Application of antisense oligonucleotide drugs in amyotrophic lateral sclerosis and Huntington's disease.

IF 10.8 1区医学 Q1 NEUROSCIENCES

Translational Neurodegeneration Pub Date : 2025-01-21 DOI:10.1186/s40035-025-00466-9

Kaili Ou, Qingqing Jia, Dandan Li, Shihua Li, Xiao-Jiang Li, Peng Yin

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引用次数: 0

Abstract

Amyotrophic lateral sclerosis (ALS) and Huntington's disease (HD) are diverse in clinical presentation and are caused by complex and multiple factors, including genetic mutations and environmental factors. Numerous therapeutic approaches have been developed based on the genetic causes and potential mechanisms of ALS and HD. Currently, available treatments for various neurodegenerative diseases can alleviate symptoms but do not provide a definitive cure. Gene therapy, which aims to modify or express specific proteins for neuroprotection or correction, is considered a powerful tool in managing neurodegenerative conditions. To date, antisense oligonucleotide (ASO) drugs targeting the pathological genes associated with ALS and HD have shown promising results in numerous animal studies and several clinical trials. This review provides a comprehensive overview of the development, mechanisms of action, limitations, and clinical applications of ASO drugs in neurodegenerative diseases, with a specific focus on ALS and HD therapeutic strategies.

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反义寡核苷酸药物在肌萎缩性侧索硬化和亨廷顿病中的应用。

肌萎缩性侧索硬化症（ALS）和亨廷顿舞蹈病（HD）的临床表现多样，是由复杂的多种因素引起的，包括基因突变和环境因素。基于ALS和HD的遗传原因和潜在机制，已经开发了许多治疗方法。目前，对各种神经退行性疾病的现有治疗方法可以减轻症状，但不能提供明确的治愈方法。基因治疗，其目的是修改或表达特定蛋白质的神经保护或纠正，被认为是管理神经退行性疾病的有力工具。迄今为止，针对ALS和HD相关病理基因的反义寡核苷酸（ASO）药物在大量动物研究和一些临床试验中显示出令人鼓舞的结果。本文综述了ASO药物在神经退行性疾病中的发展、作用机制、局限性和临床应用，重点介绍了ALS和HD的治疗策略。

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

Translational Neurodegeneration Neuroscience-Cognitive Neuroscience

CiteScore

19.50

自引率

0.80%

发文量

审稿时长

10 weeks

期刊介绍： Translational Neurodegeneration, an open-access, peer-reviewed journal, addresses all aspects of neurodegenerative diseases. It serves as a prominent platform for research, therapeutics, and education, fostering discussions and insights across basic, translational, and clinical research domains. Covering Parkinson's disease, Alzheimer's disease, and other neurodegenerative conditions, it welcomes contributions on epidemiology, pathogenesis, diagnosis, prevention, drug development, rehabilitation, and drug delivery. Scientists, clinicians, and physician-scientists are encouraged to share their work in this specialized journal tailored to their fields.