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采用iPSC来源的多巴胺祖细胞进行的帕金森病个体化治疗
Personalized iPSC-derived Dopamine Progenitor Cells for Parkinson’s Disease


Jeffrey S. Schweitzer ... 其他 • 2020.05.14
相关阅读
• 人干细胞疗法在有帕金森病的猴中取得成功 • 干细胞在疾病治疗中的应用 • 澄清干细胞疗法的获益与风险

摘要


本文报告了为一例特发性帕金森病患者植入患者来源的中脑多巴胺能祖细胞的情况,植入的祖细胞是由自体诱导多能干细胞(iPSC)体外分化而来。患者特异性祖细胞是在符合《药品生产质量管理规范》的条件下制备,其特征是具有黑质致密部神经元的表型特征,在人源化小鼠模型(包括外周血单核细胞)中进行的试验表明这些细胞不具有免疫原性。这些细胞被植入一例帕金森病患者的壳核(先左半球,后右半球,间隔6个月)内,不需要免疫抑制。使用氟-18-L-二羟基苯丙氨酸进行的正电子发射断层扫描显示移植物存活。植入后18~24个月时,术后帕金森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)





作者信息

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.
From the Departments of Neurosurgery (J.S.S., B.S.C.), Neurology (T.M.H.), and Radiology (K.K., Q.L.), the Gordon Center for Medical Imaging (K.K., Q.L.), and the Division of Neuroradiology (O.R.), Massachusetts General Hospital, the Department of Pediatrics, Computational Health Informatics Program, Boston Children’s Hospital (I.-H.L., S.-W.K.), and the Connell and O’Reilly Families Cell Manipulation Core Facility, Dana–Farber/Harvard Cancer Center (J.R.), Boston, and the Department of Psychiatry (B.M.C.) and the Molecular Neurobiology Laboratory (B.S., T.-Y.P., N.L., S.K., J.J., Y.C., H.S., J.K., T.K., P.L., K.-S.K.), McLean Hospital, Belmont — all in Massachusetts; the Departments of Neurology (C.H.) and Neurosurgery (M.K.) and the Brain and Mind Research Institute (G.A.P.), Weill Cornell Medical College, New York; the Department of Neurology, Kaiser Permanente, Irvine, CA (C.N.); and the Department of Molecular and Life Sciences, Hanyang University, Seoul, South Korea (H.S.). Address reprint requests to Dr. Schweitzer or Dr. Carter at the Department of Neurosurgery, Massachusetts General Hospital, 55 Fruit St., White Bldg., Rm. 502, Boston, MA 02114, or at jschweitzer1@mgh.harvard.edu or bcarter@mgh.harvard.edu; or to Dr. Kwang-Soo Kim at the Molecular Neurobiology Laboratory, Rm. 216, McLean Hospital, 115 Mill St., Belmont, MA 02478, or at kskim@mclean.harvard.edu.

 

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