Researchers at the University of Rochester Medical Center (URMC) have identified a compound that eliminates myotonia – a symptom of muscular dystrophy – in mice. The study was published today online in the Journal of Clinical Investigation.Rochester scientists were able to design a synthetic RNA-based molecule that, when injected into mice with myotonic dystrophy, restored a critical cellular mechanism, or pathway, that controls electrical activity in muscles. In people with the disease, this function is essentially disabled and muscle cells cannot relax properly. The researchers found that once this pathway was re-established normal muscle function returned.
