Principal Investigator: Catherine Verfaillie, M.D.
Muscular dystrophies are a set of diseases that are genetically determined and result in abnormal function due to structural damage to the muscle tissue. The prototypical disease is Duchenne muscular dystrophy (DMD). This very sad, relentless disorder affects approximately 1/3500 live male births. Unfortunately, there is still no cure and no effective treatment to delay the progression of DMD. Recent research has focused on viral and non-viral mediated gene correction, and cellular therapy.
In the Stem Cell Institute at the University of Minnesota-Twin Cities, we have been investigating a specific population of multipotent adult stem cells (MASC) named Multipotent Adult Progenitor Cells (MAPC). MAPC have exhibited the ability to differentiate into tissue of all three germ layers. More specifically, we have shown that MAPC can be differentiated in vitro, into cells that have a similar phenotype to skeletal muscle. In addition, we have shown that in vivo, MAPC can fuse with muscle and differentiate into cells with a skeletal muscle phenotype. Research from our lab and other labs is promising, however, engraftment of adult stem cells is too low to be of clinical value. Our goal is to develop ways to improve engraftment.