Aptamers as Therapeutics

Abstract

Aptamers are single-stranded nucleic acid molecules that bind to and inhibit proteins and are commonly produced by systematic evolution of ligands by exponential enrichment (SELEX). Aptamers undergo extensive pharmacological revision, which alters affinity, specificity, and therapeutic half-life, tailoring each drug for a specific clinical need. The first therapeutic aptamer was described 25 years ago. Thus far, one aptamer has been approved for clinical use, and numerous others are in preclinical or clinical development. This review presents a short history of aptamers and SELEX, describes their pharmacological development and optimization, and reviews potential treatment of diseases including visual disorders, thrombosis, and cancer.

Aptamer‐Engineered Natural Killer Cells for Cell‐Specific Adaptive Immunotherapy

“…Aptamers are a group of short, single‐stranded oligonucleotides with unique 3D structures . Similar to protein antibodies, aptamers specifically recognize their targets, which can include nucleic acids, proteins, cells, and even tissue, with high affinity .…”

Polymer Microneedle Mediated Local Aptamer Delivery for Blocking the Function of Vascular Endothelial Growth Factor

“…Despite the encouraging therapeutic effects observed in the clinic, antibodies as therapeutic agents have notable limitations 15,16 . For instance, antibodies are produced biologically, which requires strict production control and causes batch-to-batch variation 17,18 . They are also susceptible to irreversible denaturation which makes them hard to store on the shelf 18 .…”

“…For instance, antibodies are produced biologically, which requires strict production control and causes batch-to-batch variation 17,18 . They are also susceptible to irreversible denaturation which makes them hard to store on the shelf 18 . Antibodies also may present adverse immunogenic effects owing to their large sizes and production in biological systems 18 .…”

“…They are also susceptible to irreversible denaturation which makes them hard to store on the shelf 18 . Antibodies also may present adverse immunogenic effects owing to their large sizes and production in biological systems 18 . Therefore, the development of antibody mimics as drugs has attracted great attention over the past two decades.…”

“…1). Nucleic acid aptamers are an emerging class of synthetic single-stranded oligonucleotides that have been studied as potential antibody mimics 18,22–25 . While systemic aptamer delivery has been widely studied for the treatment of numerous human diseases 26–28 , local aptamer delivery has not received as much attention.…”