Helper-dependent adenovirusvector and modified lentiviral vector mediated delivery of dystrophin for gene therapy of muscular dystrophy
Project/Area Number |
20591003
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Research Category |
Grant-in-Aid for Scientific Research (C)
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Allocation Type | Single-year Grants |
Section | 一般 |
Research Field |
Neurology
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Research Institution | Kumamoto University |
Principal Investigator |
UCHINO Makoto Kumamoto University, 大学院・生命科学研究部, 教授 (20117336)
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Project Period (FY) |
2008 – 2010
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Project Status |
Completed (Fiscal Year 2010)
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Budget Amount *help |
¥4,550,000 (Direct Cost: ¥3,500,000、Indirect Cost: ¥1,050,000)
Fiscal Year 2010: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2009: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2008: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
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Keywords | 筋ジストロフィー / 遺伝子治療 / ヘルパー依存アデノウイルス / 改変レンチウイルス / full-length dystrophin / dko mouse / whole-body plethysmography / helper-dependent adenovirus vector / lentivirus / lentivirus vector |
Research Abstract |
We showed that a highly efficient dystrophin-transduction to the mex and dko's diaphragm-achieved by simple intraperitoneal injection of a helper-dependent adenovirus vector (HDAdv) containing the full-length dystrophin expression cassette - provided beneficial results. we carried out the packaging of the full-length dystrophin cDNA in a genetically modified lentivirus with a useful, sufficiently functional titer that was successfully transduced into myogenic cells derived from the transgenic mdx mouse.
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Report
(4 results)
Research Products
(16 results)
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[Presentation] Lentiviral vector mediated delivery of full-length dystrophin for gene therapy of muscular dystrophy.2010
Author(s)
Kimura E, Uchino K, Suga T, Ishizaki M, Koide T, Uchida Y, Maeda Y, Yamashita S, Chamberlain JS, Uchino M.
Organizer
13^<th> Annual Meeting of the American Society of Gene and Cell Therapy
Place of Presentation
Marriott Wardman Park Hotel, Washington DC, USA.
Related Report
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