| Budget Amount *help |
¥17,160,000 (Direct Cost: ¥13,200,000、Indirect Cost: ¥3,960,000)
Fiscal Year 2019: ¥5,330,000 (Direct Cost: ¥4,100,000、Indirect Cost: ¥1,230,000)
Fiscal Year 2018: ¥5,590,000 (Direct Cost: ¥4,300,000、Indirect Cost: ¥1,290,000)
Fiscal Year 2017: ¥6,240,000 (Direct Cost: ¥4,800,000、Indirect Cost: ¥1,440,000)
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| Outline of Final Research Achievements |
To treat diseases caused by point mutations in the gene, we have developed a site-specific repairing method for genetic codes of the mutated RNAs, and succeeded in developing an artificial enzyme complex that catalyzes A⇒I (G) or C⇒U conversions in cells. The enzyme complex was able to act specifically on the mutated points of RNA by guide RNAs complementary to the target RNAs, and was able to repair about 40% of the mutated RNAs in cells. We also successfully repaired the genetic code of mutated P-type ATPase RNA in primary cultured fibroblasts derived from macular mice as a disease model, confirming the restoration of cytochrome C oxidase activity, whose activity is almost undetectable in macular mice.
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