| Budget Amount *help |
¥46,410,000 (Direct Cost: ¥35,700,000、Indirect Cost: ¥10,710,000)
Fiscal Year 2011: ¥14,950,000 (Direct Cost: ¥11,500,000、Indirect Cost: ¥3,450,000)
Fiscal Year 2010: ¥14,950,000 (Direct Cost: ¥11,500,000、Indirect Cost: ¥3,450,000)
Fiscal Year 2009: ¥16,510,000 (Direct Cost: ¥12,700,000、Indirect Cost: ¥3,810,000)
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| Research Abstract |
Toward gene therapy of Duchenne muscular dystrophy(DMD), we transferred the human artificial chromosome with 2. 4 Mb genomic dystrophin gene(DYS-HAC) to the DMD mouse model(mdx)-derived mesoangioblasts, then the corrected mesoangioblasts were transplanted to mdx-mice. As a result, we succeeded in the long term-amelioration of the dystrophic phenotype in the mdx mice. Thus, a combination of HAC-mediated gene replacement and transplantation with mesoangioblasts will be useful for gene therapy of DMD.
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