Development of ultrasound-responsive nanobubbles containing genome editing tools and establishment of disease treatment system
| Project/Area Number |
18H03552
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| Research Category |
Grant-in-Aid for Scientific Research (B)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Review Section |
Basic Section 90130:Medical systems-related
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| Research Institution | Tokyo University of Pharmacy and Life Science |
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Principal Investigator |
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| Co-Investigator(Kenkyū-buntansha) |
吉川 大和 東京薬科大学, 薬学部, 准教授 (20274227)
丸山 一雄 帝京大学, 薬学部, 特任教授 (30130040)
高橋 葉子 (遠藤葉子) 東京薬科大学, 薬学部, 助教 (30453806)
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| Project Period (FY) |
2018-04-01 – 2021-03-31
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| Project Status |
Completed (Fiscal Year 2020)
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| Budget Amount *help |
¥17,420,000 (Direct Cost: ¥13,400,000、Indirect Cost: ¥4,020,000)
Fiscal Year 2020: ¥3,510,000 (Direct Cost: ¥2,700,000、Indirect Cost: ¥810,000)
Fiscal Year 2019: ¥4,940,000 (Direct Cost: ¥3,800,000、Indirect Cost: ¥1,140,000)
Fiscal Year 2018: ¥8,970,000 (Direct Cost: ¥6,900,000、Indirect Cost: ¥2,070,000)
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| Keywords | ナノバブル / 超音波 / ドラッグデリバリーシステム / ゲノム編集 / 筋ジストロフィー / 超音波応答性ナノバブル / 核酸遺伝子デリバリー |
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| Outline of Final Research Achievements |
In the treatment of Duchenne muscular dystrophy (DMD) disease, methods of introducing genome-editing systems into muscle tissue that enable permanent gene editing are considered to be important. In this study, we successfully delivered and introduced the CRISPR-Cas9 system into the skeletal muscle tissue of a DMD model mouse using ultrasound-responsive nanobubbles. In fact, the CRISPR-Cas9 system restored the expression of the defective dystrophin protein not only as an expression plasmid vector but also when introduced as RNA molecules (Cas9mRNA/sgRNA). Therefore, this delivery system using nanobubble is expected to be a useful tool in DMD gene therapy based on genome editing.
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| Academic Significance and Societal Importance of the Research Achievements |
DMDモデルマウスを利用した治療研究において，超音波応答性ナノバブルにより，CRISPR/Cas9システムを細胞内送達させ，実際に機能発現した結果は，他の遺伝性疾患治療にも適用可能と考えられる．mRNA送達にも有用性を発揮できることから，がん治療や再生医療分野における新たな治療システム開発に繋がる可能性がある．本研究成果は，さらに標的リガンドを修飾したナノバブルを用いることで，疾患部位の診断と治療の一体化システム（セラノスティクス）の構築に繋がることから，学術的及び社会的にも大きな意義があると考えられる．
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Report
(4 results)
Research Products
(18 results)
