Spotting Stubborn Indirect Effects in Oligonucleotide Therapeutics
Time: 3:45 pm
day: Day Two
Details:
- 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
