Development of a novel gene therapy method via CRISPR/Cas9 mediated in vivo genome editing
Project/Area Number |
15K18330
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Research Category |
Grant-in-Aid for Young Scientists (B)
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Allocation Type | Multi-year Fund |
Research Field |
Neurophysiology / General neuroscience
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Research Institution | Gunma University |
Principal Investigator |
Konno Ayumu 群馬大学, 大学院医学系研究科, 講師 (40509048)
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Project Period (FY) |
2015-04-01 – 2018-03-31
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Project Status |
Completed (Fiscal Year 2017)
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Budget Amount *help |
¥4,290,000 (Direct Cost: ¥3,300,000、Indirect Cost: ¥990,000)
Fiscal Year 2017: ¥1,430,000 (Direct Cost: ¥1,100,000、Indirect Cost: ¥330,000)
Fiscal Year 2016: ¥1,300,000 (Direct Cost: ¥1,000,000、Indirect Cost: ¥300,000)
Fiscal Year 2015: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
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Keywords | 遺伝子治療 / アデノ随伴ウイルスベクター / CRISPR/Cas9 / ゲノム編集 / 脊髄小脳失調症 / AAV |
Outline of Final Research Achievements |
In this study, we aimed to develop a novel gene therapy method for spinocerebellar ataxia type 1 with CRISPR/Cas9 mediated in vivo genome editing using a gene delivery by adeno-associated virus vector. Although we examined SpCas9 and SaCas9 for in vivo knockout, we could not observe the amelioration of an ataxia in a mouse model of spinocerebellar ataxia type 1. Further investigations for higher efficiencies of a gene expression and/or a gene editing are needed.
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Report
(4 results)
Research Products
(26 results)
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[Journal Article] Elavl3 is essential for the maintenance of Purkinje neuron axons2018
Author(s)
Ogawa Yuki、Kakumoto Kyoko、Yoshida Tetsu、Kuwako Ken-ichiro、Miyazaki Taisuke、Yamaguchi Junji、Konno Ayumu、Hata Junichi、Uchiyama Yasuo、Hirai Hirokazu、Watanabe Masahiko、Darnell Robert B.、Okano Hideyuki、Okano Hirotaka James
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Journal Title
Sci Rep.
Volume: 8
Issue: 1
Pages: 2722-2722
DOI
Related Report
Peer Reviewed / Open Access / Int'l Joint Research
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