The first exon duplication mouse model of Duchenne muscular dystrophy: A tool for therapeutic development
Neuromuscular Disorders(2015)
摘要
•Exon 2 duplication is the most frequent exonic duplication associated with DMD.•We created the 1st mouse carrying an exon duplication developing muscular dystrophy.•The exon 2 duplication results in a disrupted reading frame, and minimal protein expression.•The Dup2 mouse is both pathologically and physiologically similar to mdx.•This new model will be a powerful tool for testing duplication-specific therapies.
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关键词
Duchenne muscular dystrophy,Duplication,Mouse model,Exon skipping
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