治疗神经系统疾病的 RNA 疗法。

3区 生物学 Q2 Biochemistry, Genetics and Molecular Biology
Lien Nguyen
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引用次数: 0

摘要

神经系统疾病是一组主要影响中枢神经系统的疾病,可对学习新技能、说话、呼吸、行走、判断、决策和其他基本生活技能产生重大负面影响。近十年来,神经系统疾病对我们社会的影响显著增加,已成为导致残疾和死亡的主要原因之一。世界卫生组织已将包括阿尔茨海默病和其他痴呆症在内的神经系统疾病确定为现代生活中的健康危机。目前正在进行的大量研究工作侧重于了解疾病遗传学、分子机制和开发治疗干预措施。由于对有效疗法的迫切需求,以及最近在开发更多类似药物的 RNA 分子的工具包和理解方面取得的进展,人们对开发治疗神经系统疾病的 RNA 疗法的兴趣与日俱增。本文将讨论神经系统疾病的遗传学和发病机制、如何利用 RNA 分子开发这类疾病的疗法、RNA 疗法面临的挑战以及这一新兴治疗干预工具的未来前景。
本文章由计算机程序翻译,如有差异,请以英文原文为准。
RNA therapeutics for neurological disease.

Neurological disorders are the group of diseases that primarily affect the center nervous system, which could lead to a significant negative impact on the ability of learning new skills, speaking, breathing, walking, judging, making decision, and other essential living skills. In the last decade, neurological disorders have significantly increased their impact to our community and become the one of leading causes of disability and death. The World Health Organization has identified neurological disorders including Alzheimer's disease and other dementia as the health crisis for the modern life. Tremendous ongoing research efforts focus on understanding of disease genetics, molecular mechanisms and developing therapeutic interventions. Because of the urgent need of the effective therapeutics and the recent advances in the toolkits and understanding for developing more drug-like RNA molecules, there is a growing interest for developing RNA therapeutics for neurological disorders. This article will discuss genetics and mechanisms of neurological disorders and how RNA-based molecules have been used to develop therapeutics for this group of diseases, challenges of RNA therapeutics and future perspectives on this rising therapeutic intervention tool.

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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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