| Budget Amount *help |
¥4,940,000 (Direct Cost: ¥3,800,000、Indirect Cost: ¥1,140,000)
Fiscal Year 2016: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
Fiscal Year 2015: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2014: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
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| Outline of Final Research Achievements |
Duchenne muscular dystrophy (DMD) is a severe congenital disease due to mutations in the dystrophin gene. Supplementation of dystrophin using rAAV has promise as a treatment of DMD, however, host immune responses against the vector as well as transgene products have been denoted in the clinical studies. Here, we transduced rAAV9-microdystrophin(uDys) with MSCs to DMD dog to investigate the therapeutic effects of an rAAV-uDys under conditions of immune tolerance. MSCs and rAAV9-Luc/rAAV9-uDys were intravenously injected into the normal or CXMDJ dog at 8 weeks old. Seven days after injection, MSCs were systemically injected again. At 8 days after 1st injection, rAAV9-Luc/rAAV9-uDys was intramuscularly or intravenously injected into the same dog. Administration of rAAV following MSCs treatment resulted in higher expression of transgene, compared to the rAAV transduction alone. The CXMDJ treated with MSCs and rAAV9-uDys showed functional improvement than other DMD dogs of same age.
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