Basic research for clinical treatment of neuropathic Gaucher disease by chemical chhaperones
| Project/Area Number |
20390297
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Research Field |
Pediatrics
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| Research Institution | Tottori University |
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Principal Investigator |
OHNO Kousaku Tottori University, 医学部, 教授 (70112109)
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| Co-Investigator(Renkei-kenkyūsha) |
SUGIURA Titose 鳥取大学, 医学部・附属病院, 講師 (90325027)
INOUE Takehiko 鳥取大学, 医学部・附属病院, 助教 (80346361)
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| Project Period (FY) |
2008 – 2010
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| Project Status |
Completed (Fiscal Year 2010)
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| Budget Amount *help |
¥18,720,000 (Direct Cost: ¥14,400,000、Indirect Cost: ¥4,320,000)
Fiscal Year 2010: ¥5,720,000 (Direct Cost: ¥4,400,000、Indirect Cost: ¥1,320,000)
Fiscal Year 2009: ¥5,070,000 (Direct Cost: ¥3,900,000、Indirect Cost: ¥1,170,000)
Fiscal Year 2008: ¥7,930,000 (Direct Cost: ¥6,100,000、Indirect Cost: ¥1,830,000)
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| Keywords | ゴーシェ病 / シャペロン / 経口治療薬 / モデルマウス / リソゾーム |
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| Research Abstract |
There are no effective treatment for neurological manifestations in Gaucher disease. We found N-octyl-β-valienamine (NOV) could increase the protein level and enzyme activity of F213I mutant GBA in culture cells. We have tried to establish a model mouse expressing F213I protein, but failed to establish the mice. The NOV effects on normal mice were evaluated. No adverse effect was observed and GBA activity increased with increasing NOV dose in tissues. We also found new compounds, bicyclic nojirimycin analogues, show a chemical chaperone activity on mutant GBAs.
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Report
(4 results)
Research Products
(30 results)
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[Journal Article] Chemical chaperone therapy : chaperone effect on mutant enzyme and cellular pathophysiology in β-galactosidase deficiency.2011
Author(s)
Higaki K, Li L, Udin Bahrudin, Okuzawa S, Takamura A, Yamamoto K, Adachi K, Rubigilda C. Paraguison, Takai T, Ikehata H, Tominaga L, Hisatome I, Iida M, Ogawa S, Matsuda J, Ninomiya H, Sakakibara Y, Ohno K, Suzuki Y, Nanba E.
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Journal Title
Related Report
Peer Reviewed
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[Journal Article] A fluorescent SP2-iminosugar with pharmacological chaperone activity for Gaucher disease : Cellular uptake and intracellular distribution studies.2010
Author(s)
Luan Z, Higaki K, Aguilar-Moncayo M, Li L, Ninomiya H, Nanba E, Ohno K, Garcia-Moreno M, Mellet CO, Fernandez JMG, Suzuki Y.
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Journal Title
ChemBioChem 11(17)
Pages: 2453-2464
Related Report
Peer Reviewed
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