The therapeutic potential of antisense-mediated exon skipping.

  title={The therapeutic potential of antisense-mediated exon skipping.},
  author={Gert-Jan van Ommen and Judith C. T. van Deutekom and Annemieke Aartsma-Rus},
  journal={Current opinion in molecular therapeutics},
  volume={10 2},
Antisense-mediated exon skipping is an approach that uses antisense oligonucleotides (AONs) to modulate splicing by hiding specific sites essential for exon inclusion from the splicing machinery. AONs that block aberrant splice sites can restore normal splicing, whereas AONs targeting alternative splice sites can switch splicing patterns from detrimental to beneficial isoforms or produce non-functional mRNAs that lead to gene knockdown. Furthermore, AONs have also been used to restore a… CONTINUE READING
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