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2015 Fiscal Year Final Research Report

Study for autophagy in lysosomal diseases and development of drug screening

Research Project

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Project/Area Number 25461543
Research Category

Grant-in-Aid for Scientific Research (C)

Allocation TypeMulti-year Fund
Section一般
Research Field Pediatrics
Research InstitutionOsaka University

Principal Investigator

Sakai Norio  大阪大学, 医学(系)研究科(研究院), 教授 (30314313)

Project Period (FY) 2013-04-01 – 2016-03-31
KeywordsKrabbe disease / galactosialidosis / Fabry disease / autophagy / chaperone
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.

Free Research Field

小児科学

URL: 

Published: 2017-05-10  

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