| Budget Amount *help |
¥4,420,000 (Direct Cost: ¥3,400,000、Indirect Cost: ¥1,020,000)
Fiscal Year 2010: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2009: ¥2,730,000 (Direct Cost: ¥2,100,000、Indirect Cost: ¥630,000)
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| Research Abstract |
Congenital defects of ColQ cause endplate AChE deficiency and myasthenic syndrome. The intravenous administration of adeno-associated virus COLQto Colq-/-mice recovers motor functions, synaptic transmission, as well as the morphology of the NMJ. ColQ-tailed AChE is specifically anchored to NMJ. Furthermore, the in vivo injection of recombinant ColQ-tailed AChE protein complex into the gluteus maximus muscle of Colq-l- mice led to accumulation of AChE in non-injected forelimbs. We demonstrated for the first time in vivo that the ColQ protein contains a tissue-targeting signal that is sufficient for anchoring itself to the NMJ.
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