| Budget Amount *help |
¥24,700,000 (Direct Cost: ¥19,000,000、Indirect Cost: ¥5,700,000)
Fiscal Year 2015: ¥7,280,000 (Direct Cost: ¥5,600,000、Indirect Cost: ¥1,680,000)
Fiscal Year 2014: ¥8,710,000 (Direct Cost: ¥6,700,000、Indirect Cost: ¥2,010,000)
Fiscal Year 2013: ¥8,710,000 (Direct Cost: ¥6,700,000、Indirect Cost: ¥2,010,000)
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| Outline of Final Research Achievements |
Triplet repeat disorders are caused by abnormal expansion of trinucleotide repeat sequences (repeat), and the longer the repeat the more severe the condition. These repeats also lengthen with age, contributing to progression of the disease. Therefore, controlling the repeat length is important from a treatment perspective. Here we conducted research with the aim of elucidating the repeat expansion mechanism as well as developing therapies to stabilize or shorten the repeat length. As a result, we have identified factors that affect repeat expansion, using repeat disorder model cells and patient-derived iPS cells. We also discovered a compound that stabilizes or shortens the repeat length, the effect has been demonstrated in cell models, and we can expect future clinical application of this discovery.
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