This chapter provides an example of a significant discovery that resulted from what researchers initially perceived as a negative clinical finding, which they then explored further in the laboratory. The main stumbling block impeding many therapeutic approaches, including the treatment of Duchenne muscular dystrophy (DMD), is the difficulty associated with delivering therapeutic agents at levels sufficient to provide benefit. Stem cells offer many exciting possibilities for the development of novel treatments to alleviate numerous devastating diseases. In particular, muscle-derived stem cells (MDSC)-based therapy holds vast therapeutic potential due to MDSCs' survival ability, unique immuneprivileged behavior, long-term proliferative potential, and pluripotency. Such characteristics strongly indicate that these cells could be useful for the treatment of DMD and other conditions of the musculoskeletal system. These cells provide a good example of what can happen when clinicians and researchers work hand-in-hand to appropriately interpret the outcome of clinical applications. Much work remains to be done before applications based on these cells progress from bench back to the bedside, but the preliminary data reviewed here strongly suggest that the future application of these techniques is promising.
ASJC Scopus subject areas
- Biochemistry, Genetics and Molecular Biology(all)