| Project/Area Number |
25461303
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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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| Research Field |
Neurology
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| Research Institution | National Center of Neurology and Psychiatry |
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Principal Investigator |
Nagata Tetsuya 国立研究開発法人国立精神・神経医療研究センター, 神経研究所 遺伝子疾患治療研究部, 客員研究員 (50362976)
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| Co-Investigator(Kenkyū-buntansha) |
Okada Takashi 日本医科大学, 医学(系)研究科(研究院), 教授 (00326828)
Saito Takashi 独立行政法人国立精神・神経医療研究センター, 神経研究所 遺伝子疾患治療研究部, 流動研究員 (40625969)
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| Project Period (FY) |
2013-04-01 – 2016-03-31
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| Project Status |
Completed (Fiscal Year 2015)
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| Budget Amount *help |
¥5,070,000 (Direct Cost: ¥3,900,000、Indirect Cost: ¥1,170,000)
Fiscal Year 2015: ¥1,170,000 (Direct Cost: ¥900,000、Indirect Cost: ¥270,000)
Fiscal Year 2014: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2013: ¥2,340,000 (Direct Cost: ¥1,800,000、Indirect Cost: ¥540,000)
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| Keywords | エクソン・スキップ / トランスレーショナルリサ ーチ / アンチセンス・オリゴヌク レオチド / アデノ随伴ベクター / デュシェンヌ型筋ジストロ フィー / デュシェンヌ型筋ジストロフィー / アンチセンス・オリゴヌクリオチド |
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| Outline of Final Research Achievements |
Antisense-mediated exon skipping is one of the most promising therapeutic tools for the treatment of Duchenne muscular dystrophy (DMD), which is caused by mutations in the dystrophin gene. Here, we tried to develop the adeno-associated virus (AAV)-induced exon skipping therapy for DMD.U7 snRNAs harboring antisense motifs induce skipping of exon 51, and thus restore dystrophin expression. Furthermore, we show the efficacy of these constructs in vivo in mdx. Our constructs are promising for the optimization of therapeutic exon skipping for DMD.
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