Hydrodynamic Gene Delivery to Non-human Primates

2017 Fiscal Year Final Research Report

• Project/Area Number: 26860354
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Applied pharmacology
• Research Institution: Niigata University
• Principal Investigator: KAMIMURA KENYA 新潟大学, 医歯学系, 助教 (00579146)
• Research Collaborator: Terai Shuji 新潟大学, 医歯学系, 教授 ; Aoyagi Yutaka 新潟医療センター, 消化器病センター長 ; Suda Takeshi 新潟大学, 医歯学総合病院, 特任教授 ; Yokoo Takeshi 新潟大学, 医歯学総合研究科, 客員研究員 ; Fujisawa Nobuyoshi 新潟大学, 脳研究所, 助教 ; Yoshida Hidenori 新潟大学, 医歯学系, 助教 ; Oda Masafumi 新潟大学, 地域創生推進機構, 教授 ; Liu Dexi ジョージア大学, 教授
• Project Period (FY): 2014-04-01 – 2018-03-31
• Keywords: 遺伝子治療 / 肝臓特異的遺伝子導入 / ハイドロダイナミック遺伝子導入法 / 核酸医薬 / ドラッグデリバリー

Outline of Final Research Achievements

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.

Free Research Field

消化器病学