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Molecular regulation of muscle stem cell function in regenerative myogenesis


Molecular regulation of muscle stem cell function in regenerative myogenesis


Status: Submitted

08 Oct 2023

I am interested in understanding the mechanisms that control stem cell function and applying this knowledge to improve health. Our studies are focused on muscle stem cells and how they function in the growth and repair of skeletal muscle. The research is critical for developing novel treatments for many muscle-wasting diseases, for example Duchenne Muscular Dystrophy (DMD). Our studies have already transformed our understanding of the mechanisms that control muscle stem cell function and have led to the development of a protein called Wnt7a as a novel therapy. First, we will investigate the mechanism by which Wnt7a stimulates muscle regeneration and test therapeutic strategies to treat muscle-wasting disease. The experiments will direct the development of Wnt7a as a drug and clinical trials will be conducted with Wnt7a in the near future. Second, we will define the mechanisms that regulate the division of muscle stem cells as this is a central control point that governs regeneration efficiency. We will investigate these control mechanisms with the longer-term goal of identifying targets and hence drugs that could be used to stimulate muscle stem cell function. Third, we will map the regulatory networks of proteins called transcription factors that control the expression of genes in muscle stem cells from healthy versus diseased tissue. This information is key to understanding how these stem cells function. Finally, we will identify new drugs capable of stimulating muscle stems cells to enhance muscle regeneration. In parallel, we will validate our findings on human muscle stem cells to transition our therapeutic strategies into the clinic. In conclusion, the research will advance our knowledge of stem cell function and lead to novel treatments for diseases involving wasting of skeletal muscle. Importantly, this work will improve the health of patients, and also benefit the families and care givers that are affected by these devastating diseases.

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