Budget Amount *help |
¥4,680,000 (Direct Cost: ¥3,600,000、Indirect Cost: ¥1,080,000)
Fiscal Year 2010: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2009: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2008: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
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Research Abstract |
We have shown the clinical effectiveness of antisense therapy for Duchenne muscular dystrophy (DMD) which converts out-of-frame to in-frame mutation by inducing the exon skipping by antisense oligonucleotide. To enhance the effect of this therapy, we developed the in vitro model system of antisense therapy based on the mutation spectrum of Japanese DMD cases. Furthermore, the effect of antisense oligonucleotide on dystrophin production and signal transduction system was investigated. The effect of this therapy was different among DMD cases, and it is important to examine the effect of antisense therapy using individual in vitro system of each case.
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