| Budget Amount *help |
¥15,730,000 (Direct Cost: ¥12,100,000、Indirect Cost: ¥3,630,000)
Fiscal Year 2017: ¥5,070,000 (Direct Cost: ¥3,900,000、Indirect Cost: ¥1,170,000)
Fiscal Year 2016: ¥5,200,000 (Direct Cost: ¥4,000,000、Indirect Cost: ¥1,200,000)
Fiscal Year 2015: ¥5,460,000 (Direct Cost: ¥4,200,000、Indirect Cost: ¥1,260,000)
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| Outline of Final Research Achievements |
About half of the genetic mutation causing human disease is a single nucleotide mutation. Deletion via NHEJ (nonhomologous end joining) can be relatively easily induced by using recent genomic editing techniques including CRISPR-Cas9, but knock-in using HDR (homologous recombination) efficiency was less than 1%, which was extremely inefficient. Therefore, we developed the CRONUS system in which CRISPR-Cas9 and guide-RNA were incorporated in the cells with a piggyBac transposon vector in advance, and Cas9 activity can be induced with two kinds of drugs. This made it possible to induce a desired single base modification at efficiencies as high as 20% to 30%, far exceeding the efficiency (<1%) so far.
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