| Budget Amount *help |
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2019: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2018: ¥1,950,000 (Direct Cost: ¥1,500,000、Indirect Cost: ¥450,000)
Fiscal Year 2017: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
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| Outline of Final Research Achievements |
This study is aimed to elucidate the action mechanism of small molecules that alleviate disease phenotype in spinocerebellar ataxia type 31 (SCA31) model. In this study, we found naphthyridine carbamate dimer (NCD) as small molecules targeting UGGAA repeat in SCA31. NCD inhibited the interaction of UGGAA repeats with RNA-binding proteins and the formation of nuclear RNA foci consisting of UGGAA repeat. We demonstrated that NCD alleviated UGGAA repeat-mediated RNA toxicity in SCA31 Drosophila model, suggesting that targeting UGGAA repeat by small molecules have a potential for treatment of SCA31.
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