Biomimetic Chemistry of siRNA Delivery to CNS
Time: 3:30 pm
day: Day 1
Details:
- Synthetic small interfering RNAs (siRNAs) are potent inhibitors of gene expression. These molecules are perfect examples of biomimetic chemistry as synthetic siRNAs act through the natural RNA interference (RNAi) pathway
- To deliver therapeutic siRNAs for CNS applications, we have developed approaches that include extensive chemical modification of the siRNAs and lipidic nucleic acids (siRNA-lipid conjugates)
- This presentation will cover the chemical biology of RNA therapeutics including the chemical modifications employed in each RNA strand and novel motifs to ensure uptake into cells, recognition of these modifications and motifs by the slicer enzyme Argonaute-2 and its recognition for potency improvement, silencing efficiency, on-target specificity, safety, and metabolic stability due to nucleolytic degradation
