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Summary of the CinDel therapeutic approach. DMD gene of a DMD patient has a deletion of exons 51, 52, and 53 compared to the wild-type dystrophin. This produces a reading frame shift when the DNA is translated into a mRNA that results into a stop codon in exon 54 and aborts transcription. When the exons 50 and 54 are cut by the CinDel treatment, a hybrid exon 50/54 is formed and the reading frame is restored, allowing the normal transcription of the mRNA.
