Skip to main content
. 2022 Oct 19;13:1006304. doi: 10.3389/fphar.2022.1006304

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