Oligonucleotide Synthesis and State-of-the-Art Drug Discovery

Oligonucleotide therapeutics are currently attracting attention as one of the new drug discovery modalities for difficult-to-treat diseases. In addition, research on the development of oligonucleotide therapeutics is becoming more active, and research areas related to nucleic acid chemistry have made rapid progress in recent years. This chapter focuses on antisense oligonucleotides (ASOs), as typical oligonucleotide therapeutics, and introduces the mechanism of action, sequence design guidelines, and chemical modifications to enhance antisense activity and reduce toxicity.