Muscles are vital for everyday human life. Skeletal muscle contractions enable us to move, stop, and stay upright. Without these complex fiber-like cells, voluntary functions, including swallowing and speaking, would be impossible. At the same, our ability to pump blood, a life-sustaining involuntary function, is only possible because of the cardiac muscle that enables our hearts to contract.
Impairments to skeletal muscle or cardiac muscle can lead to problems with specific organs or entire systems. The impact is often devastating. Muscle disorders, like Duchenne muscular dystrophy, X-linked myotubular myopathy, and ALS, while rare, can severely limit mobility and, in some cases, reduce life expectancies dramatically. While new therapies offer hope, there are no cures for these diseases.
At the Institute for Stem Cell and Regenerative Medicine, researchers in multiple labs are using stem cells, gene therapy, and cutting-edge devices and powerful analytical technology to study and address the root causes of muscle diseases, creating a pathway that is already moving treatments into clinical trials. Here is a summary of recent progress in muscle research at ISCRM.
The promising clinical trials for Duchenne muscular dystrophy and myotubular myopathy show the translational value of muscle research at ISCRM. To build an even more robust pipeline to patient care, ISCRM researchers are collaborating with clinicians on the front lines of medicine, including Nassim Rad, MD, who practices at the UW Medical Center Rehabilitation Medicine Clinic and Mike Bamshad, MD, chief of the Division of Genetic Medicine in the Department of Pediatrics at the UW School of Medicine.