fig7

Figure 7. Therapeutic exon skipping. An ASO targeting and blocking an intronic splice acceptor or donor site or an exonic splice enhancer site enforces exon skipping. In the case of the picture, the ASO targets and binds to the acceptor site of intron N+1, resulting in the skipping of ExonN+2. The exons N+1 and N+3 must be in frame to ensure the expression of a protein devoid of only exon N+2, which might still be fully or partially functional.