Gene Editing to Correct Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a progressive muscle wasting genetic disease which leads to degeneration of cardiac and skeletal muscles. Only boys are affected with DMD while girls can be asymptomatic carriers. The disease is caused by over 3000 different mutations in the dystrophin gene. Researchers from the U.S. and Germany have used the gene editing tool CRISPR-Cas9 in a novel way to produce healthy heart muscle in Duchenne muscular dystrophy patients.