Advances in aptamer technology for target-based drug discovery
Yingxian Cui, Yifan Chen, You-Bo Zhang, Liqin Zhang
Abstract
Aptamer therapeutics represent a class of target-based therapies that leverage their high specificity and affinity for diverse molecular targets. As single-stranded DNA or RNA oligonucleotides, aptamers offer advantages in therapeutic applications. A critical aspect of aptamer drug development is the selection process, which has seen significant advancements through various in vitro selection methods, including Systematic Evolution of Ligands by Exponential Enrichment and its emerging variations. Recent progress has also introduced functional screening strategies that directly identify pharmacologically active aptamers, accelerating drug discovery. The applications of aptamers in disease treatment are expanding across oncology, neurodegenerative disorders, infectious diseases and other diseases. Aptamers exhibit versatile mechanisms of action, including blocking interactions, recruiting protein machinery, and inhibiting target functions. By addressing key limitations and presenting future directions, this review provides a comprehensive perspective on the recent evolving landscape of aptamer technology and its transformative potential in modern medicine.