Fine-tuning of polymer amphiphile for high performance of messenger RNA delivery
| Project/Area Number |
18K18378
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| Research Category |
Grant-in-Aid for Early-Career Scientists
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| Allocation Type | Multi-year Fund |
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| Review Section |
Basic Section 90120:Biomaterials-related
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| Research Institution | The University of Tokyo |
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Principal Investigator |
Kim HyunJin 東京大学, 大学院医学系研究科(医学部), 特任助教 (10755002)
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| Project Period (FY) |
2018-04-01 – 2020-03-31
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| Project Status |
Completed (Fiscal Year 2019)
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| Budget Amount *help |
¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2019: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
Fiscal Year 2018: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
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| Keywords | カチオン性ポリマー / mRNA 配送 / CRISPR-Casテクノロジー / cationic polymer / mRNA transfection / amphiphile / CRISPR-Cas technology |
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| Outline of Final Research Achievements |
For broad application of mRNA therapeutics, this proposal newly developed cationic amphiphilic polymers. The polymers consist of endosome escapable moiety and hydrophobic moiety. The hydrophobic moiety provides the balance between mRNA-loaded nanoparticle stability in the extracellular environment and rapid release of mRNA in the cytoplasm. The best polymers showed better luciferase and Cas9 mRNA translation efficacy than other control polymers and commercially-available lipofectamine in cultured cell condition. The polymers also induced high firefly mRNA translation in the mouse brain when intracerebroventricularly injected.
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| Academic Significance and Societal Importance of the Research Achievements |
科学的な観点から、この研究はまず、logPに基づく疎水性最適化がポリマーの性能を大幅に向上させることを示唆している。一方、これらのポリマーは、その高い性能により、現在、日本および国際特許に出願されている。これらのポリマーは、日本と米国の2つのベンチャー企業からもライセンス供与された。この研究がCRISPR-Cas9テクノロジーを使用して一部の遺伝子疾患治療に引き続き適用される場合、この提案は特定の患者に多大な利益をもたらすでしょう。
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Report
(3 results)
Research Products
(11 results)
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[Journal Article] Glucose-linked sub-50-nm unimer polyion complex-assembled gold nanoparticles for targeted siRNA delivery to glucose transporter 1-overexpressing breast cancer stem-like cells.2019
Author(s)
Y. Yi, H. J. Kim, M. Zheng, P. Mi, M. Naito, B. S. Kim, H. S. Min, K. Hayashi, F. Perche, K. Toh, X. Liu, Y. Mochida, H. Kinoh, H. Cabral, K. Miyata, K. Kataoka
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Journal Title
J. Control. Release
Volume: 295
Pages: 268-277
DOI
Related Report
Peer Reviewed / Int'l Joint Research
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