| Budget Amount *help |
¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000)
Fiscal Year 2016: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
Fiscal Year 2015: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
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| Outline of Final Research Achievements |
To clarify the effect of medical-induction of proteolytic machinery in the spinal and bulbar muscular atrophy (SBMA), a neuromuscular disease caused by the CAG repeat expansion in the androgen receptor (AR) gene, we investigated effect of tacrolimus derivative in the SBMA model. Tacrolimus derivative increased the expression of proteolytic machinery-related molecule in cell models of SBMA. Mutant AR was reduced in the presence of tacrolimus derivative. Administration of the tacrolimus derivative ameliorated behavioral parameters in SBMA Tg mice. These observations suggest that tacrolimus derivative is a promising therapeutic candidate for motor neuron diseases including SBMA.
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