Article
Pluripotent Stem Cells in Disease Modeling and Drug Discovery for Myotonic Dystrophy Type 1
10 Feb 2023
Abstract excerpt
Myotonic dystrophy type 1 (DM1) is a progressive multisystemic disease caused by the expansion of a CTG repeat tract within the 3′ untranslated region (3′ UTR) of the dystrophia myotonica protein kinase gene (DMPK). Although DM1 is considered to be the most frequent myopathy of genetic origin in adults, DM1 patients exhibit a vast diversity of symptoms, affecting many different organs. Up until now, different in...
Topics
Join the communities discussing this publication.