Budget Amount *help |
¥5,070,000 (Direct Cost: ¥3,900,000、Indirect Cost: ¥1,170,000)
Fiscal Year 2016: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2015: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2014: ¥1,820,000 (Direct Cost: ¥1,400,000、Indirect Cost: ¥420,000)
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Outline of Final Research Achievements |
In this study, I established two type of CRISPR-Cas9 systems using vaculovirus vectors that work to knock out or mutate causative genes of hemochromatosis, porphyria, glycogenosis, amino acid metabolism abnormality, urea cycle disorder, constitutional jaundice, fibrotic cystic liver disease and Wilson’s disease. I transfected theses constructs into HEK293 cells and harvested vaculoviruses. However, when I inoculated HuH7 cells and HepG2 cells with the vaculoviruses, the cell growths’ were seriously inhibited. From these results, I decided to improve vaculovirus vectors. Now I confirming whether the targeted genome editing was occurred using the improved CRISPR-Cas9 systems. Further experiments are necessary. I will establish in vitro hereditary liver diseases models using human primary hepatocytes derived from human hepatocytes transplanted chimeric mice and in vivo models using human hepatocytes transplanted chimeric mice with these CRISPR-Cas9 systems.
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