CRISPR-Generated Animal Models of Duchenne Muscular Dystrophy

Abstract excerpt

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive neuromuscular disorder most commonly caused by mutations disrupting the reading frame of the dystrophin (DMD) gene. DMD codes for dystrophin, which is critical for maintaining the integrity of muscle cell membranes. Without dystrophin, muscle cells receive heightened mechanical stress, becoming more susceptible to damage. An active body of research co...

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

3d1e96c8-70e8-5924-abc8-aa4d6ebb4df0

DOI

10.20944/preprints202003.0048.v1