Implication of Synoviolin on Limb-Girdles Muscle Dystrophy.
| Project/Area Number |
15K15091
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Pathological medical chemistry
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| Research Institution | Tokyo Medical University |
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Principal Investigator |
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| Project Period (FY) |
2015-04-01 – 2017-03-31
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| Project Status |
Completed (Fiscal Year 2016)
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| Budget Amount *help |
¥3,510,000 (Direct Cost: ¥2,700,000、Indirect Cost: ¥810,000)
Fiscal Year 2016: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2015: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
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| Keywords | 分子病理学 / 筋ジストロフィー / 創薬開発 / タンパク質分解系 / ユビキチン / 分子病態学 |
|---|
| Outline of Final Research Achievements |
The functional discrepancy in missense mutation between in vitro and in vivo remains to be uncovered. To explain this observation, protein degradation system has been interested. For example, type2 Limb-Girdle Muscle dystrophy is known to be caused by missense mutant of α-sarcoglycan gene. The missense mutant type protein has approximately 30% of residual activity in vitro but the protein is not detected in cell, and Synoviolin plays a crucial role in the degradation of missense α-sarcoglycan protein.
To develop the innovative therapy, we attempted to understand the physiological and pathological role of Synoviolin. By using muscle-specific its knock out mice, we found the gene plays an important role in muscle. We previously developed Synoviolin inhibitor and this inhibitor had protective effect on muscle atrophy model. These two data clearly indicate Synoviolin as a therapeutic target of muscle diseases including Limb-Girdle muscle dystrophy and muscle atrophy.
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Report
(3 results)
Research Products
(26 results)
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[Journal Article] Notch2 signaling regulates the proliferation of murine bone marrow-derived mesenchymal stem/stromal cells via c-Myc expression.2016
Author(s)
Yukio S, Mabuchi Y, Miyamoto K, Araki D, Niibe K, Houlihan DD, Morikawa S, Nakagawa T, Nakajima T, Akazawa C, Hori S, Okano H, Matsuzaki Y
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Journal Title
PLOS ONE
Volume: 11（11）
Issue: 11
Pages: 0165946-0165946
DOI
Related Report
Peer Reviewed / Open Access / Int'l Joint Research
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[Journal Article] The E3 ligase synoviolin controls body weight and mitochondrial biogenesis through negative regulation of PGC-1β.2015
Author(s)
Fujita H, Yagishita N, Aratani S, Saito-Fujita T, Morota S, Yamano Y, Hansson MJ, Inazu M, Kokuba H, Sudo K, Sato E, Kawahara K, Nakajima F, Hasegawa D, Higuchi I, Sato T, Araya N, Usui C, Nishioka K, Nakatani Y, Maruyama I, Usui M, Hara N, Uchino H, Elmer E, Nishioka K, Nakajima T.
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Journal Title
EMBO J.
Volume: 34
Issue: 8
Pages: 1042-55
DOI
Related Report
Peer Reviewed / Open Access
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[Presentation] シノビオリン阻害剤の探索。2016
Author(s)
藤田英俊、荒谷聡子、西岡久寿樹、中島利博
Organizer
日本線維筋痛症学会第 8 回学術集会
Place of Presentation
KFC Hall & Rooms(東京 墨田区)
Year and Date
2016-09-17
Related Report
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