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间充质干细胞治疗帕金森病的挑战与转化考量

Challenges and translational considerations of mesenchymal stem/stromal cell therapy for Parkinson's disease.

作者信息

Fričová Dominika, Korchak Jennifer A, Zubair Abba C

机构信息

Department of Laboratory Medicine and Pathology and Center for Regenerative Medicine, Mayo Clinic, Jacksonville, FL, USA.

Institute of Neuroimmunology, Slovak Academy of Sciences, Bratislava, Slovak Republic.

出版信息

NPJ Regen Med. 2020 Nov 3;5(1):20. doi: 10.1038/s41536-020-00106-y.

DOI:10.1038/s41536-020-00106-y
PMID:33298940
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC7641157/
Abstract

Parkinson's disease (PD) is the second most common neurodegenerative disease characterized by the progressive loss of dopaminergic neurons in the substantia nigra pars compacta and the presence of Lewy bodies, which gives rise to motor and non-motor symptoms. Unfortunately, current therapeutic strategies for PD merely treat the symptoms of the disease, only temporarily improve the patients' quality of life, and are not sufficient for completely alleviating the symptoms. Therefore, cell-based therapies have emerged as a novel promising therapeutic approach in PD treatment. Mesenchymal stem/stromal cells (MSCs) have arisen as a leading contender for cell sources due to their regenerative and immunomodulatory capabilities, limited ethical concerns, and low risk of tumor formation. Although several studies have shown that MSCs have the potential to mitigate the neurodegenerative pathology of PD, variabilities in preclinical and clinical trials have resulted in inconsistent therapeutic outcomes. In this review, we strive to highlight the sources of variability in studies using MSCs in PD therapy, including MSC sources, the use of autologous or allogenic MSCs, dose, delivery methods, patient factors, and measures of clinical outcome. Available evidence indicates that while the use of MSCs in PD has largely been promising, conditions need to be standardized so that studies can be effectively compared with one another and experimental designs can be improved upon, such that this body of science can continue to move forward.

摘要

帕金森病(PD)是第二常见的神经退行性疾病,其特征是黑质致密部多巴胺能神经元进行性丧失以及路易小体的出现,这会引发运动和非运动症状。不幸的是,目前针对帕金森病的治疗策略仅能治疗疾病症状,只是暂时改善患者生活质量,不足以完全缓解症状。因此,基于细胞的疗法已成为帕金森病治疗中一种新的有前景的治疗方法。间充质干/基质细胞(MSCs)因其再生和免疫调节能力、有限的伦理问题以及低肿瘤形成风险,已成为细胞来源的主要竞争者。尽管多项研究表明间充质干细胞有减轻帕金森病神经退行性病理的潜力,但临床前和临床试验中的变异性导致了治疗结果不一致。在本综述中,我们努力突出帕金森病治疗中使用间充质干细胞的研究中的变异性来源,包括间充质干细胞来源、自体或异体间充质干细胞的使用、剂量、给药方法、患者因素以及临床结果的衡量指标。现有证据表明,虽然在帕金森病中使用间充质干细胞总体上很有前景,但需要规范条件,以便能有效地相互比较研究,并改进实验设计,从而使这一科学领域能够继续向前发展。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/11f7/7641157/d4b0a35781b1/41536_2020_106_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/11f7/7641157/d4b0a35781b1/41536_2020_106_Fig1_HTML.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/11f7/7641157/d4b0a35781b1/41536_2020_106_Fig1_HTML.jpg

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