CORDIS Project
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This project focuses on developing a gene therapy strategy for Duchenne muscular dystrophy by using CRISPR/Cas9 technology to repair specific genetic mutations. It aims to restore dystrophin expression by targeting tandem duplications in the DMD gene, potentially offering a new treatment approach for this muscle-wastin…
Duchenne muscular dystrophy is an X-linked recessive muscle-wasting disease, characterized by progressive weakening of skeletal, respiratory, and cardiac muscle followed by necrosis and fibrosis. DMD affects ~1:3,500 live male births and is associated with delayed motor milestones. DMD occurs as a result of mutations within the DMD gene that lead to premature termination of translation.
The most frequent type of mutations are exonic deletions and duplications that induce a frame-shift in the pro…
ASSOCIATION GENETHON
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