2013 Fiscal Year Final Research Report
Hydrodynamic Gene Delivery to Non-human Primates
| Project/Area Number |
23790595
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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 |
Applied pharmacology
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| Research Institution | Niigata University |
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Principal Investigator |
KAMIMURA Kenya 新潟大学, 医歯学総合病院, 医員 (00579146)
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| Project Period (FY) |
2011 – 2013
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| Keywords | 遺伝子治療 / ハイドロダイナミック遺伝子導入法 / 肝臓 / 大動物 / 血管造影手技 / 血友病 |
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| Research Abstract |
Development of a safe and effective method for gene delivery is an essential step toward gene therapy. We have previously reported the effectiveness of image-guided liver-lobe specific hydrodynamic gene delivery. Here, we have assessed the efficiency and the safety of the procedure in non-human primates in this study. The injection catheter was inserted in the lobular hepatic veins and the hydrodynamic injection of the marker plasmid DNA was performed with a various parameters including, injection pressure, volume, flow rate, etc. Based on the results obtained, the optimum parameters were defined followed by the long-term gene expression study using the parameters injecting plasmid expressing human factor IX DNA. The efficiency and safety of the procedure have been confirmed and the development of the gene delivery system and the pre-clinical study are ongoing research programs.
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