Revealing Structurally Constrained Internucleotide (E)-Vinyl Phosphonate Backbone Impacts siRNA Potency & Allele Specificity

Time: 12:00 pm
day: Pre-Conference Discussion Day

Details:

  • Adding structure-constraining of sugars, such as 2′-fluoro and LNA, has been shown to enhance oligonucleotide stability and affinity to target mRNA
  • Exploring the synthesis and impact of a structurally-constrained inter-nucleotide (E)-vinylphosphonate backbone (iE-VP) where we show iE-VP can modulate metabolic stability, enzyme interactions and siRNA activity
  • Demonstrating how iE-VP induces significant mismatch-discriminating properties without compromising on-target silencing
  • Explaining how our study covers a broad range of interests and paves the path towards fully allele-specific siRNAs for CNS disorders such as Huntington’s disease

Speakers: