Generation of episomal RNA viral vector for stem cell gene therapy
| Project/Area Number |
16K19069
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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 |
Human genetics
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| Research Institution | Kyoto University |
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Principal Investigator |
Makino Akiko 京都大学, ウイルス・再生医科学研究所, 助教 (30571145)
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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 |
¥4,030,000 (Direct Cost: ¥3,100,000、Indirect Cost: ¥930,000)
Fiscal Year 2017: ¥1,560,000 (Direct Cost: ¥1,200,000、Indirect Cost: ¥360,000)
Fiscal Year 2016: ¥2,470,000 (Direct Cost: ¥1,900,000、Indirect Cost: ¥570,000)
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| Keywords | ウイルスベクター / 幹細胞 / RNAウイルスベクター / 遺伝子治療 / 幹細胞遺伝子治療 / ボルナウイルスベクター / X-SCID / エピソーマル |
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| Outline of Final Research Achievements |
Borna disease virus (BoDV) is an RNA virus persistently infecting stem cells. In this study, we aimed to construct a long-term stable gene transfer system that realizes stem cell therapy using the virus. The BoDV vector expressing IL2RG, which is the causative gene of X-SCID, was prepared and inoculated into hematopoietic stem cells. This vector stably expressed the target gene, however, the transduction efficiency was low. The vector in which the G protein was replaced with CnBV-1 increased the efficiency by 15-fold as compared with BoDV. The improved vector significantly increased the efficiency of transduction into stem cells including iPS cells. BoDV vector with X/P gene of VSBV also increased the transduction efficiency. The improved BoDV vector prepared in this study could be a new platform for stem cell gene therapy.
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Report
(3 results)
Research Products
(12 results)
