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Antisense-induced exon skipping in DMD. Deletion of exon 49 and 50 in the DMD gene creates a frameshift mutation which results in a premature stop codon and no dystrophin produced. In the case shown, AOs can specifically bind to exon 51 and interfere with the splicing machinery to exclude exon 51 from the mature mRNA, thereby restoring the reading frame and producing truncated but partly functional dystrophin.
