Myotonic Dystrophy Type 1 (DM1) is a highly disabling multisystemic disease with no cure available. DM1 symptoms affect mainly the nervous, the cardiac, and the musculoskeletal systems, although other alterations in other systems or organs have also been reported. The onset of symptoms occurs during adolescence in its most common form, and affected individuals have a significantly shortened lifespan.
ARTHEx proposes a novel therapeutic approach for the treatment of DM1. We propose to inhibit miRNAs repressing MBNL proteins to preserve MBNL levels. This approach has shown promise in preclinical in vitro and in vivo models of the disease