Development of an analytical platform for novel brain glycosylated sterols toward applying for the treatment of neurodegenerative diseases

2019 Fiscal Year Final Research Report

• Project/Area Number: 17K15608
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: General medical chemistry
• Research Institution: Institute of Physical and Chemical Research
• Principal Investigator: Akiyama Hisako 国立研究開発法人理化学研究所, 脳神経科学研究センター, 研究員 (80623462)
• Project Period (FY): 2017-04-01 – 2020-03-31
• Keywords: 糖脂質 / 糖化ステロール / グルコシルセラミド分解酵素

Outline of Final Research Achievements

I developed an analytical method specific to novel glycosylated sterols that we discovered in the brain, and analyzed the metabolic pathways and physiological functions of the glycosylated sterols. The results showed that the metabolism of the glycosylated sterols is mediated by glucosylceramide-degrading enzymes (GBA). In addition, the expression of glycosylated sterols was altered in the brains of mouse model of Parkinson's disease with impaired GBA function.

Free Research Field

脂質生化学

Academic Significance and Societal Importance of the Research Achievements

脳内新規糖化ステロール群の代謝を担うことが明らかになったグルコシルセラミド分解酵素は、その機能不全がパーキンソン病や小脳失調などの神経変性疾患の原因となる。本研究で開発した分析法を用いることで、糖化ステロール代謝が難治性神経変性疾患発症の新たな分子基盤となり得る可能性を検証できるようになり、本研究成果の学術的・社会的意義は大きい。