Neuroprotective Potential of Cell-Based Therapies in ALS: From Bench to Bedside.

Neuroprotective Potential of Cell-Based Therapies in ALS: From Bench to Bedside.
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DOI:
10.3389/fnins.2017.00591
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发表时间:
2017
影响因子:
4.3
通讯作者:
Sykova E
Sykova E
中科院分区:
医学2区
文献类型:
--
作者:
Forostyak S;Sykova E

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运动神经元(MN)退行性变是肌萎缩性侧索硬化症(ALS)的主要特征,这是一种进行性神经系统疾病。ALS的诊断基本上是临床诊断。最常见的症状包括反映损伤和随后的肌肉功能丧失的逐渐神经退化。最新的ALS没有预防或治愈疾病的治疗方法。现代治疗策略包括以抗谷氨酸能、抗氧化、抗凋亡和抗炎分子为重点的神经保护治疗。干细胞的应用和基因治疗为研究人员发现新的机制和治疗药物提供了有力的工具,也为患者和家属开辟了新的视角。在此,我们综述了与ALS相关的基础、转化和临床干细胞研究的最新进展。我们概述了应用细胞疗法治疗神经退行性疾病的临床前和临床研究结果。一个特别的重点是神经保护特性的成体间充质间质细胞(MSC)应用于ALS患者。最后,我们综述了胚胎和诱导多能干细胞在神经退行性疾病特别是ALS中的建模和应用的最新进展。
Motor neurons (MN) degeneration is a main feature of amyotrophic lateral sclerosis (ALS), a neurological disorder with a progressive course. The diagnosis of ALS is essentially a clinical one. Most common symptoms include a gradual neurological deterioration that reflect the impairment and subsequent loss of muscle functions. Up-to-date ALS has no therapy that would prevent or cure a disease. Modern therapeutic strategies comprise of neuroprotective treatment focused on antiglutamatergic, antioxidant, antiapoptotic, and anti-inflammatory molecules. Stem cells application and gene therapy has provided researchers with a powerful tool for discovery of new mechanisms and therapeutic agents, as well as opened new perspectives for patients and family members. Here, we review latest progress made in basic, translational and clinical stem cell research related to the ALS. We overviewed results of preclinical and clinical studies employing cell-based therapy to treat neurodegenerative disorders. A special focus has been made on the neuroprotective properties of adult mesenchymal stromal cells (MSC) application into ALS patients. Finally, we overviewed latest progress in the field of embryonic and induced pluripotent stem cells used for the modeling and application during neurodegeneration in general and in ALS in particular.
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