| Project/Area Number |
22K06119
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Review Section |
Basic Section 43020:Structural biochemistry-related
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| Research Institution | National Center for Child Health and Development |
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Principal Investigator |
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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| Project Period (FY) |
2022-04-01 – 2024-03-31
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| Project Status |
Discontinued (Fiscal Year 2023)
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| Budget Amount *help |
¥4,290,000 (Direct Cost: ¥3,300,000、Indirect Cost: ¥990,000)
Fiscal Year 2024: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2023: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2022: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
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| Keywords | Hemophilia A / Artificial intelligence / Machine learning / Bioinformatics / Protein design / Hemophilia |
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| Outline of Research at the Start |
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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| Outline of Annual Research Achievements |
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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