Chemical Biology and Chemical Genetics, Life and Medical Sciences (LIMES) Institute, University of Bonn, Gerhard-Domagk-Str. 1, 53121, Bonn, Germany.
Center of Aptamer Research and Development (CARD), University of Bonn, Gerhard-Domagk-Str. 1, 53121, Bonn, Germany.
Mol Diagn Ther. 2019 Jun;23(3):301-309. doi: 10.1007/s40291-019-00400-6.
Aptamers are synthetic DNA or RNA oligonucleotide ligands with great potential for therapeutic applications. A vast number of disease-related targets have been used to identify agonistic, antagonistic, or inhibitory aptamers, or aptamer-based targeting ligands. However, only a few aptamers have reached late-stage clinical trials so far and the commercial infrastructure is still far behind that of other therapeutic agents such as monoclonal antibodies. The desirable properties of aptamers such as selectivity, chemical flexibility, or cost-efficiency are faced by challenges, including a short half-life in vivo, immunogenicity, and entrapment in cellular organelles. Aptamer research is still in an early stage, and a deeper understanding of their structure, target interactions, and pharmacokinetics is necessary to catch up to the clinical market. In this review, we will discuss the benefits and limitations in the development of therapeutic aptamers, as well as the advances and future directions of aptamer research. The progress towards effective therapies seems to be slow, but it has not stopped and the best is yet to come.
适配子是具有巨大治疗应用潜力的合成 DNA 或 RNA 寡核苷酸配体。已经使用了大量与疾病相关的靶标来鉴定激动剂、拮抗剂或抑制剂适配子，或基于适配子的靶向配体。然而，迄今为止，只有少数适配子进入了后期临床试验，商业基础设施仍远远落后于其他治疗药物，如单克隆抗体。适配子的理想特性，如选择性、化学灵活性或成本效益，面临着一些挑战，包括体内半衰期短、免疫原性和被细胞细胞器困住。适配子研究仍处于早期阶段，需要更深入地了解它们的结构、靶标相互作用和药代动力学，才能赶上临床市场。在这篇综述中，我们将讨论治疗性适配子的开发的益处和局限性，以及适配子研究的进展和未来方向。迈向有效治疗的进展似乎缓慢，但并没有停止，最好的还在后头。
