Year 5

Skeletal muscle makes up 40% of our bodies, dictates our form, is responsible for our ability to move, express ourselves, eat, breathe and to look around. Skeletal muscle injury and disease are major sources of morbidity that affect millions. The potential for treating these ailments with regenerative approaches that use stem cells is enormous because skeletal muscle naturally has its own stem cell population. To make human muscle stem cells a usable clinical tool we have, under this award,  characterized them, transplanted them, and gained insights that will be used by clinicians to induce them to function effectively. To do this, we identified appropriate human muscles to harvest stem cells from, and developed techniques to transplant them. This has enabled us to test them in models of diseases. The work has resulted in new funding from the NIH for continuation of the research, and has resulted in the development of techniques to store and share endogenous human muscle stem cells with other researchers and clinicians.  We plan to continue advancement of this research to develop  treatments for patients with several disabling muscle degenerative conditions.