2020 Fiscal Year Final Research Report
Molecular analysis of genome-editing mechanism in brain and development of novel genome-editing technology
| Project/Area Number |
18H04036
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| Research Category |
Grant-in-Aid for Scientific Research (A)
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| Allocation Type | Single-year Grants |
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| Section | 一般 |
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| Review Section |
Medium-sized Section 51:Brain sciences and related fields
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| Research Institution | Osaka University |
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Principal Investigator |
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| Project Period (FY) |
2018-04-01 – 2021-03-31
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| Keywords | ゲノム編集 / 神経 / HITI法 / SATI法 / プロジェリア症候群 |
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| Outline of Final Research Achievements |
A radical treatment method for most of inherited neuronal diseases has not been developed yet. In this study, we elucidated the molecular mechanism of genome editing in brain. Based on the mechanism, we have developed novel genome-editing method “SATI” that can repair many kinds of mutations in vivo. Using this method, we treated an HGPS premature mouse model carrying a dominant point mutation and demonstrated amelioration of aging-related phenotypes in multiple organs and extension of lifespan.
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| Free Research Field |
ゲノム編集
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| Academic Significance and Societal Importance of the Research Achievements |
本研究成果により、神経細胞におけるゲノム編集技術の分子機構の一端が明らかになり、様々なタイプの遺伝子変異を脳だけでなく様々な臓器で修復できるSATI法の開発に成功した。
今後本技術がさらに改良されることで、早老症のみならず他の神経や筋肉や網膜など様々な組織や全身に異常を持つ難治性遺伝病に対し、その原因となる異常遺伝子を病変部位で直接修復する医療への応用が期待される。
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