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破解帕金森病之谜:从药物、手术到干细胞与基因——当前及未来治疗策略的全面综述

Unravelling the Parkinson's puzzle, from medications and surgery to stem cells and genes: a comprehensive review of current and future management strategies.

作者信息

Chandrababu Krishnapriya, Radhakrishnan Vineeth, Anjana A S, Rajan Rahul, Sivan Unnikrishnan, Krishnan Syam, Baby Chakrapani P S

机构信息

Centre for Neuroscience, Department of Biotechnology, Cochin University for Science and Technology, Kochi, Kerala, 682 022, India.

Comprehensive Care Centre for Movement Disorders, Department of Neurology, Sree Chitra Tirunal Institute for Medical Sciences and Technology, Thiruvananthapuram, Kerala, India.

出版信息

Exp Brain Res. 2024 Jan;242(1):1-23. doi: 10.1007/s00221-023-06735-1. Epub 2023 Nov 28.

Abstract

Parkinson's disease (PD) is a neurodegenerative disorder, prevalent in the elderly population. Neuropathological hallmarks of PD include loss of dopaminergic cells in the nigro-striatal pathway and deposition of alpha-synuclein protein in the neurons and synaptic terminals, which lead to a complex presentation of motor and non-motor symptoms. This review focuses on various aspects of PD, from clinical diagnosis to currently accepted treatment options, such as pharmacological management through dopamine replacement and surgical techniques such as deep brain stimulation (DBS). The review discusses in detail the potential of emerging stem cell-based therapies and gene therapies to be adopted as a cure, in contrast to the present symptomatic treatment in PD. The potential sources of stem cells for autologous and allogeneic stem cell therapy have been discussed, along with the progress evaluation of pre-clinical and clinical trials. Even though recent techniques hold great potential to improve the lives of PD patients, we present the importance of addressing the safety, efficacy, ethical, cost, and regulatory concerns before scaling them to clinical use.

摘要

帕金森病(PD)是一种神经退行性疾病,在老年人群中普遍存在。PD的神经病理学特征包括黑质-纹状体通路中多巴胺能细胞的丧失以及α-突触核蛋白在神经元和突触终末的沉积,这导致了运动和非运动症状的复杂表现。本综述聚焦于PD的各个方面,从临床诊断到目前公认的治疗选择,如通过多巴胺替代进行药物治疗以及深部脑刺激(DBS)等手术技术。与目前PD的对症治疗相比,本综述详细讨论了新兴的基于干细胞疗法和基因疗法作为治愈方法的潜力。已经讨论了用于自体和异体干细胞治疗的干细胞潜在来源,以及临床前和临床试验的进展评估。尽管最近的技术在改善PD患者生活方面具有巨大潜力,但我们强调在将这些技术扩大到临床应用之前,解决安全性、有效性、伦理、成本和监管问题的重要性。

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