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克服帕金森病中推进干细胞疗法的方法学挑战。

Overcoming Methodological Challenges for Advancing Stem Cell Therapies in Parkinson's Disease.

机构信息

School of Health and Biomedical Sciences, RMIT University, Melbourne, VIC, Australia.

University of Melbourne, Florey Institute of Neuroscience and Mental Health, Parkville, VIC, Australia.

出版信息

Cell Transplant. 2024 Jan-Dec;33:9636897241246355. doi: 10.1177/09636897241246355.

DOI:10.1177/09636897241246355
PMID:38634440
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11027592/
Abstract

The quest for new and improved therapies for Parkinson's disease (PD) remains of paramount importance, despite previous trial failures. There is a current debate regarding the potential of stem cell research as a therapeutic approach for PD. The studies of dopaminergic fetal stem cells for PD treatment, their design, and the results of the initial surgical placebo-controlled trials were reviewed in this study. Some of the fundamental methodological challenges and possible strategies to resolve them were proposed. In this article, we argue that the most important impact lies in the proof-of-principle demonstrated by clinical trials for cell replacement strategies in reconstructing the human brain. While some researchers argue that the considerable technical challenges associated with cell therapies for PD warrant the discontinuation of further development using stem cells, we believe that the opposing viewpoints are instrumental in identifying a series of methodological misunderstandings. Here, we propose to expose key challenges to ensure the advancement of the field and unlock the potential of stem cell therapies in PD treatment. Overall, this review underscores the need for further research and innovation to overcome the hurdles in realizing the potential of stem cell-based therapies for PD.

摘要

尽管先前的试验失败了,但寻找治疗帕金森病(PD)的新方法和改进疗法仍然至关重要。目前,干细胞研究作为 PD 治疗方法的潜力存在争议。本研究回顾了用于 PD 治疗的多巴胺能胎儿干细胞的研究、设计以及初步手术安慰剂对照试验的结果。提出了一些解决基本方法学挑战的可能策略。在本文中,我们认为最重要的影响在于临床试验为重建人脑的细胞替代策略提供了原理证明。虽然一些研究人员认为与 PD 细胞疗法相关的重大技术挑战需要停止进一步使用干细胞的开发,但我们认为,相反的观点有助于确定一系列方法学误解。在这里,我们提出了一些关键挑战,以确保该领域的发展,并释放 PD 治疗中干细胞疗法的潜力。总的来说,这篇综述强调了需要进一步研究和创新,以克服实现基于干细胞的 PD 治疗潜力的障碍。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b989/11027592/1e22a330a7a9/10.1177_09636897241246355-fig1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b989/11027592/1e22a330a7a9/10.1177_09636897241246355-fig1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/b989/11027592/1e22a330a7a9/10.1177_09636897241246355-fig1.jpg

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本文引用的文献

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Assessment of heterogeneity among participants in the Parkinson's Progression Markers Initiative cohort using α-synuclein seed amplification: a cross-sectional study.采用 α-突触核蛋白种子扩增评估帕金森进展标志物倡议队列参与者的异质性:一项横断面研究。
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利用横断范式增强一种实验性人细胞治疗的修复机制。
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Recent Advances in the Development of Stem-Cell-Derived Dopaminergic Neuronal Transplant Therapies for Parkinson's Disease.近年来,基于干细胞的多巴胺能神经元移植疗法治疗帕金森病的研究进展。
Mov Disord. 2021 Aug;36(8):1772-1780. doi: 10.1002/mds.28628. Epub 2021 May 8.
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Gene and Cell-Based Therapies for Parkinson's Disease: Where Are We?基于基因和细胞的帕金森病疗法:我们在哪里?
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Recent developments in the treatment of Parkinson's Disease.帕金森病治疗的最新进展。
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