小核苷酸药物在神经系统疾病治疗领域的临床研究进展
Clinical Research Progress of Oligonucleotide Drugs in the Field of Neurotherapy
董漪 1董强1
作者信息
- 1. 复旦大学附属华山医院神经科,上海 200040
- 折叠
摘要
目前,多项小核苷酸药物已进入临床试验阶段,并取得了显著进展.小核苷酸药物的主要作用机制是通过特异性靶向神经细胞内的mRNA或miRNA调控基因表达,从而达到治疗神经系统疾病的目的.在脑卒中、阿尔茨海默病、帕金森病等常见病治疗中,小核苷酸药物展现出了良好的疗效和安全性.同时,在周围神经病和骨骼肌肉系统疾病等罕见病治疗中,小核苷酸药物也显示出了促进神经再生和修复的潜力.这些研究成果为神经系统疾病治疗领域带来了新的希望和突破,未来有望为神经系统疾病患者提供新的治疗选择.也为小核苷酸药物在其他疾病领域的应用提供了借鉴和参考.
Abstract
In recent years,significant progression has been made in the clinical research of oligonucleotide drugs.These drugs specifically target mRNA or miRNA within neuronal cells to regulate gene expression,thereby achieving the goal of treating neurological diseases.Oligonucleotide drugs have demonstrated good efficacy and safety in the treatment of neurodegenerative diseases such as Alzheimer's disease and Parkinson's disease.Additionally,in the treatment of rare disease such as peripheral neuropathy,myopathy,oligonucleotide drugs have shown potential in promoting neural regeneration and repairment.A numberi of multiple oligonucleotide drugs have entered the clinical trial stages and are expected to provide a new therapeutic options for patients with neurological diseases in the future.These research findings bring new hope and breakthroughs to the field of neurological treatment and serve as a reference for the application of oligonucleotide drugs in other disease areas.
关键词
小核苷酸药物/神经系统疾病/脑卒中/治疗Key words
oligonucleotide drugs/neurological disorder/stroke/treatment引用本文复制引用
出版年
2024
NETL
NSTL
万方数据