CRISPR Technologies for the Treatment of Duchenne Muscular Dystrophy.

The emerging clustered regularly interspaced short palindromic repeats (CRISPR)-mediated genome editing technologies have progressed remarkably in recent years, opening up the potential of precise genome editing as a therapeutic approach to treat various diseases. The CRISPR/CRISPR associated (Cas) system is an attractive platform for the treatment of Duchenne muscular dystrophy (DMD), which is a neuromuscular disease caused by mutations in the DMD gene. CRISPR/Cas can be used to permanently repair the mutated DMD gene, leading to expression of the encoded protein, dystrophin, in systems ranging from cells derived from DMD patients to animal models of DMD. However, the development of more efficient therapeutic approaches and delivery methods remains a great challenge for DMD. Herein, we review various therapeutic strategies that use CRISPR/Cas to correct or bypass DMD mutations and discuss their therapeutic potential, as well as obstacles that lie ahead.