Yu Yuanyuan, Liang Chao, Lv Quanxia, Li Defang, Xu Xuegong, Liu Baoqin, Lu Aiping, Zhang Ge
Institute for Advancing Translational Medicine in Bone & Joint Diseases, School of Chinese Medicine, Hong Kong Baptist University, Hong Kong, China.
Zhengzhou Hospital of Traditional Chinese Medicine, Zhengzhou 450007, China.
Int J Mol Sci. 2016 Mar 11;17(3):358. doi: 10.3390/ijms17030358.
Monoclonal antibodies are the dominant agents used in inhibition of biological target molecules for disease therapeutics, but there are concerns of immunogenicity, production, cost and stability. Oligonucleotide aptamers have comparable affinity and specificity to targets with monoclonal antibodies whilst they have minimal immunogenicity, high production, low cost and high stability, thus are promising inhibitors to rival antibodies for disease therapy. In this review, we will compare the detailed advantages and disadvantages of antibodies and aptamers in therapeutic applications and summarize recent progress in aptamer selection and modification approaches. We will present therapeutic oligonucleotide aptamers in preclinical studies for skeletal diseases and further discuss oligonucleotide aptamers in different stages of clinical evaluation for various disease therapies including macular degeneration, cancer, inflammation and coagulation to highlight the bright commercial future and potential challenges of therapeutic oligonucleotide aptamers.
单克隆抗体是用于疾病治疗中抑制生物靶分子的主要药物，但存在免疫原性、生产、成本和稳定性等方面的问题。寡核苷酸适配体与单克隆抗体对靶标的亲和力和特异性相当，同时具有最小的免疫原性、高产率、低成本和高稳定性，因此有望成为与抗体竞争疾病治疗的抑制剂。在本综述中，我们将比较抗体和适配体在治疗应用中的详细优缺点，并总结适配体筛选和修饰方法的最新进展。我们将介绍用于骨骼疾病临床前研究的治疗性寡核苷酸适配体，并进一步讨论用于包括黄斑变性、癌症、炎症和凝血等各种疾病治疗的不同临床评估阶段的寡核苷酸适配体，以突出治疗性寡核苷酸适配体光明的商业前景和潜在挑战。
