Disease-specific interventions: The use of cell and gene therapies for Parkinson disease.

Q2 Medicine Handbook of clinical neurology Pub Date : 2024-01-01 DOI:10.1016/B978-0-323-90120-8.00003-4

Roger A Barker, Philip C Buttery

引用次数: 0

Abstract

Approaches to repair the brain around the loss of the nigrostriatal dopaminergic pathways in Parkinson disease (PD) are not new and have been attempted over many years. However, of late, the situation has moved forward in two main ways. In the case of cell therapies, the ability to make large numbers of authentic midbrain dopaminergic neuroblasts from human pluripotent stem cell sources has turned what was an interesting avenue of research into a major area of investment and trialing, by academics in conjunction with Pharma. In the case of gene therapies, their use around dopamine replacement has waned, as the interest in using them for disease modification targeting PD-specific pathways has grown. In this chapter, we discuss all these developments and the current status of cell and gene therapies for PD.

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针对特定疾病的干预措施：使用细胞和基因疗法治疗帕金森病。

围绕帕金森病（PD）黑质多巴胺能通路的缺失修复大脑的方法并不新鲜，多年来一直在尝试。然而，近来情况主要在两个方面发生了变化。就细胞疗法而言，从人类多能干细胞来源制造大量真实中脑多巴胺能神经母细胞的能力，已将一个有趣的研究途径变成了学术界与制药公司共同投资和试验的主要领域。就基因疗法而言，随着人们对利用基因疗法针对帕金森病特异性通路进行疾病修饰的兴趣日益浓厚，基因疗法在多巴胺替代方面的应用已日渐式微。在本章中，我们将讨论所有这些进展以及治疗帕金森病的细胞和基因疗法的现状。

本文章由计算机程序翻译，如有差异，请以英文原文为准。

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来源期刊

Handbook of clinical neurology Medicine-Neurology (clinical)

CiteScore

4.10

自引率

0.00%

发文量

302

期刊介绍： The Handbook of Clinical Neurology (HCN) was originally conceived and edited by Pierre Vinken and George Bruyn as a prestigious, multivolume reference work that would cover all the disorders encountered by clinicians and researchers engaged in neurology and allied fields. The first series of the Handbook (Volumes 1-44) was published between 1968 and 1982 and was followed by a second series (Volumes 45-78), guided by the same editors, which concluded in 2002. By that time, the Handbook had come to represent one of the largest scientific works ever published. In 2002, Professors Michael J. Aminoff, François Boller, and Dick F. Swaab took on the responsibility of supervising the third (current) series, the first volumes of which published in 2003. They have designed this series to encompass both clinical neurology and also the basic and clinical neurosciences that are its underpinning. Given the enormity and complexity of the accumulating literature, it is almost impossible to keep abreast of developments in the field, thus providing the raison d''être for the series. The series will thus appeal to clinicians and investigators alike, providing to each an added dimension. Now, more than 140 volumes after it began, the Handbook of Clinical Neurology series has an unparalleled reputation for providing the latest information on fundamental research on the operation of the nervous system in health and disease, comprehensive clinical information on neurological and related disorders, and up-to-date treatment protocols.

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