Muscular Dystrophy Therapy Using Viral Vector-based CRISPR/Cas

Duchenne muscular dystrophy (DMD) is a fatal X-linked genetic disorder caused by mutations in the DMD gene, which encodes the dystrophin protein. A lack of dystrophin disrupts the skeletal musculature, resulting in severe muscle degeneration. Currently, the clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated (Cas) system offers an evolved and precise tool of programmed genomic modification and is being widely studied as a therapeutic tool for treating various genetic diseases. Recently, CRISPR-mediated DMD therapy has been intensively studied as a means of correcting or bypassing disease-causing mutations, resulting in the permanent repair of mutated DMD gene and rescues of dystrophin expression. However, delivery methods remain a major barrier for CRISPR-mediated genome editing. Various viral vectors have been utilized as vehicles for sequences encoding CRISPR/Cas components. Efforts have been made to optimize the viral vector systems for efficient delivery of these components to treat DMD. Herein, we review diverse aspects of several viral vectors combined with CRISPR/Cas systems for DMD therapy and discuss their therapeutic potential and the challenges ahead.