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

详细技术说明
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.
*Abstract

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.

*Inquiry
Margaret Barkett, PhD The Research Institute at Nationwide Children's Hospital Office of Technology Commercialization 700 Children's Drive Columbus OH 43205 T: 614 355-2957 F: 614 722-2716 Margaret.barkett@nationwidechildrens.org
*IP Issue Date
None
*IP Type
Other Patent
国家
Not Available
申请号码
None
国家/地区
美国

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