2023 Fiscal Year Annual Research Report
Unleashing the power of AI to create a novel treatment for hemophilia A
| Project/Area Number |
22K06119
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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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| Keywords | Hemophilia A / Artificial intelligence / Machine learning / Bioinformatics / Protein design |
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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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