How to design antisense oligonucleotides (ASOs) for high on-target potency and minimal off-target effect

How to design antisense oligonucleotides (ASOs) for high on-target potency and minimal off-target effect

Antisense oligonucleotides (ASOs) are synthetic nucleotides that are designed to modulate gene expression.

There are different types of ASOs with different mechanisms of action. RNase H1 gapmer ASOs are chemically modified and designed to cleave their target RNAs. Steric blocking ASOs, are often designed to block the splicing machinery to alter splicing outcomes, or to block the ribosome to inhibit translation.

By watching this webinar, you’ll pick up insightful tips on gapmer ASOs, which use the endogenous RNase H1 enzyme to degrade a target RNA.

In this webinar you will learn:

  • Brief history of ASOs
  • Chemical modifications of ASOs and guidelines on selecting the right modification
  • Design guidelines for enriching high potency ASOs
  • Guidelines for in vitro ASO delivery

Published on: July 12, 2023