Biology Department, Indian Institute of Science Education and Research (IISER), 900 NCL Innovation Park, Dr. Homi Bhabha Road, Pune, 411008, India,
J Mol Med (Berl). 2013 Dec;91(12):1333-42. doi: 10.1007/s00109-013-1085-2. Epub 2013 Sep 18.
Aptamers are single-stranded oligonucleotides that fold into well-defined three-dimensional shapes, allowing them to bind their targets with high affinity and specificity. They can be generated through an in vitro process called "Systemic Evolution of Ligands by Exponential Enrichment" and applied for specific detection, inhibition, and characterization of various targets like small organic and inorganic molecules, proteins, and whole cells. Aptamers have also been called chemical antibodies because of their synthetic origin and their similar modes of action to antibodies. They exhibit significant advantages over antibodies in terms of their small size, synthetic accessibility, and ability to be chemically modified and thus endowed with new properties. The first generation of aptamer drug "Macugen" was available for public use within 25 years of the discovery of aptamers. With others in the pipeline for clinical trials, this emerging field of medical biotechnology is raising significant interest. However, aptamers pose different problems for their development than for antibodies that need to be addressed to achieve practical applications. It is likely that current developments in aptamer engineering will be the basis for the evolution of improved future bioanalytical and biomedical applications. The present review discusses the development of aptamers for therapeutics, drug delivery, target validation and imaging, and reviews some of the challenges to fully realizing the promise of aptamers in biomedical applications.
适配子是单链寡核苷酸,可折叠成具有明确三维形状的分子,使其能够与靶标高亲和力和特异性结合。它们可以通过称为“指数富集的配体系统进化”的体外过程产生,并应用于特定的检测、抑制和表征各种目标,如小分子有机和无机分子、蛋白质和整个细胞。由于其合成来源和与抗体相似的作用模式,适配子也被称为化学抗体。与抗体相比,它们具有明显的优势,如体积小、合成可及性以及能够进行化学修饰,从而具有新的特性。第一代适配体药物“Macugen”在发现适配体后的 25 年内就可供公众使用。随着其他药物进入临床试验阶段,这个新兴的医疗生物技术领域引起了极大的兴趣。然而,适配体在开发过程中面临的问题与抗体不同,需要解决这些问题才能实现实际应用。当前适配体工程的发展很可能为未来生物分析和生物医学应用的改进奠定基础。本文综述了适配体在治疗、药物输送、靶标验证和成像方面的发展,并讨论了在充分实现适配体在生物医学应用中的潜力方面所面临的一些挑战。