CRISPR Therapeutics for Duchenne Muscular Dystrophy

Abstract excerpt

Duchenne muscular dystrophy (DMD) is an X-linked recessive neuromuscular disorder with a prevalence of approximately 1 in 3,500-5,000 males. DMD manifests as childhood-onset muscle degeneration, followed by loss of ambulation, cardiomyopathy, and death in early adulthood due to a lack of functional dystrophin protein. Out-of-frame mutations in the dystrophin gene are the most common underlying cause of DMD. Gene e...

Topics

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• CRISPR-Cas Systems
• Disease Management
• Dystrophin
• Exons
• Gene Editing
• Genetic Therapy
• Muscular Dystrophy, Duchenne
• Treatment Outcome

Identifiers and source

Literature Corpus work

1ae881c3-3019-5059-a3ba-600f8a7a562a

DOI

10.20944/preprints202201.0265.v1