New Human-Like Mouse Model for Duchenne Muscular Dystrophy and Testing of a Gene Editing Treatment

A novel dystrophic mouse model was developed by deleting exon 45 in the human DMD gene using CRISPR/Cas9.

• Mutations in the DMD gene disrupt the reading frame, leading to Duchenne muscular dystrophy.
• Therapeutic strategies, including exon skipping and CRISPR/Cas9, aim to restore the DMD reading frame.
• The new mouse model allows for testing of these therapeutic strategies in a living organism.
• Application of CRISPR/Cas9 targeting exons 45-55 in the model may restore dystrophin production.

Simplified