| Budget Amount *help |
¥4,420,000 (Direct Cost: ¥3,400,000、Indirect Cost: ¥1,020,000)
Fiscal Year 2013: ¥650,000 (Direct Cost: ¥500,000、Indirect Cost: ¥150,000)
Fiscal Year 2012: ¥1,690,000 (Direct Cost: ¥1,300,000、Indirect Cost: ¥390,000)
Fiscal Year 2011: ¥2,080,000 (Direct Cost: ¥1,600,000、Indirect Cost: ¥480,000)
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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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