The skeletal muscle is the most abundant tissue in our body.
They are key for proper posture, balance, mobility, a capacity to breathe, as well as supporting many other metabolic processes. Many skeletal muscle diseases result from gene errors in the cells of the skeletal muscle, which can worsen with time. These conditions cause the involuntary wasting of the muscles and can be both age-related and inherited. Such muscle disorders have a severe impact on a patient’s quality of life, and in some cases result in disability or premature death.
New gene editing technologies, when applied to stem cells of the muscle, have the potential to correct the genetic errors that cause muscle diseases. reNEW researchers are exploring the pathways that govern muscle degeneration, as well as developing models of human muscle disease. Such human skeletal muscle models enable the study of muscle degeneration and can provide a more accurate way to test the value of new drug treatments. In addition, our researchers are testing the efficacy of new gene editing therapies in human muscle cells for the treatment of muscular dystrophies.