TABLE 1.
Drugs or candidates for exon skipping, their targeted exons in the dystrophin gene, and the percentage of DMD patients having mutations in the targeted exons (Aartsma-Rus et al., 2009; Bladen et al., 2015; Vulin et al., 2015). The candidate drug, scAAV9.U7.ACCA, delivers small nuclear RNA (snRNA) using AAV9 for skipping one or two copies of exon 2 in DMD patients with exon 2 duplication.
| Targeted exons | Candidates drugs utilizing exon skipping strategy | % Of DMD patients Having mutation |
|---|---|---|
| 2 | scAAV9.U7.ACCA | <11% (duplication) |
| 44 | BMN 044 | 8% |
| NS-089/NCNP-02 | ||
| 45 | Casimersen | 9% |
| DS-5141b | ||
| BMN 045 | ||
| 51 | SRP-5051 | 13–14% |
| WVE-210201 | ||
| Drisapersen | ||
| Eteplirsen | ||
| 53 | Viltolarsen | 8% |
| BMN 053 | ||
| WVE-N531 | ||
| Golodirsen |