Ligand for Potential Treatment of Myotonic Dystrophy Type 1 (DM1)

Dr. Zimmerman from the University of Illinois at Urbana-Champaign has developed an oligomeric ligand that can have potential applications in the treatment of DM1. This Ligand on account of its multivalent nature, is an improvement over the previous small molecule (TF12039) designed by the inventor. The oligomeric ligand has been shown to rescue mis-splicing activity in DM1 cell models. Climbing defects in adult DM1 drosophila flies was also show to have been rescued on treatment with this oligomeric ligand.