Technology Insight: therapy for Duchenne muscular dystrophy—an opportunity for personalized medicine?

Duchenne muscular dystrophy is an X-linked recessively inherited disorder of skeletal and cardiac muscle that is caused by mutations in the dystrophin gene. In this article, Lim and Rando describe new mutation-specific therapeutic strategies that use nonviral agents to target nonsense point mutations or frameshift deletions at the dystrophin locus, with the ultimate aim of developing 'personalized' gene therapy for Duchenne muscular dystrophy.