---

Lead investigator

Collaborators & Co-Investigators

• Élise Duchesne, PhD
• Christian Beausejour, PhD

Research Sites & Affiliations

• Universite du Quebec a Chicoutimi, Chicoutimi, Quebec
• CHU Sainte-Justine, Montreal, Quebec

Budget: $60,000

Disorders: Myotonic Dystrophy

Research Areas: Discover Novel Treatments & Therapies

Abstract: 

Myotonic dystrophy type 1 is one of the most frequent genetic muscle diseases in humans. The disease is characterized by muscle weakness and atrophy. Moreover, the regenerative capacity of muscle stem cells, the engine of muscle repair, is reduced in the disease. Therefore, there is a high therapeutic potential for strategies targeting muscle stem cells in myotonic dystrophy type 1; however, this avenue remains unexplored. Our goal is to investigate a new therapeutic strategy aiming to target defective muscle stem cells and restore their regenerative potential. To do so, we will use a preclinical animal model of myotonic dystrophy type 1 to validate the efficacy of these novel therapeutic molecules on muscle regeneration and physical function. Improving muscle regeneration could help to mitigate disease progression and improve the quality of life of the patients. Overall, this project will explore a novel therapeutic avenue for patients that currently have limited therapeutic options.

Impact: