适配体作为治疗药物。

Nimjee Shahid M, White Rebekah R, Becker Richard C, Sullenger Bruce A

Department of Neurological Surgery, The Ohio State University Wexner Medical Center, Columbus, Ohio 43210; email:

Department of Surgery, Duke University Medical Center, Durham, North Carolina 27705; email:

Annu Rev Pharmacol Toxicol. 2017 Jan 6;57:61-79. 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.

适体是与蛋白质结合并抑制蛋白质的单链核酸分子，通常通过指数富集配体系统进化技术（SELEX）产生。适体经过广泛的药理学修饰，从而改变亲和力、特异性和治疗半衰期，为满足特定临床需求定制每种药物。25年前首次描述了治疗性适体。迄今为止，一种适体已获批用于临床，还有许多其他适体正处于临床前或临床开发阶段。本综述介绍了适体和SELEX的简短历史，描述了它们的药理学发展和优化，并综述了包括视觉障碍、血栓形成和癌症在内的疾病的潜在治疗方法。