Abstract
Various metabolic and monogenic diseases have genetic defects in the liver and are often caused by lack or overproduction of a critical gene product in its cells. Conventional therapeutic methods are often insufficient or unavailable to manage genetic diseases in the liver; therefore, more efficient strategies are urgently needed. Gene therapy emerged as novel method of treatment relying on liver-directed transfer of a gene-coding sequence to produce the missing gene products or a nucleic acid sequence to inhibit the production of the gene product in excess. Practically, the success of the nucleic acid-based approaches is dependent on the availability of a method capable of delivering the gene or nucleic acid therapeutics to liver cells. Various methods of gene delivery have been developed, including viral methods and nonviral methods comprising synthetic vectors and physical methods. Hydrodynamic gene delivery is among the most efficient and the most commonly used method for liver-directed gene delivery. Here we will briefly summarize the principles and the progress that have been made in hydrodynamic gene delivery toward clinical applications.
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Acknowledgment
The authors would like to thank Enago for proofreading the manuscript and Ms. Ryan Fugett for English editing. This work was supported in part by Grant-in-Aid for Scientific Research from the Japanese Society for the Promotion of Sciences 22890064, 23790595, and 26860354, and Takara Bio Research Grant from JSGT, and Adaptable and Seamless Technology Transfer Program through target-driven R&D, JST (AS251Z01854P), and AstraZeneca R&D Grant to Kenya Kamimura and from the National Institute of Health (EB002946 and HL075542) to Dexi Liu.
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Kamimura, K. et al. (2016). Image-Guided Hydrodynamic Gene Delivery to the Liver: Toward Clinical Applications. In: Terai, S., Suda, T. (eds) Gene Therapy and Cell Therapy Through the Liver. Springer, Tokyo. https://doi.org/10.1007/978-4-431-55666-4_8
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DOI: https://doi.org/10.1007/978-4-431-55666-4_8
Publisher Name: Springer, Tokyo
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