Exon Skipping
Genes are often made of sections called exons. When a cell makes RNA, it stitches certain exons together to create the final message used to make a protein. Exon skipping uses a therapy, often an ASO, to encourage the cell to leave out a specific exon. In some diseases, skipping an exon can restore a more usable reading frame or reduce the effect of a mutation, even if the resulting protein is not perfect.
Why it matters
Exon skipping is especially important in some genetic diseases where changing RNA splicing can turn a severe genetic error into a milder or more functional protein message.
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ASO therapy uses short pieces of synthetic genetic material to change how RNA is read, processed, or reduced inside cells.
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Source: GENEration Hope editorial analysis
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