Spotting Stubborn Indirect Effects in Oligonucleotide Therapeutics

Time: 3:45 pm
day: Day Two


  • Identification of ASOs complementary to two regions within the first intron of FXN pre-mRNA, which could increase FXN mRNA by 2-fold in patient fibroblasts
  • Identification of two guanosine-rich motifs (CCGG and G4) within the phosphorothioate-based ASOs that were required for FXN activation
  • An atlas of gene activation platform technologies