2016年10月
Liver-targeted hydrodynamic gene therapy: Recent advances in the technique
WORLD JOURNAL OF GASTROENTEROLOGY
- 巻
- 22
- 号
- 40
- 開始ページ
- 8862
- 終了ページ
- 8868
- 記述言語
- 英語
- 掲載種別
- 研究論文(学術雑誌)
- DOI
- 10.3748/wjg.v22.i40.8862
- 出版者・発行元
- BAISHIDENG PUBLISHING GROUP INC
One of the major research focuses in the field of gene therapy is the development of clinically applicable, safe, and effective gene-delivery methods. Since the first case of human gene therapy was performed in 1990, a number of gene-delivery methods have been developed, evaluated for efficacy and safety, and modified for human application. To date, viral-vector-mediated deliveries have shown effective therapeutic results. However, the risk of lethal immune response and carcinogenesis have been reported, and it is still controversial to be applied as a standard therapeutic option. On the other hand, delivery methods for non-viral vector systems have been developed, extensively studied, and utilized in in vivo gene-transfer studies. Compared to viral-vector mediated gene transfer, nonviral systems have less risk of biological reactions. However, the lower gene-transfer efficiency was a critical hurdle for applying them to human gene therapy. Among a number of nonviral vector systems, our studies focus on hydrodynamic gene delivery to utilize physical force to deliver naked DNA into the cells in the living animals. This method achieves a high gene-transfer level by DNA solution injections into the tail vein of rodents, especially in the liver. With the development of genome editing methods, in vivo gene-transfer therapy using this method is currently the focus in this research field. This review explains the method principle, efficiency, safety, and procedural modifications to achieve a high level of reproducibility in large-animal models.
- リンク情報
- ID情報
-
- DOI : 10.3748/wjg.v22.i40.8862
- ISSN : 1007-9327
- eISSN : 2219-2840
- Web of Science ID : WOS:000386598800002