2017 Fiscal Year Final Research Report
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 |
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| Research Field |
Neurophysiology / General neuroscience
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| Research Institution | Gunma University |
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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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| 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 |
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| 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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| Free Research Field |
神経科学
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