Lin Mengsi, Hu Xinyi, Chang Shiyi, Chang Yan, Bian Wenjun, Hu Ruikun, Wang Jing, Zhu Qingwen, Qiu Jiaying
Department of Prenatal Screening and Diagnosis Center, Affiliated Maternity and Child Health Care Hospital of Nantong University, Nantong, Jiangsu Province 226001, China.
School of Medicine, Nantong University, Nantong, Jiangsu Province 226001, China.
Evid Based Complement Alternat Med. 2021 Jun 10;2021:6678422. doi: 10.1155/2021/6678422. eCollection 2021.
Antisense nucleic acids are single-stranded oligonucleotides that have been specially chemically modified, which can bind to RNA expressed by target genes through base complementary pairing and affect protein synthesis at the level of posttranscriptional processing or protein translation. In recent years, the application of antisense nucleic acid technology in the treatment of neuromuscular diseases has made remarkable progress. In 2016, the US FDA approved two antisense nucleic acid drugs for the treatment of Duchenne muscular dystrophy (DMD) and spinal muscular atrophy (SMA), and the development to treat other neurodegenerative diseases has also entered the clinical stage. Therefore, ASO represents a treatment with great potential. The article will summarize ASO therapies in terms of mechanism of action, chemical modification, and administration methods and analyze their role in several common neurodegenerative diseases, such as SMA, DMD, and amyotrophic lateral sclerosis (ALS). This article systematically summarizes the great potential of antisense nucleic acid technology in the treatment of hereditary neurodegenerative diseases.
反义核酸是经过特殊化学修饰的单链寡核苷酸，它能通过碱基互补配对与靶基因表达的RNA结合，并在转录后加工或蛋白质翻译水平影响蛋白质合成。近年来，反义核酸技术在神经肌肉疾病治疗中的应用取得了显著进展。2016年，美国食品药品监督管理局（FDA）批准了两种用于治疗杜氏肌营养不良症（DMD）和脊髓性肌萎缩症（SMA）的反义核酸药物，治疗其他神经退行性疾病的研发也已进入临床阶段。因此，反义寡核苷酸（ASO）代表着一种极具潜力的治疗方法。本文将从作用机制、化学修饰和给药方式等方面总结ASO疗法，并分析其在几种常见神经退行性疾病，如SMA、DMD和肌萎缩侧索硬化症（ALS）中的作用。本文系统总结了反义核酸技术在治疗遗传性神经退行性疾病方面的巨大潜力。
