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小脑移植工作组文件:我们是否准备好用细胞疗法治疗小脑疾病?

Task Force Paper On Cerebellar Transplantation: Are We Ready to Treat Cerebellar Disorders with Cell Therapy?

机构信息

Department of Pathophysiology, Faculty of Medicine in Pilsen, Charles University, alej Svobody 1655/76, 323 00, Plzen, Czech Republic.

Laboratory of Neurodegenerative Disorders, Biomedical Center, Faculty of Medicine in Pilsen, Charles University, alej Svobody 1655/76, 323 00, Plzen, Czech Republic.

出版信息

Cerebellum. 2019 Jun;18(3):575-592. doi: 10.1007/s12311-018-0999-1.

Abstract

Restoration of damaged central nervous system structures, functional recovery, and prevention of neuronal loss during neurodegenerative diseases are major objectives in cerebellar research. The highly organized anatomical structure of the cerebellum with numerous inputs/outputs, the complexity of cerebellar functions, and the large spectrum of cerebellar ataxias render therapies of cerebellar disorders highly challenging. There are currently several therapeutic approaches including motor rehabilitation, neuroprotective drugs, non-invasive cerebellar stimulation, molecularly based therapy targeting pathogenesis of the disease, and neurotransplantation. We discuss the goals and possible beneficial mechanisms of transplantation therapy for cerebellar damage and its limitations and factors determining outcome.

摘要

恢复受损的中枢神经系统结构、功能恢复和预防神经退行性疾病中的神经元丢失是小脑研究的主要目标。小脑具有高度组织化的解剖结构,有众多的输入/输出,小脑功能复杂,小脑共济失调的范围广泛,这使得小脑疾病的治疗极具挑战性。目前有几种治疗方法,包括运动康复、神经保护药物、非侵入性小脑刺激、针对疾病发病机制的分子靶向治疗和神经移植。我们讨论了移植治疗小脑损伤的目标和可能有益的机制及其局限性和决定结果的因素。

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