2015 Fiscal Year Final Research Report
Study for autophagy in lysosomal diseases and development of drug screening
| Project/Area Number |
25461543
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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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| Research Field |
Pediatrics
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| Research Institution | Osaka University |
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Principal Investigator |
Sakai Norio 大阪大学, 医学(系)研究科(研究院), 教授 (30314313)
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| Project Period (FY) |
2013-04-01 – 2016-03-31
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| Keywords | Krabbe disease / galactosialidosis / Fabry disease / autophagy / chaperone |
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| Outline of Final Research Achievements |
Lysosomal diseases are rare and intractable disease and are targets of Japanese health science. However the treatment method are limited such as enzyme replacement therapy and hematopoietic stem cell transplantation which might has severe adverse effect. In this study we performed basic research for Krabbe disease, galactosialidosis and Fabry disease for the screening the low molecule oral drug candidates. With this research we found several cellular pathology for lysosomal diseases and the possibility of chaperone treatment and basic mechanism of it.
With these results it is applied to check the effect of candidate drug in cellular level and the screen the drug for each mutation of the disease.
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| Free Research Field |
小児科学
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