| Budget Amount *help |
¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000)
Fiscal Year 2018: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2017: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2016: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
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| Outline of Final Research Achievements |
Down syndrome (DS) is the most frequent genetic cause of intellectual disability, affecting one in 1,000 live births worldwide. DS is caused by the extra chromosome 21, the prenatal diagnosis of DS is now feasible. Yet, no therapy is currently available. We screened for neurogenesis inducers which can restore impaired proliferation of neural stem cells in DS/DS models and identified ALGERNON (altered generation of neurons) as an inhibitor against DYRK1A encoded in DS critical region of chromosome 21. Treatment of pregnant dams with ALGERNON normalized the morphological abnormal brain development, and most remarkably, impaired neurocognitive behaviors in DS-offspring. These data suggest that the neurogenic phenotype of DS can be prevented by ALGERNON prenatal therapy.
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