- Molecular and cellular physiology of skeletal muscle
- Gene therapy treatment of muscle diseases
California Institute of Technology, 1973
Non-viral Gene Therapy for Treatment of Muscular Dystrophies
The research in our lab at the University of Washington has focused on the molecular and cellular mechanisms that cause Duchenne (DMD) and other muscular dystrophies and myopathies. We use the information gained from this basic research to identify signaling pathways that are dysfunctional in dystrophic muscle and are potential therapeutic targets for treatment of these devastating diseases.
Recently, we developed a non-viral platform for targeted delivery of genes of any size to skeletal and cardiac muscle. This platform could be used to express full-length dystrophin to boys with Duchenne Muscular Dystrophy. This treatment would approximate a cure.
In addition to research conducted in our laboratory at the University of Washington in Seattle, we founded Myosana Therapeutics in 2018 based on the intellectual property related to our novel gene therapy platform.