Enhancement of exon skipping activity by reduction in the secondary structure content of LNA-based splice-switching oligonucleotides

Chem Commun (Camb). 2019 Jun 11;55(48):6850-6853. doi: 10.1039/c8cc09648a.

Abstract

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.

MeSH terms

  • Deoxyguanosine / analogs & derivatives
  • Deoxyguanosine / chemistry
  • Exons*
  • Inosine / analogs & derivatives
  • Inosine / chemistry
  • Nucleic Acid Conformation
  • Oligonucleotides / chemistry*
  • Oxidation-Reduction

Substances

  • Oligonucleotides
  • locked nucleic acid
  • Inosine
  • 7-deaza-2'-deoxyguanosine
  • Deoxyguanosine
  • deoxyinosine