| 研究課題/領域番号 |
22K06119
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| 研究種目 |
基盤研究(C)
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| 配分区分 | 基金 |
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| 応募区分 | 一般 |
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| 審査区分 |
小区分43020:構造生物化学関連
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| 研究機関 | 国立研究開発法人国立成育医療研究センター |
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研究代表者 |
daSilvaLopes TiagoJose 国立研究開発法人国立成育医療研究センター, 細胞医療研究部, 専門職 (10903429)
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| 研究期間 (年度) |
2022-04-01 – 2024-03-31
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| 研究課題ステータス |
中途終了 (2023年度)
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| 配分額 *注記 |
4,290千円 (直接経費: 3,300千円、間接経費: 990千円)
2024年度: 1,300千円 (直接経費: 1,000千円、間接経費: 300千円)
2023年度: 1,300千円 (直接経費: 1,000千円、間接経費: 300千円)
2022年度: 1,690千円 (直接経費: 1,300千円、間接経費: 390千円)
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| キーワード | Hemophilia A / Artificial intelligence / Machine learning / Bioinformatics / Protein design / Hemophilia |
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| 研究開始時の研究の概要 |
In 2021 we established artificial intelligence (AI) methods to study the properties of the protein causing hemophilia A - a rare disease caused by defects in the FVIII protein and that impairs the blood coagulation. We observed a remarkable agreement between our results and hundreds of in vitro and clinical reports. Therefore, we are designing a novel FVIII-like protein that can be produced in non-mammalian cell lines and is invisible to the immune system. In all, we are creating the next generation of therapeutics and overcoming the two major burdens of actual hemophilia treatments.
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| 研究実績の概要 |
This project demonstrated the viability of using artificial intelligence to design therapeutic proteins for a rare disease. We used the latest methods to generate almost 200 candidate molecules, that are now undergoing in vitro testing by a collaborator at the Jichi Medical University Hospital. This number consists a dramatic reduction in the number of candidates that is traditionally tested in pre-clinical studies.
Importantly, we generated our therapeutic proteins using a technique called cell-free expression, which does not involve the use of any bacteria or mammalian cell lines; this further reduced the costs and labor involved in the process.
Overall, we are satisfied with the progress of this project, the conscious use of resources and the training of younger researchers.
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