美国马萨诸塞州总医院Jeffrey S. Schweitzer、Bob S. Carter联合麦克莱恩医院Kwang-Soo Kim团队在研究中取得进展。他们探讨了个性化iPSC衍生的多巴胺能祖细胞治疗帕金森病的效果。该成果发表在2020年5月14日出版的《新英格兰医学杂志》上。

研究组报告了将患者自体诱导的多能干细胞（IPSC）衍生出中脑多巴胺能祖细胞，将其植入原发性帕金森病患者的疗效。患者特异性祖细胞是在良好的生产规范条件下产生的，具有黑质致密部神经元的表型特征；在人源化小鼠模型（涉及外周血单核细胞）中进行的试验表明，这些细胞缺乏免疫原性。

将这些细胞植入到帕金森氏病患者的壳核（先左半球，之后右半球，相隔6个月），无需进行免疫抑制。用氟-18-L-二羟基苯丙氨酸进行正电子发射断层扫描显示移植物存活。植入后18到24个月，帕金森氏病患者的临床症状指标稳定或有所改善。

附：英文原文

Title: Personalized iPSC-Derived Dopamine Progenitor Cells for Parkinson’s Disease

Author: Jeffrey S. Schweitzer, M.D., Ph.D.,, Bin Song, M.D., Ph.D.,, Todd M. Herrington, M.D., Ph.D.,, Tae-Yoon Park, Ph.D.,, Nayeon Lee, Ph.D.,, Sanghyeok Ko, Ph.D.,, Jeha Jeon, Ph.D.,, Young Cha, Ph.D.,, Kyungsang Kim, Ph.D.,, Quanzheng Li, Ph.D.,, Claire Henchcliffe, M.D., D.Phil.,, Michael Kaplitt, M.D., Ph.D.,, Carolyn Neff, M.D.,, Otto Rapalino, M.D.,, Hyemyung Seo, Ph.D.,, In-Hee Lee, Ph.D.,, Jisun Kim, Ph.D.,, Taewoo Kim, Ph.D.,, Gregory A. Petsko, D.Phil.,, Jerome Ritz, M.D.,, Bruce M. Cohen, M.D., Ph.D.,, Sek-Won Kong, M.D.,, Pierre Leblanc, Ph.D.,, Bob S. Carter, M.D., Ph.D.,, and Kwang-Soo Kim, Ph.D.

Issue&Volume: 2020-05-13

Abstract: Summary 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.

DOI: 10.1056/NEJMoa1915872

Source: https://www.nejm.org/doi/full/10.1056/NEJMoa1915872