| Budget Amount *help |
¥16,900,000 (Direct Cost: ¥13,000,000、Indirect Cost: ¥3,900,000)
Fiscal Year 2020: ¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2019: ¥4,160,000 (Direct Cost: ¥3,200,000、Indirect Cost: ¥960,000)
Fiscal Year 2018: ¥3,380,000 (Direct Cost: ¥2,600,000、Indirect Cost: ¥780,000)
Fiscal Year 2017: ¥2,470,000 (Direct Cost: ¥1,900,000、Indirect Cost: ¥570,000)
Fiscal Year 2016: ¥2,730,000 (Direct Cost: ¥2,100,000、Indirect Cost: ¥630,000)
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| Outline of Final Research Achievements |
Myotonic dystrophy (MyD) is the most common inherited muscle disease in adults, affecting not only skeletal muscle but also multiple organs, including the heart muscle and brain. In this disease, abnormal RNAs transcribed from expanded CTG repeats disrupt splicing regulatory mechanisms and cause widespread splicing abnormalities. In this study, we developed therapeutic strategies using nucleic acid drugs and small molecules targeting the abnormal RNAs, which are central to the pathogenesis of MyD, and demonstrated therapeutic effects in skeletal muscle, cardiac muscle, and central nervous system in animal models.
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