Department of Surgery, The Fourth Affiliated Hospital of School of Medicine, and International School of Medicine, International Institutes of Medicine, Zhejiang University, Yiwu 322000, China.
Department of Surgery, The Fourth Affiliated Hospital of School of Medicine, and International School of Medicine, International Institutes of Medicine, Zhejiang University, Yiwu 322000, China; Department of Colorectal Surgery and Oncology, Key Laboratory of Cancer Prevention and Intervention, Ministry of Education, 2nd Affiliated Hospital, Zhejiang University School of Medicine, Jiefang Road 88th, Hangzhou 310009, China.
Int Immunopharmacol. 2024 Dec 5;142(Pt B):113157. doi: 10.1016/j.intimp.2024.113157. Epub 2024 Sep 16.
Nucleic acid is an essential biopolymer in all living cells, performing the functions of storing and transmitting genetic information and synthesizing protein. In recent decades, with the progress of science and biotechnology and the continuous exploration of the functions performed by nucleic acid, more and more studies have confirmed that nucleic acid therapy for living organisms has great medical therapeutic potential. Nucleic acid drugs began to become independent therapeutic agents. As a new therapeutic method, nucleic acid therapy plays an important role in the treatment of genetic diseases, viral infections and cancers. There are currently 19 nucleic acid drugs approved by the Food and Drug Administration (FDA). In the following review, we start from principles and advantages of nucleic acid therapy, and briefly describe development history of nucleic acid drugs. And then we give examples of various RNA therapeutic drugs, including antisense oligonucleotides (ASO), mRNA vaccines, small interfering RNA (siRNA) and microRNA (miRNA), aptamers, and small activating RNA (saRNA). In addition, we also focused on the current status of nucleic acid drugs used in cancer therapy and the breakthrough in recent years. Clinical trials of nucleic acid drugs for cancer treatment are under way, conventional radiotherapy and chemotherapy combined with the immunotherapies such as checkpoint inhibitors and nucleic acid drugs may be the main prospects for successful cancer treatment.
核酸是所有活细胞中必不可少的生物聚合物,具有存储和传递遗传信息以及合成蛋白质的功能。近几十年来,随着科学和生物技术的进步以及对核酸所发挥功能的不断探索,越来越多的研究证实,核酸治疗生物体具有巨大的医学治疗潜力。核酸药物开始成为独立的治疗药物。作为一种新的治疗方法,核酸疗法在治疗遗传疾病、病毒感染和癌症方面发挥着重要作用。目前,美国食品和药物管理局 (FDA) 已批准了 19 种核酸药物。在下面的综述中,我们从核酸疗法的原理和优势出发,简要描述了核酸药物的发展历史。然后,我们举例说明了各种 RNA 治疗药物,包括反义寡核苷酸 (ASO)、mRNA 疫苗、小干扰 RNA (siRNA) 和 microRNA (miRNA)、适体和小激活 RNA (saRNA)。此外,我们还重点关注了用于癌症治疗的核酸药物的现状和近年来的突破。用于癌症治疗的核酸药物的临床试验正在进行中,常规放疗和化疗与检查点抑制剂等免疫疗法相结合,以及核酸药物可能是癌症治疗成功的主要前景。