Development of AAV-induced exonskipping therapy for Duchenne muscular dystrophy
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 |
Section | 一般 |
Research Field |
Neurology
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Research Institution | National Center of Neurology and Psychiatry |
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 |
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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Report
(4 results)
Research Products
(18 results)
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[Journal Article] Prognostic impact of venous thromboembolism in patients with Duchenne muscular dystrophy: Prospective multicenter 5-year cohort study.2015
Author(s)
Kimura K, Morita H, Daimon M, Kawata T, Nakao T, Lee SL, Hirokawa M, Ebihara A, Nakajima T, Ozawa T, Yonemochi Y, Aida I, Motoyoshi Y, Mikata T, Uchida I, Komori T, Kitao R, Nagata T, Takeda S, Komaki H, Segawa K, Takenaka K, Komuro I.
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Journal Title
Int J Cardiol.
Volume: 191
Pages: 178-80
DOI
Related Report
Peer Reviewed / Open Access
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[Journal Article] Necroptosis drives motor neuron death in models of both sporadic and familial ALS.2014
Author(s)
Re DB, Le Verche V, Yu C, Amoroso MW, Politi KA, Phani S, Ikiz B, Hoffmann L, Koolen M, Nagata T, Papadimitriou D, NagyP, Mitsumoto H, Kariya S, Wichterle H, Henderson CE, Przedborski S.
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Journal Title
Neuron
Volume: 81(5)
Issue: 5
Pages: 1001-1008
DOI
Related Report
Peer Reviewed
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[Presentation] Assessment of the Dystrophin Gene Exon 53 Skipping Using DMD Patient-Derived Fibroblasts for Exploratory Clinical Trial of Antisense Drug NS-065/NCNP-01.2014
Author(s)
Saito T, Nagata T, Masuda S, Tanihata J, Ohata M, Tamaura A, Kanazawa M, Minami N, Goto K, Hayashi Y, Iwasawa K, Tatezawa K, Fukuda K, Mizutani T, Shimizu R, Suzuki M, Yamaguchi K, Tachimori H, Nishino I, Goto Y, Komaki H, Takeda S.
Organizer
American society of gene & cell therapy 17th Annual meeting
Place of Presentation
Marriott Wardman Park Hotel
Year and Date
2014-05-20 – 2014-05-24
Related Report
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