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肌萎缩侧索硬化症全景:一种致命的复杂神经系统疾病。

The panoramic view of amyotrophic lateral sclerosis: A fatal intricate neurological disorder.

机构信息

Department of Immunology and Microbiology, School of Medicine, University of Texas Rio Grande Valley, McAllen, TX 78504, USA; South Texas Center of Excellence in Cancer Research, School of Medicine, University of Texas Rio Grande Valley, McAllen, TX 78504, USA.

Narula Research LLC, 107 Boulder Bluff, Chapel Hill, NC 27516, USA.

出版信息

Life Sci. 2022 Jan 1;288:120156. doi: 10.1016/j.lfs.2021.120156. Epub 2021 Nov 19.

DOI:10.1016/j.lfs.2021.120156
PMID:34801512
Abstract

Amyotrophic lateral sclerosis (ALS) is a progressive and fatal neurological disease affecting both upper and lower motor neurons. In the United States alone, there are 16,000-20,000 established cases of ALS. The early disease diagnosis is challenging due to many overlapping pathophysiologies with other neurological diseases. The etiology of ALS is unknown; however, it is divided into two categories: familial ALS (fALS) which occurs due to gene mutations & contributes to 5-10% of ALS, and sporadic ALS (sALS) which is due to environmental factors & contributes to 90-95% of ALS. There is still no curative treatment for ALS: palliative care and symptomatic treatment are therefore essential components in the management of these patients. In this review, we provide a panoramic view of ALS, which includes epidemiology, risk factors, pathophysiologies, biomarkers, diagnosis, therapeutics (natural, synthetic, gene-based, pharmacological, stem cell, extracellular vesicles, and physical therapy), controversies (in the clinical trials of ALS), the scope of nanomedicine in ALS, and future perspectives.

摘要

肌萎缩侧索硬化症(ALS)是一种影响上下运动神经元的进行性和致命性神经退行性疾病。仅在美国,就有 16000-20000 例已确诊的 ALS 病例。由于与其他神经退行性疾病有许多重叠的病理生理学,早期疾病诊断具有挑战性。ALS 的病因尚不清楚;然而,它分为两类:家族性 ALS(fALS)是由于基因突变引起的,占 ALS 的 5-10%,散发性 ALS(sALS)是由于环境因素引起的,占 ALS 的 90-95%。目前仍然没有治愈 ALS 的方法:因此,姑息治疗和对症治疗是这些患者治疗的重要组成部分。在这篇综述中,我们全面介绍了 ALS,包括流行病学、危险因素、病理生理学、生物标志物、诊断、治疗(天然、合成、基于基因、药理学、干细胞、细胞外囊泡和物理治疗)、争议(在 ALS 的临床试验中)、纳米医学在 ALS 中的应用范围和未来展望。

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