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聊聊Sci

189-人胚干细胞治疗帕金森病1/2a期临床试验

29 Oct 2025

Description

这篇文章详细介绍了一项针对帕金森病(PD)患者进行的人胚胎干细胞(hESC)衍生多巴胺祖细胞(A9-DPC)移植的单中心、开放标签、剂量递增的I/IIa期临床试验。研究的主要发现是,将高纯度A9-DPC移植到双侧壳核是安全且耐受性良好的,在12个月的随访期内没有出现肿瘤形成或细胞过度生长等严重不良事件。探索性疗效结果表明,运动症状有所改善,尤其是在高剂量组,这与多巴胺转运蛋白(DAT)结合增加的PET成像结果相一致,表明移植物存活并具有功能整合的潜力。尽管样本量较小且缺乏对照组,研究者认为这些结果支持细胞替代疗法作为PD的潜在治疗方法,并呼吁未来进行更大规模、双盲的对照试验。References: Chang, Jin Woo, et al. "Phase 1/2a clinical trial of hESC-derived dopamine progenitors in Parkinson’s disease." Cell (2025).

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