Newly designed AONs induce SMN2 exon 7 inclusion. (a) Relative positions of newly designed AONs targeting ISS-N1 in intron 7 of SMN2. Green line: newly designed AONs (LNA/DNA mixmers). (b) The screen of AONs (LNA/DNA mixmers) by RT-PCR of SMN2 in human SMA patient fibroblasts (GM03813). Transfection of AONs was performed at 200, 100, 50, and 5 nM. Transfection of at least 5 nM of AONs #1–5 induced the exon 7 inclusion. Top band: exon 7-included. Bottom band: exon 7-excluded. M: mock control AON, NT: non-treated, H: healthy cells, B: blank.