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Lead investigator

Collaborators & Co-Investigators

• Craig Campbell, MD
• Jeffrey Chamberlain, MD, PhD
• Derrick Rancourt, PhD

Research Sites & Affiliations

• London Health Sciences Centre Research Inc. (Lawson Health Research Institute), London, Ontario

Budget: $99,038

Disorders: Duchenne/Becker Muscular Dystrophy

Research Areas: Discover Novel Treatments & Therapies

Abstract: 

Duchenne muscular dystrophy (DMD) affects about 30,000 boys in North America, making it the most common family-inherited disease that begins during early childhood. In people with DMD, their bodies cannot correctly make the protein dystrophin. Because of this, their muscles are easily damaged and break down. Scar tissue forms, making it harder for muscles to work. Most patients die in their mid-twenties. Currently, DMD has no cure. Most DMD research is based on replacing dystrophin to build back healthy muscles. However, when boys are diagnosed with DMD around 3-5 years old, their muscles struggle to get enough oxygen and nutrients because of their poor blood supply. This significantly impairs muscle repair and makes efforts to replace dystrophin more difficult. Few studies have focused on improving blood flow to the affected muscles. Excitingly, there is now evidence of how blood flow is changed. This study suggests blood vessels are leaky, and the protein Angiopoietin -1 (Ang-1), which normally closes blood vessels, is lower in DMD-affected muscle than usual. This finding has led to the primary goal of this project: to determine if Ang-1 can close blood vessels and improve the chances of repairing muscle in lab-grown muscle by replacing dystrophin.

Impact: