Figure 7. Kinase inhibitors as potential therapeutics for DM1. DM1 molecular phenotype is characterized by formation of nuclear multi-protein CUGexp RNA foci, CUGBP1 protein hyperphosphorylation, and upregulation, as well as aberrant alternative splicing of several pre-mRNAs. These features result, among others, from unspecific activation of various protein kinases in the presence of mutant DMPK transcripts. Utilization of small-molecule kinase inhibitors, C16 and C51, causes significant improvements of DM1 phenotype and their mechanism of action do not involve targeting of mutant CUG repeat transcripts.