| Project/Area Number |
16K15477
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| Research Category |
Grant-in-Aid for Challenging Exploratory Research
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| Allocation Type | Multi-year Fund |
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| Research Field |
Neurology
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| Research Institution | Gunma University |
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Principal Investigator |
Hirai Hirokazu 群馬大学, 大学院医学系研究科, 教授 (70291086)
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| Co-Investigator(Renkei-kenkyūsha) |
HOSOI Nobutake 群馬大学, 大学院医学系研究科, 講師 (90543570)
KONNO Ayumu 群馬大学, 大学院医学系研究科, 講師 (40509048)
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| Research Collaborator |
MATSUZAKI Yasunori 群馬大学, 大学院医学系研究科, 助教 (50738200)
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| Project Period (FY) |
2016-04-01 – 2018-03-31
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| Project Status |
Completed (Fiscal Year 2017)
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| Budget Amount *help |
¥3,380,000 (Direct Cost: ¥2,600,000、Indirect Cost: ¥780,000)
Fiscal Year 2017: ¥2,340,000 (Direct Cost: ¥1,800,000、Indirect Cost: ¥540,000)
Fiscal Year 2016: ¥1,040,000 (Direct Cost: ¥800,000、Indirect Cost: ¥240,000)
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| Keywords | 小脳 / プルキンエ細胞 / 細胞種特異的プロモーター / CRISPR/Cas9 / ゲノム編集 / AAV / ウイルスベクター / 遺伝子治療 / SCA1 / AAVベクター / アデノ随伴ウイルスベクター / 脊髄小脳失調症 / 遺伝子 / 再生医学 / 神経科学 / 脳神経疾患 |
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| Outline of Final Research Achievements |
In this research, we aimed to establish a technique that allows elimination of a disease-causing gene from specific cell populations in the brain, using adeno-associated virus (AAV) vectors in combination with CRISPR/Cas9 system. Initially, we characterized neuron-specific, astrocyte-specific and cerebellar Purkinje cell-specific promoters available for AAV vector-mediated transgene expression. The results were published in 3 papers. Then, using the AAV vectors, we examined whether a gene responsible for developmental defects of the brain could be knocked out from Purkinje cells by CRISPR/Cas9 system. The results showed successful removal of the gene from around two thirds of Purkinje cells.
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