Repairing DMD Genes in Mice

A team of researchers, led by HSCI Executive Committee member Amy Wagers, was able to restore the production of functional dystrophin protein in mouse models of Duchenne muscular dystrophy. Using CRISPR-Cas9 to excise faulty exons, the team was able to restore the Dmd reading frame in myofibers, cardiomyocytes, and muscle stem cells. Read the New York Times article or the Science journal article