| Budget Amount *help |
¥5,330,000 (Direct Cost: ¥4,100,000、Indirect Cost: ¥1,230,000)
Fiscal Year 2014: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
Fiscal Year 2013: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
Fiscal Year 2012: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
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| Outline of Final Research Achievements |
Toward the development of a feasible mitochondrial gene therapy in clinic, the plasmids consist of a mitochondria-specific promotor and terminal signal with genes, which are modified for a mitochondria-specific codon usage, were developed, and we confirmed no expression from the plasmids in the nucleus using a confocal microscopy.
Because hydrodynamic gene delivery could not efficiently deliver the plasmids to mitochondria, proteins binding to the mitochondrial localization signal were employed for enhancing the delivery. In order to prevent the dissociation between the localization signal and proteins, we newly developed hydrodynamic gene delivery injecting a half volume with a half speed of the authentic hydrodynamic delivery.
Now we are evaluating the effectiveness of the modified hydrodynamic system in the delivery of the plasmids to the mitochondria in mice.
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