Renal diseases as targets of gene therapy.
A number of renal pathologies exist that have seen little or no improvement in treatment methods over the past 20 years. These pathologies include acute and chronic kidney diseases as well as posttransplant kidney survival and host rejection. A novel approach to treatment methodology may provide new insight to further progress our understanding of the disease and overall patient outcome. Recent advances in human genomics and gene delivery systems have opened the door to possible cures through the direct modulation of cellular genes. These techniques of gene therapy have not been extensively applied to renal pathologies, but clinical trials on other organ systems and kidney research in animal models hold promise. Techniques have employed viral and nonviral vectors to deliver gene modulating compounds directly into the cell. These vectors have the capability to replace defective alleles, express novel genes, or suppress the expression of pathogenic genes in a wide variety of kidney cell types. Focus has also been placed on ex vivo modification of kidney tissue to promote allograft survival and limit the resulting immune response to the transplanted organ. This could prove a valuable alternative to current immunosuppressive drugs and their deleterious effects on patients. While continued research and clinical trials are needed to identify a robust system of gene delivery, gene therapy techniques have great potential to treat kidney disease at the cellular level and improve patient quality of life.
Phillips
Brett
B
Division of Immunogenetics, Department of Pediatrics, University of Pittsburgh School of Medicine, Pittsburgh, Pa., USA.
Giannoukakis
Nick
N
Trucco
Massimo
M
D015316:Genetic Therapy / Q000379:methods*; D006085:Graft Survival; D006801:Humans; D007668:Kidney / Q000503:physiopathology; D007674:Kidney Diseases / Q000235:genetics* / Q000601:surgery / Q000628:therapy*; D016030:Kidney Transplantation; D014184:Transplantation, Homologous