How can exon skipping help?
As the name suggests, the principle of exon skipping is to encourage the cellular machinery to ‘skip over’ an exon. Small pieces of DNA called antisense oligonucleotides (AOs) or ‘molecular patches’ are used to mask the exon that you want to skip, so that it is ignored during protein production. In our example, if we use a ‘molecular patch’ designed to mask exon 51: Exon 49 can now join up to exon 52 and continue to make the rest of the protein, with exons 50 and 51 missing in the middle: Therefore, exon skipping may be able to reduce the symptoms of Duchenne muscular dystrophy, to those more like Becker muscular dystrophy.
