Exon Skipping

Overview

Therapeutic RNA splicing modulation strategy using ASOs or PMOs to force pre-mRNA splicing to skip mutant exons, enabling production of truncated but partially functional protein such as dystrophin in DMD. Exon skipping uses synthetic oligonucleotides (ASOs or PMOs) targeting pre-mRNA splice sites to force the spliceosome to skip a mutated exon, restoring partial reading frame and producing a truncated but partially functional protein — in DMD this generates near-normal truncated dystrophin. Different DMD exon mutations create progressively smaller patient populations beyond exon 51; rare exons affect as few as 10, 3, or 2 patients each, creating fundamental manufacturing and economic challenges for individualized genetic medicines. (Segment 2 (05:00-10:00) / Segment 5 (20:00-25:00))