Aptamers are single-stranded
RNA or
DNA molecules that bind to target molecules by forming complex three-dimensional structures. Their target molecules vary widely—including proteins, peptides, carbohydrates, lipids, small compounds, and
metal ions—and because of their
strong binding affinity and high specificity, aptamers have been put to practical use in fields such as therapeutics, diagnostics, and separation technologies. As pharmaceuticals, the world's first
aptamer drug, Macugen®, was approved in the United States in 2004 as a treatment for neovascular age-related
macular degeneration, and several other aptamers are now in clinical development. Research is also progressing on their use as tools in
drug delivery systems (DDS), and the importance of aptamers in the pharmaceutical field is expected to continue growing. In this article, we provide an overview of
aptamer acquisition methods and optimization strategies for therapeutic use, and introduce the latest developments in
aptamer-based drugs currently in clinical trials.