2021 Fiscal Year Final Research Report
A new oligonucleotide treatment with CRISPR/Cas13 using model mice of spinocerebellar ataxia
| Project/Area Number |
19K07994
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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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| Review Section |
Basic Section 52020:Neurology-related
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| Research Institution | Hiroshima University |
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Principal Investigator |
MATSUDA Yukiko 広島大学, 原爆放射線医科学研究所, 研究員 (10735301)
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| Co-Investigator(Kenkyū-buntansha) |
森野 豊之 徳島大学, 大学院医歯薬学研究部(医学域), 教授 (10397953)
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| Project Period (FY) |
2019-04-01 – 2022-03-31
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| Keywords | 脊髄小脳変性症 |
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| Outline of Final Research Achievements |
To develop a novel therapeutic method inhibiting the expression of the mutant gene for point mutation type in the autosomal dominant hereditary spinocerebellar degeneration (SCD), we investigated a method knocking-down the transcript of the mutant gene by CRISPR/Cas13d using SCA42 knock-in mice. We have made an adeno-associated virus incorporating Cas13d system, which specifically recognizes the target gene. We have established the method suppressing the expression of the target gene in mouse cerebellar culture. In the future, we will search conditions with higher specificity for the mutant gene and examine the therapeutic effect in knock-in mice.
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| Free Research Field |
神経科学
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| Academic Significance and Societal Importance of the Research Achievements |
点変異型常染色体優性遺伝性脊髄小脳変性症において、病因である変異型mRNAを選択的に分解することができれば病状の改善を期待できる。ターゲット遺伝子を特異的に認識するCas13dシステムを組み込んだアデノ随伴ウイルスによりマウス小脳培養細胞系にてターゲット遺伝子の発現を抑制出来る系を確立した。今後さらなる検討を必要とするが、新しい治療法の開発において有効な方法と期待される。
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