Abstract
Duchenne muscular dystrophy (DMD) is a lethal, X-linked recessive neuromuscular disorder, characterized by progressive muscle degeneration and premature death. DMD is caused by mutations in the DMD gene, leading to a severe reduction or absence of the protein dystrophin. Multiple therapeutic strategies, including gene therapy, aim to restore a functional dystrophin (mini-dystrophin) protein to muscle cells. The ability to accurately quantify dystrophin/mini-dystrophin is essential in determining the level of gene transduction.
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