Stem and progenitor cell-based therapy of myelin disorders.

Q2 Medicine

Handbook of clinical neurology Pub Date : 2024-01-01 DOI:10.1016/B978-0-323-90120-8.00015-0

Steven A Goldman, Robin J M Franklin, Joana Osorio

引用次数: 0

Abstract

Much of clinical neurology is concerned with diseases of-or involving-the brain's subcortical white matter. Common to these disorders is the loss of myelin, reflecting the elimination or dysfunction of oligodendrocytes and fibrous astrocytes. As such, the introduction of glial progenitor cells, which can give rise to new oligodendrocytes and astrocytes alike, may be a feasible strategy for treating a broad variety of conditions in which white matter loss is causally involved. This review first covers the sourcing and production of human glial progenitor cells, and the preclinical evidence for their efficacy in achieving myelin restoration in vivo. It then discusses both pediatric and adult disease targets for which transplanted glial progenitors may prove of therapeutic value, those challenges that remain in the clinical application of a glial cell replacement strategy, and the clinical endpoints by which the efficacy of this approach may be assessed.

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基于干细胞和祖细胞的髓鞘疾病疗法。

临床神经病学主要关注大脑皮质下白质的疾病或涉及大脑皮质下白质的疾病。这些疾病的共同特点是髓鞘脱失，反映了少突胶质细胞和纤维星形胶质细胞的消失或功能障碍。因此，引入能产生新的少突胶质细胞和星形胶质细胞的胶质祖细胞，可能是治疗各种因白质丢失引起的疾病的可行策略。这篇综述首先介绍了人类胶质祖细胞的来源和生产，以及临床前证据证明其在体内实现髓鞘恢复的功效。然后讨论移植神经胶质祖细胞可能被证明具有治疗价值的儿科和成人疾病靶点、神经胶质细胞替代策略在临床应用中仍然面临的挑战，以及评估这种方法疗效的临床终点。

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.