One approach to improving muscle strength in Duchenne is to replace unhealthy muscle cells that lack dystrophin with donor stem cells that have normal levels of dystrophin. These cells might come from a relative unaffected by Duchenne or even a person with Duchenne’s own cell, after dystrophin is restored though a technique like gene therapy.
Although we’ve heard much more in recent years about drug approaches to therapy, work continues to better understand how to use stem cells to restore muscle tissue. In a new article from Purdue, Dr. Shihuan Kuang describes how stem cell therapy would work for Duchenne and his discovery that mimicking the environment in the muscle is the best way to grow healthy muscle stem cells in culture dishes.
Sharon Hesterlee, Ph.D.
Read more PPMD Staff Blogs