Gene therapy using the in vivo gene targeting technology

Research Project

Project/Area Number	26670140
Research Category	Grant-in-Aid for Challenging Exploratory Research
Allocation Type	Multi-year Fund
Research Field	General medical chemistry
Research Institution	Kyoto University
Principal Investigator	Matsuda Takahiko 京都大学, 物質－細胞統合システム拠点, 研究員 (40313093)
Project Period (FY)	2014-04-01 – 2017-03-31
Project Status	Completed (Fiscal Year 2016)
Budget Amount *help	¥3,770,000 (Direct Cost: ¥2,900,000、Indirect Cost: ¥870,000) Fiscal Year 2016: ¥910,000 (Direct Cost: ¥700,000、Indirect Cost: ¥210,000) Fiscal Year 2015: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000) Fiscal Year 2014: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Keywords	ゲノム編集 / 網膜 / 内在性蛋白質 / 遺伝子治療
Outline of Final Research Achievements	By combining the CRISPR/Cas9 genome editing technology with an electroporation technique, we succeeded in creating GFP (RFP) knock-in alleles, from which GFP (RFP)-tagged endogenous proteins are produced, in neurons and glial cells in vivo in the postnatal mouse retina.