Therapeutic Agents for Treatment of Myotonic Dystrophy


Dr. Zimmerman's lab has developed a series of bioactive compounds for the treatment of myotonic dystrophy. The compounds disrupt the MBNL-(CUG) foci in vivo and partially rescue the missplicing of cTNT and IR mRNAs. These novel compounds, utilizing bisamidinium-based inhibitors, show a 1000-fold greater inhibition potency than a previous compound and are able to degrade the causative agent of the disease.