Year 3

In the third year of the New Faculty Physician Scientist award, my colleagues and I have made progress on all 3 Aims. The majority of our focus in accordance with our planned timeline has been on Aims 2 and 3, and we published our first manuscript based on Aim 1 results. Aim 1 focused on identification of appropriate donor muscles for human muscle stem cell applications and characterization of endogenous human muscle stem cells. As human muscle stem cells had not been definitively characterized or transplanted, the initial main focus of this CIRM grant was to do so with stem cells from diverse muscles and, combined with our clinical understanding of expendability of different muscles, choose human muscles that will be appropriate to harvest stem cells from in future applications. We have accomplished that and published the essential results in September 2015. For the experiments in Aim 2, we are testing injury and transplantation methods for human muscle stem cells that could potentially be used clinically and have made advances that will allow us to effectively test preclinical therapeutic approaches that are the subject of Aim 3. In the past reporting period work on Aim 3, has focused on developing preclinical models in which to test human muscle stem cell transplantation. We are characterizing these models in anticipation of testing human muscle stem cells in them in the next reporting period.