About the Project
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.
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