GENE-THERAPY FOR RENAL DISEASES

Gene therapy is a promising therapeutic approach for a variety of rena l diseases including both inherited and acquired diseases. In vivo gen e transfer in the kidney using viral or non-viral vectors have been re ported. These approaches have been tested in a few animal models of re nal diseases, including experimental glomerulonephritis, ischemic rena l failure, and carbonic anhydrase II deficiency. Selection of vectors, routes, and therapeutic genes is critical to the success of gene ther apy targeted to the specific compartment of the kidney. Limitations of gene therapy for renal diseases exist and consist of: duration of tra nsgene expression is short, transfection efficiency is not adequate, i mmune reactions are induced by adenoviral vector, and insertional muta genesis may be caused by retroviral and adeno-associated viral vectors . Further studies are needed for improvement of gene delivery, minimiz ation of side effects and development of cell-specific and long-term r egulated gene expression.