Exon skipping offers several benefits, particularly in pediatric patients:
Reduced Severity: By allowing the production of a partially functional protein, the severity of the genetic disorder can be significantly reduced. Targeted Therapy: This technique is highly specific, targeting only the faulty exon and minimizing off-target effects. Potential for Early Intervention: Since many genetic disorders manifest early in life, exon skipping can be used for early intervention, potentially improving long-term outcomes.
