Year 6 NCE

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. However, to make human muscle stem cells a usable clinical tool we needed to be able to characterize them, transplant them, and induce them to function effectively. To accomplish these goals, our objectives of this project were to are to identify appropriate human muscles to harvest stem cells from, to develop techniques to transplant them, and to test them in models of diseases. The outcomes of the work resulted in characterization of muscle stem cells in various different human muscles, and the development of effective techniques to transplant the cells into muscles. Approaches were also developed to store and distribute these cells among researchers and eventually to patients. The effect of transplanting human muscle stem cells in animal models of several injuries and diseases was tested, providing insight into how muscle stem cells may be used to contribute to treatment of muscle diseases. The work has been published and additional publications are forthcoming.  Invention disclosures have been filed. The career goals for this New Faculty award included development of a robust, innovative muscle stem cell research program for the PI that would create a sustainable combination of clinical and research activity involving ongoing translation from bench to bedside and from bedside to bench, and to yield pathways to new funding to launch additional projects that would develop from this one. These goals were also achieved. The PI has developed an independent federally funded research program in muscle stem cell and regeneration research. The research program of the PI can this continue with the gaol of furthering translational research aiming to treat muscle diseases and injuries.