Article
A new dystrophin-deficient rat model mirroring exon skipping in patients with DMD exon 45 deletions.
Disease models & mechanisms - 1 Jan 2026
Wang Tao, Daoud Cynthia, Dubois Auriane, Corre Guillaume, Bellec Jessica, Bovolenta Matteo, Philidet Louise, Dorval Alan, Bourg Nathalie, Roudaut Carinne, Albini Sonia, Warthi Ganesh, Jaber Abbass, Richard Isabelle
Abstract excerpt
Pathogenic variants in the dystrophin (DMD) gene cause muscle-wasting disorders ranging from the milder Becker muscular dystrophy (BMD) to the more severe Duchenne muscular dystrophy (DMD). Exon 45 deletion is the most-frequent single-exon deletion in patients diagnosed with DMD. Here, we generated a novel rat model with an exon 45 deletion using CRISPR/Cas9. The DmdΔ45 rat recapitulate key features of DMD,...
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