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2022 Fiscal Year Final Research Report

Development of RNA replicon-loaded micelles for in situ preparation of iPS cells

Research Project

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Project/Area Number 21K18070
Research Category

Grant-in-Aid for Early-Career Scientists

Allocation TypeMulti-year Fund
Review Section Basic Section 90120:Biomaterials-related
Research InstitutionKanagawa Institute of Industrial Sclence and Technology

Principal Investigator

Miyazaki Takuya  地方独立行政法人神奈川県立産業技術総合研究所, 「貼るだけ人工膵臓」プロジェクト (松元P), 非常勤研究員 (80844779)

Project Period (FY) 2021-04-01 – 2023-03-31
Keywordsブロック共重合体 / 高分子ミセル / RNAレプリコン / 遺伝子治療 / 山中因子 / iPS細胞 / ドラッグデリバリーシステム / 核酸医薬
Outline of Final Research Achievements

In situ preparation of iPS cells is attractive approach while its in vivo applications have been hampered by low safety and efficacy. Thus, we have developed replicon-loaded polymeric micelles by engineering polycation to protect RNA payload and RNA to avoid genome insertion. Moreover, we have introduced functional amino acids as pendants in the polymers to promote accumulation in cytosol and to accelerate production of Yamanaka factors. In this study, we have developed polymeric micelles translating Yamanaka factors in vivo with high safety and efficacy aimed to in situ production of iPS cells.

Free Research Field

生体医工学

Academic Significance and Societal Importance of the Research Achievements

iPS細胞を用いた細胞治療は難治性疾患の新規治療法として注目されているが、患者由来細胞の摘出や山中因子の導入、iPS細胞の単離といった煩雑な工程が患者様ごとに必要となる。そこで、本研究では、1本の注射剤で安全かつ効率的にiPS細胞を産出する方法を確立した。本研究で得られた成果を特定の疾患に応用することで、臨床応用に向けた開発を進めることができると考えられる。

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Published: 2024-01-30  

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