TS-000199 — Facioscapulohumeral Muscular Dystrophy (FSHD) is the third most common muscular dystrophy, affecting 1 in 20,000 individuals. There is no current treatment for FSHD; therefore, animal models of the disease are essential for testing potential therapies. Researchers at Nationwide Children’s Hospital have developed a mouse model that recapitulates the FSHD phenotype and develops myopathy. This is an inducible FSHD mouse model that stably expresses the disease-causing gene, DUX4, from the mouse genome using the human DUX4 promoter. Importantly, in comparison to other FSHD mouse models, this particular inducible model circumvents lethality and leakiness problems seen in past models of the disease. Available for purchase through Jax Labs jax.org Stock No: 032779 Potential Applications/Markets: The FSHD field is lacking a good mouse model that recapitulates FSHD phenotypes and develops myopathy. Opportunity/Seeking: Licensing

TS-000178 — 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.