Figure 2

Antisense-mediated exon skipping. Left panel: in this example, a mutation within exon 32 results in a premature stop codon (indicated by the transition of black to white in the pre-mRNA (top) and mRNA (middle), which leads to a prematurely truncated protein (bottom). Right panel: when antisense oligonucleotides (AON) targeting exon 32 are used, they will hybridize to this exon, thus hiding it from the splicing machinery, resulting in the skipping of this exon. As exon 32 in in-frame (its length is divisible by three), skipping will not disrupt the reading frame (the mRNA becomes black in the middle panel) and a full-length protein, which misses a little bit in the middle, can be generated (bottom).