Muscular dystrophy cured with CRISPR gene therapy which replaced dysfunctional genes in all muscles in adult mice.

Muscular dystrophy was cured in adult mice using a new gene-editing technique called CRISPR/AAV which replaced the dysfunctional dystrophin gene a with functional one in all muscle cells and increased muscle strength. The breakthrough here is that the CRISPR/AAV was able to reach all muscles, including in the heart, after being injected into an adult mouse’s bloodstream. Duchenne muscular dystrophy affects 1 in 5,000 males and most are wheelchair-bound by age 10 and don’t live beyond their 20s or early 30s.