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Integrated Symposium of Basic and Clinical Neuroscience:Inducued pluripotent stem (iPS) cells: from basic research to clinical application

開催日 2014/9/11
時間 9:00 - 11:00
会場 Room A(Main Hall)
Chairperson(s) 髙橋 良輔 / Ryosuke Takahashi (京都大学大学院医学研究科・臨床神経学 / Department of Neurology, Kyoto University Graduate School of Medicine, Japan)
岡野 栄之

Challenges towards stem cell therapy for Parkinson's disease

  • S1-A-1-3
  • 高橋 淳 / Jun Takahashi:1 
  • 1:京都大学 iPS細胞研究所 / Center for iPS Cell Research and Application, Kyoto University, Japan 

Human embryonic stem cells (ESCs) and induced pluripotent stem cells (iPSCs) can provide a promising source of midbrain dopaminergic (DA) neurons for cell replacement therapy for Parkinson's disease (PD). To evaluate safety and efficacy of the human ESC-derived DA neurons, we induced neural progenitor cells from human ESCs by a modified SDIA (stromal cell-derived inducing activity) method. When the cells were transplanted into the bilateral striatum of monkey models of PD, they did not form tumors and survived as DA neurons as long as 12 months proved by immunofluorescence and PET studies. In addition, the monkeys showed behavioral improvement after 3 months post-transplantation. We also generated DA neurons from human induced pluripotent stem cells (iPSCs) without feeder cells, and confirmed that these cells could survive as long as 6 months in the monkey brain. These results support the idea that human ESCs/iPSCs can be used as a source for cell replacement therapy of PD. However, ESC/iPSC-derived donor cells may inevitably contain tumorigenic or inappropriate cells. Therefore, as a next step, we have developed a method for 1) scalable DA neuron induction on human laminin fragment and 2) sorting DA progenitor cells using a floor plate marker. The sorting of DA progenitor cells is favorable in terms of both safety and efficacy of the transplantation, and we have now established a protocol for the clinical application of human iPSCs to treat Parkinson's disease.

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