Department of Biochemistry, University of Otago, Dunedin 9016, New Zealand.
Centre for Molecular Medicine and Innovative Therapeutics, Murdoch University, Perth 6150, Australia.
Int J Mol Sci. 2021 Mar 24;22(7):3295. doi: 10.3390/ijms22073295.
Cancer is one of the leading causes of death worldwide. Conventional therapies, including surgery, radiation, and chemotherapy have achieved increased survival rates for many types of cancer over the past decades. However, cancer recurrence and/or metastasis to distant organs remain major challenges, resulting in a large, unmet clinical need. Oligonucleotide therapeutics, which include antisense oligonucleotides, small interfering RNAs, and aptamers, show promising clinical outcomes for disease indications such as Duchenne muscular dystrophy, familial amyloid neuropathies, and macular degeneration. While no approved oligonucleotide drug currently exists for any type of cancer, results obtained in preclinical studies and clinical trials are encouraging. Here, we provide an overview of recent developments in the field of oligonucleotide therapeutics in oncology, review current clinical trials, and discuss associated challenges.
癌症是全球主要死因之一。在过去几十年中，包括手术、放疗和化疗在内的常规疗法已使许多类型癌症的生存率得到提高。然而，癌症的复发和/或转移至远处器官仍然是主要挑战，导致存在巨大的、未满足的临床需求。寡核苷酸疗法，包括反义寡核苷酸、小干扰 RNA 和适体，在杜氏肌营养不良症、家族性淀粉样神经病和黄斑变性等疾病适应症方面显示出有希望的临床结果。虽然目前尚无任何类型癌症的批准寡核苷酸药物，但临床前研究和临床试验的结果令人鼓舞。在这里，我们概述了肿瘤学领域寡核苷酸疗法的最新进展，综述了当前的临床试验，并讨论了相关挑战。
