An international team of scientists has developed a novel exon-skipping therapy mixture that has the potential to treat more than 40% of people with Duchenne muscular dystrophy (DMD).
The therapy was described in a study, “Development of DG9 peptide-conjugated single- and multi-exon skipping therapies for the treatment of Duchenne muscular dystrophy,” published in the journal PNAS.
DMD is caused by mutations in the DMD gene, which provides instructions for making dystrophin, a protein that is essential for muscle health. Like other protein-coding genes, the DMD gene contains sections that code for the protein, called exons, which are interspersed with non-coding regions called introns. The DMD gene has 79 exons in total.