Gene therapy for kidney disease.

Gene therapy has distinct potential to treat disease at the most fundamental level. However, the ability to pursue gene therapy for renal disease has been limited by the availability of an adequate system for gene delivery to the kidney and for regulation of transgene expression. Presently, there are several limitations to overcome before clinical use of viral vector systems for targeting kidney can be considered. Non-viral vectors such as haemagglutinating virus of Japan (HVJ)-liposome mediated gene transfer and cationic liposome are promising but need to be improved. Given that the systemic delivery of the functional protein can serve as therapy for the renal diseases, skeletal muscle targeting gene therapy might be an alternative strategy for the treatment of renal disease. Gene therapy to the transplant kidney may potentially improve the graft outcome by reducing acute and chronic rejection. We review emerging strategies of gene transfer with reference to the kidney and discuss the potential application of gene therapy to renal diseases.