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