A skip in time: Frame-restoring exon skipping counters premature aging in Werner syndrome

Antisense oligonucleotide (ASO) technology is a blooming class of therapeutics quickly reaching the fame of gene therapy, offering potential for treating diseases by targeting RNA.1 Worthy of particular interest is the ability of ASOs to modulate splicing, thereby mediating exon skipping. The “skipping to correct a skip” strategy offers the advantage of producing a shorter but still functional protein; this is a clean-cut strategy able to overcome some of those mutations which compromise protein translation and/or functionality.