Department: Medical Biophysics, Western University
Duchenne muscular dystrophy (DMD) is a severe neuromuscular disorder that results from mutations in or the loss of the cytoskeletal protein, dystrophin, and is characterized by progressive degeneration of both skeletal and cardiac muscle. This disorder affects ~30,000 children (primarily boys) in North America alone, making it the most common fatal genetic disorder diagnosed in early childhood. There is no cure, with most children succumbing to the disease in their early twenties; more than 95% of these patients exhibit clinically-relevant cardiomyopathy. Given the prevalence and severity of DMD, there exists an urgent need for new and innovative ways to improve our understanding of the disease, and to advance therapies and preventive strategies to assess disease risk, delay disease progression, and aid in repair. Molecular imaging has potential to fulfill this need. While recent advances suggest that transplantation of stem cells may be able to repair damaged heart and skeletal muscle, there exists a major limitation to the use of stem cells: a lack of reliable imaging technologies for long-term monitoring of implanted stem cells, and for evaluating its effectiveness as a therapy. Another significant limitation to the use of stem/progenitor cells for the treatment of DMD, however, is the restricted ability of transplanted cells to migrate and engraft within damaged muscle. It has been suggested that this may arise due to deprivation of an appropriate microvascular environment. Therefore, my group also aims to identify growth factors /cytokines, etc. that enhance cell migration and engraftment.