RNA-targeting Cas9 corrects molecular and physiological features in pre-clinical model of myotonic dystrophy type 1

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 in a mouse model of myotonic Dystrophy (DM1). Furthermore we show that gene expression is not altered with RCas9 treatment in WT mice with or without treatment with immunosuppression We performed paired-end RNA-seq for either non-targeting (NT) and CTG-targeting (RCas9-CTG or PIN ) treated tibialis anterior muscles of myotonic dystrophy (DM1) mice called HSALR. Furthermore, we treated WT tibialis anterior muscle with either sgRNA only (G1), RCas9-CTG (G2) or RCas9-CTG along with immunosuppression with Tacrolimus and CTLA4-Ig. Following this we performed paired-end RNA-Seq