The technology to eliminate the risk of tumor formation in human iPS cells by inhibiting cholesterol biosynthesis.

2022 Fiscal Year Final Research Report

• Project/Area Number: 20K06661
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 44020:Developmental biology-related
• Research Institution: Kindai University
• Principal Investigator: Okamura Daiji 近畿大学, 農学部, 講師 (80393263)
• Project Period (FY): 2020-04-01 – 2023-03-31
• Keywords: ヒトiPS細胞 / 腫瘍原性 / コレステロール生合成 / アポトーシス

Outline of Final Research Achievements

Tumour formation by residual undifferentiated cells is still a major problem in human iPS cell therapy. We found that the cholesterol biosynthesis inhibitor killed almost all undifferentiated pluripotent stem cells and immature differentiated cells by apoptosis in serum-free medium. More importantly, the survival and function of mature differentiated cells that had stopped proliferating were not affected at all. These results are expected to serve as a basic technology for the "elimination of undifferentiated cells that may become tumours" and the "purification and enrichment of mature differentiated cells", which are the most important issues in transplantation medicine using iPS cells.

Free Research Field

発生生物学

Academic Significance and Societal Importance of the Research Achievements

当該申請技術は未分化iPS細胞の除去作用に加え、分化途上にある未成熟分化細胞も除去可能であり、セルソーティングなどでの物理的な純化や組織・細胞に特化した純化技術を必要とせず、またコレステロールというあらゆる細胞が必要とする構成分子に対する阻害剤であることから、組織・細胞または動物種を問わない極めてユニバーサルな残存する未分化iPS細胞による腫瘍リスク除去技術そして成熟分化細胞の純化技術となる可能性がある。また物理的な純化を必要としないため、目的分化細胞の集合体である既存の「細胞シート（心筋・網膜・角膜など）」に対しても極めて有効に作用する可能性がある。