Precise genomic correction by intercellular regulation of genome editing enzyme
| Project/Area Number |
15H05581
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| Research Category |
Grant-in-Aid for Young Scientists (A)
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| Allocation Type | Single-year Grants |
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| Research Field |
Medical genome science
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| Research Institution | Kyoto University |
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Principal Investigator |
Hotta Akitsu 京都大学, iPS細胞研究所, 特定拠点講師 (50578002)
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| Project Period (FY) |
2015-04-01 – 2018-03-31
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| Project Status |
Completed (Fiscal Year 2017)
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| 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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| Keywords | ゲノム編集 / iPS細胞 / CRISPR / 相同組換え / 一塩基修復 / 一塩基変異 / 一塩基置換 / CRISPR-Cas9 / 遺伝子治療 |
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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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Report
(5 results)
Research Products
(35 results)
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[Journal Article] Srf destabilizes cellular identity by suppressing cell-type-specific gene expression programs2018
Author(s)
Ikeda T, Hikichi T, Miura H, Shibata H, Mitsunaga K, Yamada Y, Woltjen K, Miyamoto K, Watanabe A, Hiratani I, Yamada Y, Yamamoto T, Hotta A, Okita K, Masui S
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Journal Title
Nat Commun.
Volume: 9
Issue: 1
Pages: 1387-1387
DOI
NAID
Related Report
Peer Reviewed / Open Access
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