Designing Antisense Oligonucleotides for High Potency and Minimal Off-Target Effects

Discover the intricacies of designing antisense oligonucleotides (ASOs) for optimal on-target potency and minimal off-target effects in this 50-minute video presentation by Integrated DNA Technologies (IDT). Explore different types of ASOs, including RNase H1 gapmer ASOs and steric blocking ASOs, and their mechanisms of action in modulating gene expression. Gain valuable insights into the design of gapmer ASOs, which utilize the endogenous RNase H1 enzyme to degrade target RNA. Learn how to create synthetic nucleotides that effectively cleave target RNAs, block splicing machinery to alter splicing outcomes, or inhibit translation by blocking ribosomes. Acquire essential knowledge and practical tips for developing highly effective ASOs with enhanced specificity and reduced off-target effects.