Our overall objective is to understand the mechanisms of pathogenesis in DM1 with a particular focus on muscle atrophy phenotypes and discover therapeutic targets and innovative treatments for patients. We use human cells, animal models, and bioinformatics to understand the contribution of miRNome alterations to the disease genesis, perform drug screenings, and evaluate the activity of Muscleblind enhancer drugs and therapeutics against novel targets. We are actively working in RNA-based drug development to achieve this, with our studies now in a preclinical state.
Our drug screening platforms offer a rapid, effective, and economical system to validate chemical compounds for their utility as drugs in the studied pathological conditions.
Our long term aspiration is the application of our research findings and the transformation of biomedical knowledge into products or services for the society. For that purpose, we are pursuing collaboration with spin-offs and start-ups.