| Budget Amount *help |
¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000)
Fiscal Year 2020: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2019: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2018: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
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| Outline of Final Research Achievements |
Exon skipping therapies for Duchenne muscular dystrophy (DMD) require new strategies to enhance the effect. We have identified intronic splicing control sequences and verified that exon skipping efficiency can be enhanced by controlling the region. First, the intron sequences were analyzed using gDNA extracted from the peripheral blood of DMD patients with deletion mutations. Furthermore, we established a system to analyze splicing patterns using peripheral blood or muscle-derived mRNA. By examining the intron sequences and splicing patterns, we investigated the intronic splicing control sequences and the effect on the exon skipping therapy.
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