RNA therapeutics for metabolic disorders.

3区 生物学 Q2 Biochemistry, Genetics and Molecular Biology
Thuy-Duong Vu, Sheng-Che Lin, Chia-Ching Wu, Dinh-Toi Chu
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引用次数: 0

Abstract

The prevalence of metabolic disorders is increasing exponentially and has recently reached epidemic levels. Over the decades, a large number of therapeutic options have been proposed to manage these diseases but still show several limitations. In this circumstance, RNA therapeutics have rapidly emerged as a new hope for patients with metabolic diseases. 57 years have elapsed from the discovery of mRNA, a large number of RNA-based drug candidates have been evaluated for their therapeutic effectiveness and clinical safety under clinical studies. To date, there are seven RNA drugs for treating metabolic disorders receiving official approval and entering the global market. Their targets include hereditary transthyretin-mediated amyloidosis (hATTR), familial chylomicronemia syndrome, acute hepatic porphyria, primary hyperoxaluria type 1 and hypercholesterolemia, which are all related to liver proteins. All of these seven RNA drugs are antisense oligonucleotides (ASO) and small interfering RNA (siRNA). These two types of treatment are both based on oligonucleotides complementary to target RNA through Watson-Crick base-pairing, but their mechanisms of action include different nucleases. Such treatments show greatest potential among all types of RNA therapeutics due to consecutive achievements in chemical modifications. Another method, mRNA therapeutics also promise a brighter future for patients with a handful of drug candidates currently under development.

治疗代谢紊乱的 RNA 疗法。
代谢性疾病的发病率呈指数级增长,最近已达到流行病的水平。几十年来,人们提出了大量治疗这些疾病的方案,但仍存在一些局限性。在这种情况下,RNA 疗法迅速崛起,成为代谢性疾病患者的新希望。从发现 mRNA 至今已有 57 年的时间,大量基于 RNA 的候选药物在临床研究中接受了治疗效果和临床安全性的评估。迄今为止,已有七种治疗代谢性疾病的 RNA 药物获得正式批准并进入全球市场。它们的靶点包括遗传性转甲状腺素介导的淀粉样变性(hATTR)、家族性乳糜泻综合征、急性肝卟啉症、原发性高草酸尿症 1 型和高胆固醇血症,均与肝脏蛋白有关。这七种 RNA 药物均为反义寡核苷酸(ASO)和小干扰 RNA(siRNA)。这两种治疗方法都是基于通过沃森-克里克碱基配对与靶 RNA 互补的寡核苷酸,但它们的作用机制包括不同的核酸酶。由于化学修饰技术的不断进步,这类疗法在所有类型的 RNA 疗法中显示出最大的潜力。另一种方法--mRNA疗法也为患者带来了光明的未来,目前有一些候选药物正在开发中。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
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来源期刊
CiteScore
5.00
自引率
0.00%
发文量
110
审稿时长
4-8 weeks
期刊介绍: Progress in Molecular Biology and Translational Science (PMBTS) provides in-depth reviews on topics of exceptional scientific importance. If today you read an Article or Letter in Nature or a Research Article or Report in Science reporting findings of exceptional importance, you likely will find comprehensive coverage of that research area in a future PMBTS volume.
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