2018 Fiscal Year Final Research Report
Development of new gene therapy for primary immunodeficiencies by suppressing non-specific genetic modifications
| Project/Area Number |
16K19653
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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 |
Pediatrics
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| Research Institution | Kyushu University |
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Principal Investigator |
YAMAMOTO Hiroyuki 九州大学, 環境発達医学研究センター, 特任助教 (00710170)
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| Project Period (FY) |
2016-04-01 – 2019-03-31
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| Keywords | 原発性免疫不全症 / ウイルスベクター / 遺伝子治療 |
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| Outline of Final Research Achievements |
To examine the possibility of using gene therapy for primary immunodeficiencies, we constructed an adeno-associated virus vector to serve as a donor template, and a sequence-specific gRNA for double strand breaks. Human cord blood CD34 positive cells with a small molecule were infected with the vector and electroporated with gRNA and Cas9 protein. This delivery method can be a new gene therapy by suppressing non-specific genetic modifications.
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| Free Research Field |
分子生物学
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| Academic Significance and Societal Importance of the Research Achievements |
ゲノム編集技術による原発性免疫不全症に対する遺伝子治療の基礎研究は、重症複合免疫不全症などを中心に既に行われているが、無ガンマグロブリン血症を対象とした研究の論文報告はまだない。無ガンマグロブリン血症に対する非特異的な遺伝子改変を抑制した新規遺伝子治療法の研究は、将来の新たな安全な治療法の確立につながる可能性がある。
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