Three teams of researchers have used CRISPR–Cas9 gene editing to treat mice that have the most common and severe form of muscular dystrophy.

Duchenne muscular dystrophy is a fatal disease caused by mutations that disable the gene encoding dystrophin, an important muscle protein. Teams led by Charles Gersbach of Duke University in Durham, North Carolina; Amy Wagers of Harvard University in Cambridge, Massachusetts; and Eric Olson of the University of Texas Southwestern Medical Center in Dallas used the CRISPR–Cas9 gene-editing technique to repair the dystrophin gene in mice that have such mutations.

The three teams used viruses to shuttle the components of the CRISPR–Cas9 system into the muscle cells of infant and adult mice. Treated mice made functional dystrophin and showed improvements in cardiac and skeletal muscle function.

Science http://doi.org/bbpn (2016); Science http://doi.org/bbps (2016); Science http://doi.org/bbpp (2016)