Microsatellite expansion RNA visualization, elimination, and reversal of molecular pathology by RNA-targeting Cas9

In this Study, we used RNA-targeting Cas9 (RCas9) to reverse characteristic Myotonic Dystrophy (DM1) cellular phenotypes such as elimination of RNA foci, MBNL relocalization, and reversal of transcriptome-wide splicing. Overall design: We performed RNA-seq for control and myotonic dystrophy (DM1) myoblasts and myotubes with various treatments including lentivirus mediated expression of either non-targeting (NT) or CTG-targeting (CTG) single guide RNA (sgRNA) and PIN-dCas9.Lentivirus mediated GFP expression was used as control.