Enhancement of exon skipping activity by reduction in the secondary structure content of LNA-based splice-switching oligonucleotides
2019
Shimo, Takenori | Tachibana, Keisuke | Kawawaki, Yuki | Watahiki, Yuuka | Ishigaki, Taku | Nakatsuji, Yusuke | Hara, Takashi | Kawakami, Junji | Obika, Satoshi
PAGE and UV melting analysis revealed that longer LNA-based splice-switching oligonucleotides (SSOs) formed secondary structures by themselves, reducing their effective concentration. To avoid such secondary structure formation, we introduced 7-deaza-2′-deoxyguanosine or 2′-deoxyinosine into the SSOs. These modified SSOs, with fewer secondary structures, showed higher exon skipping activities.
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