Development of a site-specific gene integration method to human genomes

Research Project

Project/Area Number	16K14633
Research Category	Grant-in-Aid for Challenging Exploratory Research
Allocation Type	Multi-year Fund
Research Field	Tumor therapeutics
Research Institution	Okayama University
Principal Investigator	Sera Takashi 岡山大学, ヘルスシステム統合科学研究科, 教授 (10362443)
Project Period (FY)	2016-04-01 – 2019-03-31
Project Status	Completed (Fiscal Year 2018)
Budget Amount *help	¥3,900,000 (Direct Cost: ¥3,000,000、Indirect Cost: ¥900,000) Fiscal Year 2018: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000) Fiscal Year 2017: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000) Fiscal Year 2016: ¥780,000 (Direct Cost: ¥600,000、Indirect Cost: ¥180,000)
Keywords	遺伝子導入
Outline of Final Research Achievements	In order to overcome the problems of conventional gene therapy, we aimed to develop a new technology that enables site-specific gene insertion by making an integrase spatially close to the target DNA site. To this end, a new artificial DNA binding protein is created by connecting two kinds of artificial DNA binding proteins that specifically bind to their own target sites respectively with a flexible peptide linker, and using this novel protein, in the in vitro model system, we succeeded in specifically inserting another DNA into the target DNA.
Academic Significance and Societal Importance of the Research Achievements	ゲノム上の特定の標的サイトに、望む遺伝子を特異的に挿入する、新たな方法論を提供することができる。これにより、レトロウイルスベクターの致命的な問題点を克服し、患者の有する疾患に合わせた様々な治療遺伝子をゲノム上の安全な場所に挿入することが可能となる。さらに、本手法は、ゲノム情報を自由に改変する手法の一つとして新たに加わることになり、様々な生命現象の解明や、新しいバイオテクノロジーの創出にも貢献することが大いに期待される。