Gene therapy targeting kidney diseases: routes and vehicles.

摘要

Renal gene therapy may offer new strategies to treat diseases of native and transplanted kidneys. Several experimental techniques have been developed and employed using nonviral, viral, and cellular vectors. The most efficient viral vector for in vivo transfection appears to be adenovirus. In addition, enhanced naked plasmid techniques, such as the hemagglutinating virus of Japan (HVJ)-liposome method, electroporation, the hydrodynamic method, and ultrasound with microbubbles, are promising. Trapping genetically modified macrophages in the inflamed kidneys is an elegant method for site-specific gene delivery. The choice of delivery vehicle as well as the administration route determines the site of transduction. In conclusion, for both in vivo and ex vivo renal transfection, enhanced naked plasmids, adenoviruses, and modified cell vectors offer the best prospects for effective clinical application. Moreover, the development of safer and nonimmunogenic vectors may realize clinical renal gene therapy in the near future.