A genome-wide RNAi screen to identify genes involved in the incorporation of antisense oligonucleotides into the cells
| Project/Area Number |
18K06811
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| Research Category |
Grant-in-Aid for Scientific Research (C)
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| Allocation Type | Multi-year Fund |
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| Section | 一般 |
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| Review Section |
Basic Section 47060:Clinical pharmacy-related
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| Research Institution | National Institute of Health Sciences |
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Principal Investigator |
Inoue Takao 国立医薬品食品衛生研究所, 遺伝子医薬部, 部長 (50361605)
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| Project Period (FY) |
2018-04-01 – 2022-03-31
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| Project Status |
Completed (Fiscal Year 2021)
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| Budget Amount *help |
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2020: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2019: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2018: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
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| Keywords | 核酸医薬品 |
|---|
| Outline of Final Research Achievements |
In recent years, development of nucleic acid drugs such as antisense and siRNA has been progressing, attracting attention as next-generation drugs for hereditary and intractable diseases for which there is no cure. However, the pathways and molecular mechanisms by which nucleic acid drugs are taken up from outside the cell and reach their sites of action inside the cell have not been clarified. In this study, we constructed a screening system to comprehensively search for candidate molecules involved in these pathways using Gapmer-type antisense and splice-switching oligonucleotides, and succeeded in identifying a group of candidate genes.
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| Academic Significance and Societal Importance of the Research Achievements |
アンチセンスの細胞内取り込みに関与する分子群の特定は、アンチセンスの細胞内送達を向上される技術の抄出に繋がるもので、医療分野の進展に資するものである。また、原料供給・製造/分析・品質担保に関する課題解決案の提示についても、対応するプロジェクト等が発動すれば、核酸医薬開発を進展に大きく寄与する。核酸医薬品はアンメットメディカルニーズに対応する新規モダリティとして期待が高まっていることから、本研究で得られた成果は社会的ニーズに直結すると判断される。
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Report
(5 results)
Research Products
(127 results)
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[Journal Article] 核酸医薬品の安全性評価に関する考え方 -仮想核酸医薬品をモデルにして- 第3回：既存情報の有効活用2018
Author(s)
木下潔,真木一茂,荒戸照世,太田哲也,小野寺博志,佐藤秀昭,中澤隆弘,平林容子,笛木修,三井田宏明,吉田徳幸,渡部一人,小比賀聡,井上貴雄
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Journal Title
医薬品医療機器レギュラトリーサイエンス
Volume: 49
Pages: 207-214
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