TABLE 3.
Patient customized ASOs approved by US FDA.
| S. No. | Strategy | ASO | Diseases |
|---|---|---|---|
| 1 | Modulation in splicing variants | Milasen | Batten Disease |
| 2 | Modulation in cryptic splicing variants | Atipeksen | Ataxia-telangiectasia mutation |
| 3 | Correction of splice | Jacifusen | ALS with FUS mutation |