A cell therapy approach based on iPSC-derived midbrain organoids for the restoration of motor function in a Parkinson's disease mouse model.
A cell therapy approach based on iPSC-derived midbrain organoids for the restoration of motor function in a Parkinson's disease mouse model.
复制标题
基于iPSC衍生的中脑类器官的细胞治疗方法用于恢复帕金森病小鼠模型的运动功能。
DOI：
10.1016/j.heliyon.2024.e24234
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发表时间：
2024-01-30
期刊：
影响因子：
4
通讯作者：
Li, Dan
中科院分区：
文献类型：
--
作者：
Fu, Chong-Lei;Dong, Bo-Cheng;Jiang, Xi;Li, Dan
帕金森病（Parkinson's disease，PD）是一种以黑质多巴胺能（dopaminergic，DA）神经元变性和纹状体DA传递功能丧失为特征的神经退行性疾病，因此细胞移植成为一种有效的治疗策略。在这里，我们开发了一种基于诱导多能干细胞（iPSC）衍生的中脑类器官的细胞疗法。通过将中脑类器官细胞移植到6-OHDA损伤的PD小鼠模型的纹状体区域，我们发现移植的细胞存活并高效地分化为DA神经元。此外，使用多巴胺传感器，我们观察到分化的人DA神经元可以有效地释放多巴胺，并整合到PD小鼠的神经网络中。此外，从移植后4周开始，移植小鼠的运动功能可显著改善。因此，基于iPSC衍生的中脑类器官的细胞疗法可能成为PD临床治疗的潜在策略。
Parkinson's disease (PD) is a neurodegenerative disease characterized by the degeneration of dopaminergic (DA) neurons in the substantia nigra and loss of DA transmission in the striatum, thus making cell transplantation an effective treatment strategy. Here, we develop a cellular therapy based on induced pluripotent stem cell (iPSC)-derived midbrain organoids. By transplanting midbrain organoid cells into the striatum region of a 6-OHDA-lesioned PD mouse model, we found that the transplanted cells survived and highly efficiently differentiated into DA neurons. Further, using a dopamine sensor, we observed that the differentiated human DA neurons could efficiently release dopamine and were integrated into the neural network of the PD mice. Moreover, starting from four weeks after transplantation, the motor function of the transplanted mice could be significantly improved. Therefore, cell therapy based on iPSC-derived midbrain organoids can be a potential strategy for the clinical treatment of PD.
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影响因子：
5.9
作者：
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通讯作者：
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