Unveiling the potential of antisense oligonucleotides: Mechanisms, therapies, and safety insights

Abstract

Antisense oligonucleotides (ASOs) are short, synthetic, single‐stranded deoxynucleotide sequences composed of phosphate backbone‐connected sugar rings. Designing of those strands is based on Watson‐Crick hydrogen bonding mechanism. Thanks to rapidly advancing medicine and technology, evolving of the gene therapy area and ASO approaches gain attention. Considering the genetic basis of diseases, it is promising that gene therapy approaches offer more specific and effective options compared to conventional treatments. The objective of this review is to explain the mechanism of ASOs and discuss the characteristics and safety profiles of therapeutic agents in this field. Pharmacovigilance for gene therapy products is complex, requiring accurate assessment of benefit‐risk balance and evaluation of adverse effects.