肌萎缩侧索硬化的药物治疗进展

Advances in pharmacotherapy for amyotrophic lateral sclerosis

摘要

肌萎缩侧索硬化（amyotrophic lateral sclerosis，ALS）是一种以上、下运动神经元选择性丢失为主要特征，进行性加重的神经系统变性疾病，目前尚无有效的治愈方法。随着ALS疾病机制和遗传学的研究深入，以及基因调控策略的创新，为ALS疗法开发创造了有希望的新选择。本文围绕反义寡核苷酸疗法、腺病毒相关病毒介导的基因治疗和细胞治疗在ALS中的研究进展进行综述。

Abstract

Amyotrophic lateral sclerosis（ALS） is a progressive neurodegenerative disease characterized by the selective loss of upper and lower motor neurons. Effective cures for this disease have not been available yet. However，promising options for the treatment of ALS have been approaching thanks to the growing understanding of ALS pathogenesis and genetics as well as the innovation of gene regulation strategies. This article provides a comprehensive review of advances on antisense oligonucleotide therapy， adeno-associated virus-mediated gene therapy，and cell therapy in ALS.

关键词

肌萎缩侧索硬化 / 药物治疗 / 基因治疗

Key words

amyotrophic lateral sclerosis / pharmacotherapy / gene therapy

基金资助

国家自然科学基金面上资助项目(82371430)

四川省“十四五”生命健康重大科技资助专项(2022ZDZX0023)