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Exon skipping is another technique to boost functioning protein production, by masking the faulty exon harboring a frameshift mutation, using small pieces of DNA (antisense oligonucleotides). Exon-skipping results in the loss of an exon in the alternatively spliced mRNA. In this mode, the middle exon in three consecutive exons may be included in mature mRNA under some conditions or in particular tissues, but may be excluded from the mature mRNA in others.

The goal of exon skipping is to change the splicing pattern so that an out-of-frame, DMD-type mutation becomes an in-frame, BMD-type mutation. For example, skipping exon 51 of the dystrophin gene could restore the “reading frame” in patients who have specific out-of-frame deletions in some dystrophin exons.

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