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Disease Focus: Duchenne Muscular Dystrophy

Disease Focus

Duchenne Muscular Dystrophy

Duchenne muscular dystrophy is caused by mutations in the DMD gene that encodes one of the largest structural proteins in the body, known as dystrophin. The loss of dystrophin from the cell membrane hyperactivates and upregulates mechanosensitive signaling, resulting in a cascade of secondary insults. By targeting mechanosensitive signaling nodes, our goal is to reduce secondary signaling that worsens the disease.

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