We report the implantation of patient-derived midbrain dopaminergic progenitor cells, differentiated in vitro from autologous induced pluripotent stem cells (iPSCs), in a patient with idiopathic Parkinson's disease. The patient-specific progenitor cells were produced under Good Manufacturing Practice conditions and characterized as having the phenotypic properties of substantia nigra pars compacta neurons; testing in a humanized mouse model (involving peripheral-blood mononuclear cells) indicated an absence of immunogenicity to these cells. The cells were implanted into the putamen (left hemisphere followed by right hemisphere, 6 months apart) of a patient with Parkinson's disease, without the need for immunosuppression. Positron-emission tomography with the use of fluorine-18-L-dihydroxyphenylalanine suggested graft survival. Clinical measures of symptoms of Parkinson's disease after surgery stabilized or improved at 18 to 24 months after implantation. (Funded by the National Institutes of Health and others.).

中文翻译：

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用于帕金森氏病的个性化iPSC衍生的多巴胺祖细胞。

我们报告患有特发性帕金森病的患者植入患者源性中脑多巴胺能祖细胞，与自体诱导多能干细胞（iPSCs）体外分化。患者特异性祖细胞是在良好生产规范条件下生产的，其特征在于具有黑质致密部神经元的表型特性；在人源化小鼠模型（涉及外周血单核细胞）中进行的测试表明，这些细胞缺乏免疫原性。将细胞植入帕金森氏病患者的壳（左半球，然后是右半球，相距6个月），而无需进行免疫抑制。使用氟-18-L-二羟基苯丙氨酸进行正电子发射断层扫描提示移植物存活。植入后18至24个月后，帕金森氏病症状的临床指标稳定或改善。（由美国国立卫生研究院等资助）。