| Budget Amount *help |
¥16,510,000 (Direct Cost: ¥12,700,000、Indirect Cost: ¥3,810,000)
Fiscal Year 2016: ¥3,640,000 (Direct Cost: ¥2,800,000、Indirect Cost: ¥840,000)
Fiscal Year 2015: ¥6,110,000 (Direct Cost: ¥4,700,000、Indirect Cost: ¥1,410,000)
Fiscal Year 2014: ¥6,760,000 (Direct Cost: ¥5,200,000、Indirect Cost: ¥1,560,000)
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| Outline of Final Research Achievements |
In order to establish a therapeutic strategy targeting genes in mitochondria (Mt), we examined Mt gene delivery and mitochondrial exogenous gene expression in cells derived from Mt disease patients. To date, we have been able to develop a MITO-Porter, a nano carrier for Mt delivery. In this study, we report on the successful Mt gene delivery in disease cells that have a heteroplasmic mutation in mtDNA, using MITO-Porter system. In addition, we designed a pHSP-mtLuc (CGG) analog, an artificial Mt gene expression plasmid DNA vector that contains a promotor for Mt transcription and an artificial Mt genome with a reporter gene that records adjustments to the Mt codon system. Collectively, we succeeded in achieving Mt exogenous gene expression by the Mt delivery of a pHSP-mtLuc (CGG) analog using the developed MITO-Porter system. Our Mt exogenous gene expression carriers promises to be a useful approach to Mt gene therapy and research regarding Mt molecular biology.
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