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Parameters Affecting Image-guided, Hydrodynamic Gene Delivery to Swine Liver
Kenya Kamimura1, Takeshi Suda, Guisheng Zhang
11] Division of Gastroenterology and Hepatology, Graduate School of Medical and Dental Sciences, Niigata University, Niigata, Niigata, Japan [2] Department of Pharmaceutical Sciences, University of Pittsburgh, School of Pharmacy, Pittsburgh, Pennsylvania, USA.
Molecular Therapy. Nucleic Acids
|October 17, 2013
Summary
This study optimized image-guided hydrodynamic gene delivery in large animals for liver disease gene therapy. The safe, effective method achieved sustained gene expression, supporting clinical applications.
Area of Science:
- Hepatology
- Gene Therapy
- Medical Imaging
Background:
- Developing safe and effective gene delivery to hepatocytes is crucial for treating liver diseases.
- Hydrodynamics-based gene delivery offers potential but requires optimization for large animals.
Purpose of the Study:
- To assess parameters for image-guided hydrodynamic gene delivery in large animals (pigs).
- To optimize injection parameters for safety and efficiency in liver gene therapy.
- To explore sequential injections into multiple liver lobes.
Main Methods:
- Utilized image-guided catheter insertion into the lobular hepatic vein.
- Employed a computer-controlled injector for hydrodynamic plasmid injection.
- Assessed parameters including catheter position, intravascular pressure, and injection volume.
Main Results:
- Injection parameters directly correlated with procedure safety and efficiency.
- Optimized procedure enabled sequential injections into multiple lobes in human-sized pigs.
- Sustained expression of human alpha-1 antitrypsin gene for over 2 months.
- Repeated hydrodynamic gene delivery was safely performed without adverse events.
Conclusions:
- Optimized image-guided hydrodynamic gene delivery is safe and effective in large animals.
- The method demonstrates potential for clinical application in treating liver diseases.
- Sustained gene expression and safety support further development for therapeutic use.
