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肌萎缩侧索硬化的药物治疗进展

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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.
Advances in pharmacotherapy for amyotrophic lateral sclerosis
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.

amyotrophic lateral sclerosispharmacotherapygene therapy

杨天米、商慧芳

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四川大学华西医院神经内科,成都 610041

肌萎缩侧索硬化 药物治疗 基因治疗

国家自然科学基金面上项目四川省生命健康重大科技资助专项(十四五)

823714302022ZDZX0023

2024

重庆医科大学学报
重庆医科大学

重庆医科大学学报

CSTPCD北大核心
影响因子:0.724
ISSN:0253-3626
年,卷(期):2024.49(5)
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