2013 Fiscal Year Final Research Report
Gene therapy for neurodegenerative diseases using adeno-associated viral vectors that can cross the blood-brain barriers.
| Project/Area Number |
23590473
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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 |
Experimental pathology
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| Research Institution | Jichi Medical University |
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Principal Investigator |
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| Project Period (FY) |
2011 – 2013
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| Keywords | 神経変性疾患 / 遺伝子治療 |
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| Research Abstract |
We recently developed AAV vectors that can cross the blood-brain or meningeal-brain barriers. Using these AAV vectors, we have shown beneficial effects in mouse models of neurodegenerative diseases. The early intervention of miR-196a delivered by an AAV vector ameliorated the phenotypes in a mouse model of spinal and bulbar muscular atrophy. A single intracardiac administration of the vector carrying neprilysin gene in a mice model of Alzheimer disease reduced Abeta oligomers, with concurrent alleviation of abnormal learning and memory function. Gene delivery of the RNA-editing enzyme, adenosine deaminase, which acts on RNA 2 (ADAR2) in conditional ADAR2 knockout mice prevented progressive motor dysfunction. These AAV vectors may provide a novel platform for treating neurodegenerative diseases, especially where global transduction of a therapeutic gene into the brain is necessary.
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[Journal Article] Regulation of the dopaminergic system in a murine model of aromatic L-amino acid decarboxylase deficiency2013
Author(s)
Lee N-C, Shieh Y-D, Chien Y-H, Tzen K-Y, Yu I-S, Chen P-W, Hu M-H, Hu M-k, Muramatsu S, Ichinose H, Hwu W-L
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Journal Title
Neurobiol Dis
Volume: 52
Pages: 177-190
DOI
Peer Reviewed
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[Journal Article] Dopamine release via the vacuolar ATPase V0 sector c-subunit, confirmed in N18 neuroblastoma cells, results in behavioral recovery in hemiparkinsonian mice2012
Author(s)
Jin D, Muramatsu S, Shimizu N, Yokoyama S, Hirai H, Yamada K, Liu HX, Higashida C, Hashii M, Higashida A, Asano M, Ohkuma S, Higashida H
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Journal Title
Neurochem Int
Volume: 61(6)
Pages: 907-912
DOI
Peer Reviewed
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[Journal Article] Viral delivery of miR-196a ameliorates the SBMA phenotype via the silencing of CELF22012
Author(s)
Miyazaki M, Adachi H, Katsuno M, Minamiyama M, Jiang YM, Huang Z, Doi H, Matsumoto S, Kondo N, Iida M, Tohnai G, Tanaka F, Muramatsu S, Sobue G
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Journal Title
Nat Med
Volume: 18(7)
Pages: 1136-1141
DOI
Peer Reviewed
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[Presentation] AADC gene therapy for Parkinson's disease : four years of follow-up2011
Author(s)
Muramatsu S, Fujimoto K, Kato S, Asari S, Mizukami H, Ikeguchi K, Kawakami T, Urabe M, Kume A, Sato T, Watanabe E, Ozawa K and Nakano I
Organizer
The 34th Annual Meeting of Japan Neuroscience Society
Place of Presentation
Yokohama
Year and Date
2011-09-17
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[Presentation] パーキンソン病の遺伝子治療2011
Author(s)
村松慎一,浅利さやか,川上忠孝,池口邦彦,中野今治,水上浩明,卜部匡司,久米晃啓,小澤敬也,加藤正哉,渡辺英寿,佐藤俊彦
Organizer
平成23年度第10回自治医科大学シンポジウム
Place of Presentation
自治医科大学地域医療情報研修センター
Year and Date
2011-09-02
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[Presentation] AADC gene therapy for Parkinson's disease : Four years of follow-up2011
Author(s)
Muramatsu S, Fujimoto K, Kato S, Asari S, Mizukami H, Ikeguchi K, Kawakami T, Urabe M, Kume A, Sato T, Watanabe E, Ozawa K and Nakano I
Organizer
The 17th Annual Meeting of the Japan Society of Gene Therapy, Best Presentation
Place of Presentation
Fukuoka
Year and Date
2011-07-15
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[Presentation] PET assessment of the aromatic L-amino acid decarboxylase gene expression in a phase I gene therapy study for Parkinson's disease2011
Author(s)
Asari S, Fujimoto K, Kato S, Mizukami H, Ikeguchi K, Kawakami T, Urabe M, Watanabe E, Sato T, Ozawa K, Nakano I and Muramatsu S
Organizer
15th International Congress of Parkinson's Disease and Movement Disorders
Place of Presentation
Toronto
Year and Date
2011-06-09
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