GCB researcher Charlie Gersbach and his team have used CRISPR to treat an adult mouse model of Duchenne muscular dystrophy. Gersbach, Fellow Chris Nelson, and colleagues collaborated with researchers at UNC-Chapel Hill, the University of Missouri School of Medicine, and the Broad Institute/Harvard to package CRISPR/Cas-9 into the virus AAV to deliver the gene editing tools into an adult mouse. The virus cut out the defective sections of the dystrophin gene from cells throughtout the mouse's body, enabling the gene to produce functional proteins. With cells beginning to make functional dystrophin proteins, which are important to muscle structure, the muscle strength of the mice increased. The success of this animal model is a promising early step on the path to human therapy.
Read the article in Science, and media coverage of this work in the New York Times, BBC News, the Guardian, Duke University, and Duke's Pratt School of Engineering.