Protein Kinase C inhibitor Molecule Effect Partial Reversal of Spliceopathy in Myotonic Dystrophy Type 2 Cell Line

Abstract

Myotonic dystrophy is a neuromuscular disease which manifest as two forms namely type 1 (DM1) and type 2 (DM2). One of the major molecular events associated with the disease condition is misregulation of splicing resulting in spliceopathy. This study was performed to assess the effect of protein kinase C (PKC) inhibitors- Ro 31-8220 and hypericin- on the splicing of insulin receptor (IR) and muscleblind-like (MBNL1) in myotonic dystrophy type 2 (DM2) cell line. The results of this study demonstrated that only Ro 31-8820 was able to effect partial rescue of missplicing of insulin receptor and muscleblind-like 1. It indicates that it could be a possible therapeutic agent for treatment of DM2.

Keywords: Myotonic dystrophy, Protein Kinase C inhibitor, spliceopathy, therapeutics

DOI: 10.7176/JNSR/11-14-03

Publication date:July 31^st 2020