Despite its promise, exon skipping faces several challenges:
Delivery Methods: Efficiently delivering AONs to the target tissues, especially in systemic conditions like DMD, remains a significant hurdle. Long-term Efficacy: As of now, the long-term efficacy and safety of exon skipping treatments are still being studied. Regular monitoring and repeated treatments may be required. Personalization: The technique often needs to be tailored to the specific mutation in each patient, complicating its widespread application.
