2013 Fiscal Year Final Research Report
Therapeutic systems of muscular dystrophy by morpholino oligonucleotide-loaded nanobubble and ultrasound
| Project/Area Number |
23300193
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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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| Research Field |
Medical systems
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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) |
MARUYAMA Kazuo 帝京大学, 薬学部, 教授 (30130040)
TAKAGI Norio 東京薬科大学, 薬学部, 准教授 (50318193)
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| Co-Investigator(Renkei-kenkyūsha) |
ARAMAKI Yukihiko 東京薬科大学, 薬学部, 教授 (90138959)
TAKAHASHI Yoko (遠藤 葉子) 東京薬科大学, 薬学部, 助手 (30453806)
NOMIZU Motoyoshi 東京薬科大学, 薬学部, 教授 (00311522)
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| Research Collaborator |
TANONAKA Kouichi 東京薬科大学, 薬学部, 教授
MARUNOUCHI Tetsuro 東京薬科大学, 薬学部, 大学院生
KATAGIRI Fumihiko 東京薬科大学, 薬学部, 大学院生
OMATA Daiki 東京薬科大学, 薬学部, 大学院生
HAMANO Nobuhito 東京薬科大学, 薬学部, 大学院生
ISHII Yuko 東京薬科大学, 薬学部, 大学院生
OGURI Yukiko 東京薬科大学, 薬学部, 学部生
SHIONO Hitomi 東京薬科大学, 薬学部, 学部生
AKIYAMA Saki 東京薬科大学, 薬学部, 学部生
MAYAMA Sayaka 東京薬科大学, 薬学部, 学部生
KIKUCHI Taiki 東京薬科大学, 薬学部, 学部生
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| Project Period (FY) |
2011-04-01 – 2014-03-31
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| Keywords | 超音波 / 筋ジストロフィー / ジストロフィン / アンチセンス |
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| Research Abstract |
It is expected that exon skipping, mediated by antisense oligonucleotides (AOs), is one of the most promising methods for restoration of dystrophin expression in Duchenne muscular dystrophy (DMD) treatment. However, efficient delivery method of AOs is still required for the DMD treatment. We have developed the PEG-liposomes entrapping echo-contrast gas, Bubble liposomes (BLs), which can function as a novel gene delivery tool with ultrasound (US). In this study, to develop an efficient phosphorodiamidate morpholino oligomer (PMO) delivery system, we tested the potency of the BLs combined with US to boost the delivery of PMO and increase the skipping of the mutated exon in the DMD model mouse. The results indicated that the combination of BLs and US increased the efficiency compared with PMO injection alone, leading to enhanced dystrophin expression in the US-focused muscles. Thus, this US-mediated BLs technique may provide an effective delivery method for PMO therapy for DMD muscle.
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[Journal Article] Bubble Liposomes and Ultrasound Exposure Improve Localized Morpholino Oligomer Delivery into Skeletal Muscles of Dystrophic mdx Mice2014
Author(s)
Negishi Y, Ishii Y, Shiono H, Akiyama S, Sekine S, Kojima T, Mayama S, Kikuchi T, Hamano N, Endo-Takahashi Y, Suzuki R, Maruyama K, Aramaki Y
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Journal Title
Molecular Pharmaceutics
Volume: 11
Pages: 1053-1061
DOI
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