Personalized Gene and Cell Therapy for Duchenne Muscular Dystrophy


Duchenne muscular dystrophy (DMD) is one of the most common childhood neuromuscular diseases (1: 3500 male births [1]). It was named after the French neurologist Guillaume Duchenne, who was the first to describe the symptoms in 1861. The disease DMD is caused by mutations in the X-linked DMD gene that is one of the largest genes in the human genome (79 exons coding for a 14-kb transcript), producing the giant dystrophin protein (427 kDa) (Figure 1). This sub-sarcolemmal muscle protein is associated with the dystrophin glycoprotein complex (DGC) [2].


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