Elucidation of the ALS/FTLD pathomechanism mediated by the dysfunction of ribosome biogenesis
| Project/Area Number |
18K06705
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Review Section |
Basic Section 47040:Pharmacology-related
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| Research Institution | Tokyo Medical University |
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Principal Investigator |
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| Project Period (FY) |
2018-04-01 – 2021-03-31
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| Project Status |
Completed (Fiscal Year 2020)
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| Budget Amount *help |
¥4,420,000 (Direct Cost: ¥3,400,000、Indirect Cost: ¥1,020,000)
Fiscal Year 2020: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2019: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2018: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
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| Keywords | 筋萎縮性側索硬化症 / 前頭側頭葉変性症 / C9orf72遺伝子変異 / 神経変性疾患 / 前頭側頭型認知症 / 神経細胞死 / RNA編集 / ADAR / C9ORF72遺伝子変異 / パラスペックル / NEAT1 / リボソーム生合成 / RNAヘリカーゼ / C9ORF72 / リボソーム |
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| Outline of Final Research Achievements |
I investigated the pathomechanism underlying C9orf72-linked amyotrophic lateral sclerosis and frontotemporal lobar degeneration. In the current study, I found that poly-PR, a dipeptide repeat protein derived from C9orf72 mutation, causes neuronal cell death by inhibiting the ribosome biogenesis, paraspeckles, and RNA editing.
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| Academic Significance and Societal Importance of the Research Achievements |
ALS/FTLDの発症メカニズムを両疾患において最も高頻度に認められるC9orf72遺伝子変異に着目し解析した。本研究の遂行により、複数の神経細胞死メカニズムが明らかになり、本研究の成果は両疾患の理解を深めると共に、新たな創薬ターゲットの同定に結びつくものであり、超高齢化社会を迎え、両疾患の根本的治療薬が望まれる我が国において、医学的社会的意義のある成果が得られた。
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Report
(4 results)
Research Products
(8 results)
