| Literature DB >> 32632153 |
Daisuke Doi1, Hiroaki Magotani1,2, Tetsuhiro Kikuchi1, Megumi Ikeda1,3, Satoe Hiramatsu1,3, Kenji Yoshida1,3, Naoki Amano4, Masaki Nomura4, Masafumi Umekage4, Asuka Morizane1, Jun Takahashi5.
Abstract
Induced pluripotent stem cell (iPSC)-derived dopaminergic (DA) neurons are an expected source for cell-based therapies for Parkinson's disease (PD). The regulatory criteria for the clinical application of these therapies, however, have not been established. Here we show the results of our pre-clinical study, in which we evaluate the safety and efficacy of dopaminergic progenitors (DAPs) derived from a clinical-grade human iPSC line. We confirm the characteristics of DAPs by in vitro analyses. We also verify that the DAP population include no residual undifferentiated iPSCs or early neural stem cells and have no genetic aberration in cancer-related genes. Furthermore, in vivo studies using immunodeficient mice reveal no tumorigenicity or toxicity of the cells. When the DAPs are transplanted into the striatum of 6-OHDA-lesioned rats, the animals show behavioral improvement. Based on these results, we started a clinical trial to treat PD patients in 2018.Entities:
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Year: 2020 PMID： 32632153 PMCID： PMC7338530 DOI： 10.1038/s41467-020-17165-w
Source DB: PubMed Journal: Nat Commun ISSN： 2041-1723 Impact factor: 14.919