| Budget Amount *help |
¥2,990,000 (Direct Cost: ¥2,300,000、Indirect Cost: ¥690,000)
Fiscal Year 2015: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2014: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
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| Outline of Final Research Achievements |
Polyglutamine disease is a Hereditary neurodegenerative disease caused by a CAG repeat expansion in protein-coding portions of specific genes. Expanded CAG repeats are translated into a series of glutamine residues (polyQ) and are subject to increased aggregation. We previously identified a small compound (F7) that digests polyQ proteins.
In this study, we identified the target molecule for F7 chemical. The lack of this molecule increased polyQ accumulation, and its overexpression decreased it. We also indetified the involvement of autophagy machinery in the pharmacological effect of F7 chemical. We confirmed that F7 chemical improved the neurodegeneration observed in polyQ model mice.
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