Four new mouse models of Duchenne muscular dystrophy with important human gene deletions

Four novel humanized DMD mouse models were developed with deletions of specific exons in the human DMD gene.

• The mouse models exhibit either a complete lack of dystrophin or trace levels of dystrophin.
• Muscle pathology in young adult mice includes muscle fiber degeneration, regeneration, inflammation, and fibrosis.
• Intramuscular treatment with vivo-morpholinos successfully induced exon skipping in the DMD strains.
• Exon skipping restored the disrupted reading frame and led to increased dystrophin expression.
• These models may serve as valuable tools for testing human-specific therapies for Duchenne muscular dystrophy.

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