Ontology highlight
ABSTRACT:
SUBMITTER: Zhang W
PROVIDER: S-EPMC3916045 | biostudies-literature | 2014 Feb
REPOSITORIES: biostudies-literature
Molecular therapy : the journal of the American Society of Gene Therapy 20131023 2
Myotonic dystrophy type 1 (DM1) is caused by the expansion of (CTG)n in the 3' untranslated region of the dystrophia myotonica-protein kinase (DMPK) gene, which is transcribed as (CUG)n repeats that accumulate in the nucleus. The RNA repeats specifically sequester or change the expression levels of several RNA-binding proteins, leading to aberrant splicing of many target genes. In this study, we developed artificial site-specific RNA endonucleases (ASREs) that specifically bind and cleave (CUG)n ...[more]