Stem cell therapy for spinocerebellar degeneration

本研究的目的是建立新的干细胞治疗脊髓小脑变性。我们成功地建立了一个诱导来自小鼠胚胎干细胞（ESC）的浦肯野细胞（PCs）成熟的系统。esc来源的浦肯野细胞（EDPCs）表现出成熟的形态，并与其他小脑神经元突触发生。此外，这些edpc的电生理特性与体外天然成熟pc的电生理特性一致，并通过全细胞膜片钳记录进行了评估。接下来，我们将这些EDPCs移植到浦肯野细胞变性（PCD）小鼠小脑中。移植的EDPCs在体内表现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。

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.