Mykowska Agnieszka A Sroka Joanna J Thornton Charles A CA Sobczak Krzysztof K
Nucleic acids research 20150309 6
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 sequester several factors regulating RNA metabolism, such as Muscleblind-like proteins (MBNLs). In this work, we utilized very short chemically modified antisense oligonucleotides composed exclusivel ...[more]