Analysis of retinoblastoma using human iPS cells

2018 Fiscal Year Final Research Report

• Project/Area Number: 17K19583
• Research Category: Grant-in-Aid for Challenging Research (Exploratory)
• Allocation Type: Multi-year Fund
• Research Field: Tumor biology and related fields
• Research Institution: The University of Tokyo
• Principal Investigator: Watanabe Sumiko 東京大学, 医科学研究所, 特任教授 (60240735)
• Project Period (FY): 2017-06-30 – 2019-03-31
• Keywords: 網膜芽細胞腫 / 網膜 / iPS / CRISPR/Cas9

Outline of Final Research Achievements

Retinoblastoma is a retinal cancer of young children that is caused by the mutation of Rb1 gene. Mutation of Rb1 gene is observed many other cancers, but why retina is primary target of the mutation is not well understood. To approach this question, we used human iPS~retinal organoid model. We found the expression level of RB1 is constantly increased when human iPS was cultured to for retinal organoid. RB1 expression was suppressed by CRIAPR/Cas9 mediated technology, and proliferation activity of immature RB1-minus human iPS was not affected. Early differentiation stage toward the retina of the RB1-minus human iPS cell was also not affected. We continue to analyze differentiation and proliferation activity of the cells.

Free Research Field

分子生物学

Academic Significance and Societal Importance of the Research Achievements

網膜芽細胞腫(Retinoblastoma)は、RB1遺伝子が原因遺伝子として知られながら、その発がんメカニズムはマウスの系を用いることができないため、いまだに大きな謎となっていた。今回、human iPSを用いることで、RB1遺伝子の変異がどうして、網膜で特に高い腫瘍誘導性を示す糸口が示され、human iPSの病態解析への利用、創薬研究、RB1研究と言った多方面に重要な知見や技術の提供が期待される。