Aptamers as Therapeutics

• Published in: Annual review of pharmacology and toxicology Vol. 57; no. 1; pp. 61 - 79
• Main Authors: Nimjee, Shahid M, White, Rebekah R, Becker, Richard C, Sullenger, Bruce A
• Format: Journal Article
• Language: English
• Published: United States Annual Reviews 06-01-2017
• Subjects: Animals; antidote; aptamer; Aptamers, Nucleotide - administration & dosage; Aptamers, Nucleotide - genetics; Aptamers, Nucleotide - metabolism; Humans; Neoplasms - drug therapy; Neoplasms - metabolism; pharmacology; Randomized Controlled Trials as Topic - methods; SELEX; SELEX Aptamer Technique - methods; SELEX Aptamer Technique - trends; therapeutics; Vision Disorders - drug therapy; Vision Disorders - metabolism; therapeutics; aptamer; antidote; pharmacology; SELEX
• ISSN: 0362-1642; 1545-4304
• DOI: 10.1146/annurev-pharmtox-010716-104558

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.