2021 Fiscal Year Final Research Report
Development of Boronic Acid Derivatives for the Treatment of the Lysosomal Storage Diseases Caused by Mutations in the Sulfatases
| Project/Area Number |
19K16319
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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 47010:Pharmaceutical chemistry and drug development sciences-related
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| Research Institution | Kitasato University |
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Principal Investigator |
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| Project Period (FY) |
2019-04-01 – 2022-03-31
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| Keywords | スルファターゼ / ファーマコロジカルシャペロン / ライソゾーム病 / ボロン酸 / ムコ多糖症 / シャペロン化合物 |
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| Outline of Final Research Achievements |
In human cells, there is a family of enzymes named sulfatases, which degrade the waste products in the cells. Mutations of the genes can induce loss of activities of the sulfatases, which causes storage of the waste products, malfunction of the cells, and finally death of the affected individuals. In this study, we set out to develop compounds that bind to the abnormal sulfatases and restore the activity of them, and treat the diseases caused by mutations of the sulfatases. In our preliminary study, it was suggested that boronic acids bind to the sulfatases. Thus, we synthesized various derivatives of boronic acids, and tested if they bind to the sulfatases.
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| Free Research Field |
創薬化学 (ドラッグライクネス, 希少疾病), ケミカルバイオロジー
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| Academic Significance and Societal Importance of the Research Achievements |
スルファターゼの異常に起因する疾患の一部では, 骨髄移植やスルファターゼを体外から投与する方法 (酵素補充療法)による治療が行われている. しかし, 前者は患者の身体的負担が非常に大きく, 後者では酵素を補充するのに要する時間や酵素の費用が負担となる. もし, スルファターゼの異常に起因する疾患を一般的な飲み薬で治療できるようになれば, 上述した負担をなくすことができる. 本研究では, そのような薬を創製する上で必要な情報を取得できた. したがって, 将来的な薬の開発につながり得るという意義があると言える.
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