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The Role of CRISPR and AAV in Duchenne Gene Repair
Duchenne Muscular Dystrophy (DMD) is a rare, progressive neuromuscular disorder that leads to severe muscle degeneration and early mortality. Affecting roughly 1 in 3,500 to 5,000 male births globally, DMD stems from mutations in the DMD gene, which encodes dystrophin, a protein critical for muscle fiber integrity. Without dystrophin, muscle cells become fragile and eventually die, leading to...
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