What is the level of dystrophin expression required for effective therapy of Duchenne muscular dystrophy?
(2019)
Journal Article
Wells, D. J. (2019). What is the level of dystrophin expression required for effective therapy of Duchenne muscular dystrophy?. Journal of Muscle Research and Cell Motility, 40(2), 141-150. https://doi.org/10.1007/s10974-019-09535-9
Duchenne muscular dystrophy (DMD) is a fatal X-linked muscle wasting disease. The disease is due to mutations in the DMD gene that encodes for a large intracellular protein called dystrophin. Dystrophin plays a critical role in linking the internal c... Read More about What is the level of dystrophin expression required for effective therapy of Duchenne muscular dystrophy?.