Stem cell therapy for spinocerebellar degeneration

脊髓小脑变性的干细胞疗法

• 批准号: 22659168
• 负责人: MIZUSAWA Hidehiro
• 金额: $ 2.07万
• 依托单位: Tokyo Medical and Dental University
• 依托单位国家: 日本
• 项目类别: Grant-in-Aid for Challenging Exploratory Research
• 财政年份: 2010
• 资助国家: 日本
• 起止时间: 2010 至 2011
• 项目状态: 已结题
• 来源: https://kaken.nii.ac.jp/grant/KAKENHI-PROJECT-22659168/
• 关键词: 神経分子病態学; 脊髄小脳変性症; ES細胞; 骨髄間葉系幹細胞; プルキンエ細胞; 神経新生; 再生医療; モデル動物; 脊髄小脳変成症

The purpose of this study is to establish novel stem cell therapy for spinocerebellar degeneration.We successfully established a system that induced the maturation of Purkinje cells(PCs) derived from mouse ES cells(ESC). ESC-derived Purkinje cells(EDPCs) showed the morphology of mature PCs and synaptogenesis with other cerebellar neurons. Furthermore, the electrophysiological properties of these EDPCs were compatible with those of native mature PCs in vitro, which were assessed by whole-cell patch-clamp recordings. Next, we transplanted these EDPCs into the cerebellum of Purkinje cell degeneration(PCD) mouse. Transplanted EDPCs showed abilities to survive and maintain mature Purkinje cell morphology in vivo.Thus, EDPCs show promise as a powerful source for stem cell therapies aimed at spinocerebellar degeneration.

本研究的目的是建立一种新的干细胞疗法来治疗脊髓小脑退行性变。我们成功地建立了一种诱导小鼠胚胎干细胞(ESC)来源的浦肯野细胞(PC)成熟的系统。胚胎干细胞来源的浦肯野细胞(EDPC)表现出成熟PC的形态，并与其他小脑神经元突触形成。此外，这些EDPC的电生理特性与天然成熟的PC在体外的电生理特性是相容的，这通过全细胞膜片钳记录进行了评估。接下来，我们将这些内源性神经干细胞移植到浦肯野细胞变性(PCD)小鼠的小脑中。移植的EDPC在活体中显示出存活和维持成熟浦肯野细胞形态的能力，因此，EDPC有望成为针对脊髓小脑变性的干细胞治疗的强大来源。

项目成果

東京医科歯科大学神経内科website

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• DOI: 10.1002/jnr.22501
• 发表时间: 2010-12
• 期刊: Journal of Neuroscience Research
• 影响因子: 4.2
• 作者: Haiyan Xu;K. Miki;S. Ishibashi;J. Inoue;Liyuan Sun;S. Endo;I. Sekiya;T. Muneta;J. Inazawa;M. Dezawa;H. Mizusawa

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• DOI: 10.1097/nen.0b013e318257fe7b
• 发表时间: 2012-06-01
• 期刊: JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY
• 影响因子: 3.2
• 作者: Kobayashi, Masaki;Ishibashi, Satoru;Mizusawa, Hidehiro
• 通讯作者: Mizusawa, Hidehiro