Next generation genome editing without artificial nucleases
| Project/Area Number |
17K19491
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| Research Category |
Grant-in-Aid for Challenging Research (Exploratory)
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| Allocation Type | Multi-year Fund |
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| Research Field |
Pharmaceutical Sciences and related fields
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| Research Institution | Hiroshima University |
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Principal Investigator |
KAMIYA Hiroyuki 広島大学, 医系科学研究科(薬), 教授 (10204629)
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| Co-Investigator(Kenkyū-buntansha) |
鈴木 哲矢 広島大学, 医系科学研究科(薬), 助教 (20573950)
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| Project Period (FY) |
2017-06-30 – 2020-03-31
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| Project Status |
Completed (Fiscal Year 2019)
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| Budget Amount *help |
¥6,370,000 (Direct Cost: ¥4,900,000、Indirect Cost: ¥1,470,000)
Fiscal Year 2019: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2018: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2017: ¥2,990,000 (Direct Cost: ¥2,300,000、Indirect Cost: ¥690,000)
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| Keywords | ゲノム編集 |
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| Outline of Final Research Achievements |
We previously developed tailed duplexes, several hundred-base single-stranded DNAs with an oligodeoxyribonucleotide annealed to the 3'-region of the long ss DNAs. In this study, we examined various approaches towards genome editing with tailed duplexes. We found that (i) editing efficiency was enhanced when a second mismatch was introduced into the tailed duplexes and that (ii) tailed duplexes with nearly 100-base single-stranded DNAs and with the single strand and double strand junctions close to the target positions showed high editing efficiencies. We also demonstrated that (iii) genome editing was achieved by the tailed duplex in cultured cells, without artificial nucleases.
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| Academic Significance and Societal Importance of the Research Achievements |
疾病をDNAレベルで治療する遺伝子治療の1つに、ゲノム（染色体）DNA上の変異配列を正常配列に戻す方法があり、この方法はゲノム編集に含まれる。現在のゲノム編集は、人工DNA分解酵素による染色体DNA切断に基づくが、非標的部位における切断による重篤な副作用が危惧される。そのため、標的DNAを切断することなくゲノム編集を行うことが望ましい。本研究では、研究代表者が考案したtailed duplexによりDNA切断を要しないゲノム編集が可能であることを示した。また、本方法により得られた知見は継続する研究で得られる結果と合わせ、細胞内のDNA修復機構に対する理解を深めるものとなる。
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Report
(4 results)
Research Products
(13 results)
