A Novel Mouse Model of Duchenne Muscular Dystrophy with a Duplication of DMD Exon 2

A novel mouse model for testing exon skipping therapies for DMD disease has been generated at Nationwide Children’s Hospital. This mouse model carries a duplicated exon (exon2) in the DMD gene as compared to a point mutation in the most common mdx mouse model. This unique dystrophic mouse can serve as a preclinical testing model to test various therapies that mediate exon skipping.

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