Fixing muscular dystrophy using CRISPR gene editing

Genome editing has successfully corrected mutations in Duchenne muscular dystrophy in mice, dogs, and human cells.

• Muscular dystrophies are characterized by progressive weakness and degeneration of skeletal muscle.
• Current approaches have not resulted in curative therapies for these disorders.
• Genome editing technology may allow for the correction of mutations responsible for monogenic neuromuscular diseases.
• CRISPR/Cas9 editing has shown promise in addressing the underlying genetic causes of Duchenne muscular dystrophy.
• The long-lived nature of muscle tissues suggests that a single treatment could potentially provide lasting benefits.

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