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2019 Fiscal Year Final Research Report

Development of a strategy of drug development for ABC transporter-related diseases

Research Project

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Project/Area Number 17H04007
Research Category

Grant-in-Aid for Scientific Research (B)

Allocation TypeSingle-year Grants
Section一般
Research Field Medical pharmacy
Research InstitutionThe University of Tokyo

Principal Investigator

Hayashi Hisamitsu  東京大学, 大学院薬学系研究科(薬学部), 助教 (10451858)

Project Period (FY) 2017-04-01 – 2020-03-31
Keywords輸送担体 / トランスポーター / 創薬 / タンパク質分解
Outline of Final Research Achievements

To establish a strategy of drug development for ABC transporter-related diseases, we analyzed molecular mechanism underlying degradation mechanism of cell surface-resident ABCA1, an ABC transporter that has an inhibitory effect on atherosclerosis.
This is based on our basic and clinical evidence obtained from studies of other ABC transporters. We have performed library screening, interactome analysis, and subsequent verification experiments using cultured cells and genetically modified mice and
identified multiple molecules that promote the degradation of cell surface-resident ABCA1.

Free Research Field

医療系薬学

Academic Significance and Societal Importance of the Research Achievements

生活習慣病から希少難病に至るまで、細胞膜に発現するABC輸送体の活性化により、治療が実現すると考えられる治療法未確立の疾患が明らかにされている。しかしながら、生体内でABC輸送体を活性化する方法論が未確立であるため、当該疾患群に対する医薬品開発は困難を極めている。本研究成果は、当該疾患群を対象とした医薬品開発の基盤構築に資する。

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Published: 2021-02-19  

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