Ontology highlight
ABSTRACT:
SUBMITTER: van Agtmaal EL
PROVIDER: S-EPMC5363205 | biostudies-literature | 2017 Jan
REPOSITORIES: biostudies-literature
van Agtmaal Ellen L EL André Laurène M LM Willemse Marieke M Cumming Sarah A SA van Kessel Ingeborg D G IDG van den Broek Walther J A A WJAA Gourdon Geneviève G Furling Denis D Mouly Vincent V Monckton Darren G DG Wansink Derick G DG Wieringa Bé B
Molecular therapy : the journal of the American Society of Gene Therapy 20170104 1
Myotonic dystrophy type 1 (DM1) is caused by (CTG⋅CAG)<sub>n</sub>-repeat expansion within the DMPK gene and thought to be mediated by a toxic RNA gain of function. Current attempts to develop therapy for this disease mainly aim at destroying or blocking abnormal properties of mutant DMPK (CUG)n RNA. Here, we explored a DNA-directed strategy and demonstrate that single clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9-cleavage in either its 5' or 3' unique flank promotes un ...[more]