2012 Fiscal Year Final Research Report
Development of novel therapeutic strategies for neurodegenerative lysosomal storage diseases by ameliorating autophagic degradation and brain-specific peptides
| Project/Area Number |
22390207
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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 |
NANBA Eiji 鳥取大学, 生命機能研究支援センター, 教授 (40237631)
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| Co-Investigator(Kenkyū-buntansha) |
HIGAKI Katsumi 鳥取大学, 生命機能研究支援センター, 准教授 (90294321)
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| Project Period (FY) |
2010 – 2012
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| Keywords | ライソゾーム病 / 中枢神経疾患 / 先天代謝異常症 / オートファジー / ユビキチン / ミトコンドリア |
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| Research Abstract |
Molecular pathophysiology of neurodegenerative lysosomal storage disease ia still largely unclear. In this study, we found impairments of autophagy degradation and mitochondrial function and dysregulation of histone deacetylases in cultured neuronal model of this disease. We also examined the therapeutic effects of inhibitors for autophagy and histone deacetylase in this model cells. The obtained results gave us important insight into the development of novel molecular targeting therapy for neurodegeneration of lysosomal storage diseases in the future.
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[Journal Article] A bicyclic 1-deoxygalactonojirimycin derivative as a novel pharmacological chaperone for GM1 - gangliosidosis2013
Author(s)
Takai T, Higaki K, Aguilar-Moncayo M, Mena-Barragan T, Hirano Y, Yura K, Yu L, Ninomiya H, M. Garcia-Moreno I,Sakakibara Y, Ohno K, Nanba E, Ortiz Mellet C, Jose M. Fernandez G, Suzuki Y
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Journal Title
Mol Ther
Volume: 21
Pages: 526-532
DOI
Peer Reviewed
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[Journal Article] Tuning glycosidase inhibition through aglycone interactions: Pharmacological chaperones for Fabry disease and GM1gangliosidosis2012
Author(s)
Aguilar-Moncayo M, Takai T, Higaki K, Mena-Barragan T, Hirano Y, Yura K, Li L, Yu Y, Ninomiya H, Garcia-Moreno I, Ishii S, Sakakibara Y, Ohno K, Nanba E, Ortiz Mellet C, Garcia Fernandez JM, Suzuki Y:
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Journal Title
DOI
Peer Reviewed
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[Journal Article] Therapeutic chaperone effect of N-octyl 4-epi-β-valienamine on murine G(M1)-gangliosidosis2012
Author(s)
Suzuki Y, Ichinomiya S, Kurosawa M, Matsuda J,Ogawa S, Iida M, Kubo T, Tabe M, Itoh M, Higaki K, Nanba E,Ohno K
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Journal Title
Mol Genet Metab
Volume: 106
DOI
Peer Reviewed
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[Journal Article] Genotype/phenotype of 6 Chinese cases with Niemann-Pick disease type C.2012
Author(s)
Xiong H,Higaki K, Wei C, Bao CW, Zhanf YH, Fu N, Qui J, Adachi K, Kumura Y, Ninomiya H, Nanba E,Wu XR
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Journal Title
Gene
Volume: 498
Pages: 332-335
DOI
Peer Reviewed
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[Journal Article] Chemical chaperone therapy: chaperone effect on mutant enzyme and cellular pathophysiology in β-galactosidase deficiency2011
Author(s)
HigakKLi L,Bahrudin U, Okuzawa S,Takamura A, Yamamoto K, Adachi K, Paraguison RC, 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
Hum Mutat
Volume: 32
Pages: 843-852
DOI
Peer Reviewed
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