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Lead investigator

Research Sites & Affiliations

• University of Ottawa, Ottawa, ON

Budget: $40,000

Disorders: Duchenne/Becker Muscular Dystrophy

Research Areas: Understand Diagnosis and Disorder Progression ,   Discover Novel Treatments & Therapies

Abstract: 

Grant Summary: Roughly 4 million Canadians are affected by muscular dystrophies such as Duchene Muscular Dystrophy (DMD). This disorder leads to a decline in mobility and voluntary activity. Ultimately, this results in high rates of mortality. Muscle wasting is commonly thought to be only a problem of the muscle itself. Yet, muscle stem cell (MuSC) dysfunction also plays an important role. Normally these specialized MuSC allow muscle to heal and repair itself. In patients with DMD, these cells do not work properly leading to muscle breakdown. The researchers’ recent work has shown that mitochondria are required for proper MuSC function. Unfortunately, mitochondria in MuSCs do not act well in DMD. The goal of this study is to assess whether mitochondria in MuSCs can act as a therapeutic target to treat DMD. Findings from this work will allow the researchers to target MuSCs as an intervention for DMD and other neuromuscular disorders.

Impact: