Article
The Dimeric Form of 1,3‐Diaminoisoquinoline Derivative Rescued the Mis‐splicing of Atp2a1 and Clcn1 Genes in Myotonic Dystrophy Type 1 Mouse Model
25 May 2020
Abstract excerpt
Expanded CUG repeat RNA in the dystrophia myotonia protein kinase (DMPK) gene causes myotonic dystrophy type 1 (DM1) and sequesters RNA processing proteins, such as the splicing factor muscleblind-like 1 protein (MBNL1). Sequestration of splicing factors results in the mis-splicing of some pre-mRNAs. Small molecules that rescue the mis-splicing in the DM1 cells have drawn attention as potential drugs to treat...
Read the complete abstract on PubMedTopics
Share this publication in a Topic to start or enrich a Post.
