Development of mRNA stabilization technology for mRNA therapeutics

2018 Fiscal Year Final Research Report

• Project/Area Number: 17K19357
• Research Category: Grant-in-Aid for Challenging Research (Exploratory)
• Allocation Type: Multi-year Fund
• Research Field: Molecular and Genome biology and related fields
• Research Institution: Nagoya City University
• Principal Investigator: Hoshino Shin-ichi 名古屋市立大学, 大学院薬学研究科, 教授 (40219168)
• Co-Investigator(Kenkyū-buntansha): 細田 直 名古屋市立大学, 大学院薬学研究科, 講師 (40438198)
• Project Period (FY): 2017-06-30 – 2019-03-31
• Keywords: 人工mRNA / mRNA医薬

Outline of Final Research Achievements

Much attention has been focused on the mRNA-based therapeutics as an alternative to the DNA-based therapeutics, since mRNA can be used for generation of induced pluripotent stem (iPS) cells, cancer therapy etc. without the risk of transgene insertion into genomes. However, it remains a critical issue that mRNA is unstable and transient in cells. Here, we developed a method stabilizing synthetic mRNA in cells and found our methodology to be useful especially for genome editing in cells.

Free Research Field

分子生物学

Academic Significance and Societal Importance of the Research Achievements

遺伝子治療にはこれまでDNAを用いた治療が行われてきたが、ゲノムへの挿入による発がん等の危険性が問題視されてきた。本研究では、発がん等の危険性のない安全な医薬のとして期待されるmRNAの欠点であった不安定性を克服するmRNA安定化技術を開発し、ゲノム編集における有効性を確認できたことで、今後遺伝子治療をはじめ、iPS細胞の作製やがん免疫療法などへも広く応用できる可能性を示した。