2023 Fiscal Year Final Research Report
Elucidation of the molecular basis of novel ribitol phosphate modifications and their application to the development of treatments for muscular dystrophy
| Project/Area Number |
21H02685
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Review Section |
Basic Section 48040:Medical biochemistry-related
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| Research Institution | Ehime University |
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Principal Investigator |
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| Co-Investigator(Kenkyū-buntansha) |
萬谷 博 地方独立行政法人東京都健康長寿医療センター(東京都健康長寿医療センター研究所), 東京都健康長寿医療センター研究所, 研究部長 (20321870)
永森 收志 東京慈恵会医科大学, 医学部, 教授 (90467572)
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| Project Period (FY) |
2021-04-01 – 2024-03-31
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| Keywords | 筋ジストロフィー / 糖鎖 |
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| Outline of Final Research Achievements |
Ribitol phosphate, a novel post-translational moiety, plays important roles in skeletal muscle and nerve tissues, and its abnormalities lead to muscular dystrophy and lissencephaly. The biosynthetic pathway of CDP-ribitol, a precursor required for ribitol phosphate modification, and the detailed mechanism of ribitol phosphate modification are unknown, and there is no effective treatment for ribitol phosphate-defective diseases. In this study, we elucidated the biosynthetic mechanism of CDP-ribitol and proposed an enzyme product supplementation therapy based on the pathogenesis.
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| Free Research Field |
医化学
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| Academic Significance and Societal Importance of the Research Achievements |
リビトールリン酸修飾の分子機構に不明な点は多く、また、リビトールリン酸異常症に有効な治療法は存在しない。本研究では、リビトールリン酸修飾に重要なCDP-リビトールの合成経路が明らかになり、発症メカニズムに基づいた治療戦略の提唱にも至った。これらの成果は、疾患機序の解明や治療法の開発につながり、学術的・社会的にも大きな貢献を果たした。
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