| Budget Amount *help |
¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000)
Fiscal Year 2017: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2016: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2015: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
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| Outline of Final Research Achievements |
Pharmacological chaperone therapy using the small molecule compound is promising therapy for neuropathic lysosomal storage diseases including Gaucher's disease.
We developed the pH-responsive pharmacological chaperones which is expected to obtain more high concentration of blood and cerebrospinal fluid.The chaperone effect of this compound showed a wider spectrum as compared with other chaperone compounds such as ambroxol. Also, combination of chaperone and exogenous enzyme showed synergic effect.
Additionally, secondary mitochondrial dysfunction was improved by the administration of this compound.
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