Genome engineering tools, including targeted gene editing and gene regulation, are becoming available to correct the mutations that cause neuromuscular disorders such as muscular dystrophy, spinal muscular atrophy and myotonic dystrophy. This Review summarizes the genome engineering strategies that are under preclinical evaluation for the treatment of degenerative neuromuscular disorders, focusing on the tools that show the greatest potential for clinical translation.
- Christopher E. Nelson
- Jacqueline N. Robinson-Hamm
- Charles A. Gersbach