Skeletal muscle development is a complex process and a number of regulatory factors for the different steps involved in stem cell activation and proliferation through to multinucleated, post-mitotic cells have been identified. Skeletal muscle size is dynamic and responsive to extracellular signals such as mechanical load, neural activity, hormones, growth factors, and cytokines. The loss of skeletal muscle tissue or function has a major impact on public health; a variety of conditions result in the loss of muscle, including disease-related loss (cachexia), age-associated loss (sarcopenia), enforced inactivity, such as bed rest, and muscular dystrophies.
The program will seek to develop novel medicines using strategies based on evaluating either novel pharmaceutical, antisense reagents (exon skipping), viral technologies (adeno-associated virus and lentivirus) or immunotherapies to either re-introduce normal copies of gene that are mutated in human health, alter gene expression profiles to enhance the environmental milieu of muscle or to improve oxidative stress that will positively impact muscle structure or function. The developed medicines will be potential therapeutic strategies for muscular dystrophies, age-related or disease related muscle loss or metabolic disease.