Gene therapy in renal diseases.
Kidney-targeted gene therapy could be an ideal treatment for renal diseases since the therapeutic molecule is limited in the kidney and the systemic effect may be minimized. The technical development of the gene delivery to kidney and the identification of the responsive gene for a particular disease encourage the challenge to hereditary diseases. Collagen type IV reassembling was reported to be succeeded in Alport syndrome model by introduction of exogenous COL4A5 gene. Many gene therapies are evaluated in various glomerulonephritis models and unilateral ureteral obstruction (UUO) model, and favorable results are accumulated. Transplant kidney is an ideal target for gene therapy, by which ischemia reperfusion, acute rejection and chronic allograft nephropathy can be treated. The importation of the novel technology, for example hybrid stem cell-gene therapy could promote the gene therapy of renal diseases toward clinical application.
Imai
Enyu
E
Division of Nephrology, Department of Internal Medicine, Osaka University Graduate School of Medicine, Japan. imai@medone.med.osaka-u.ac.jp
Takabatake
Yoshitsugu
Y
Mizui
Masayuki
M
Isaka
Yoshitaka
Y
D000818:Animals; D024141:Collagen Type IV / Q000235:genetics; D015316:Genetic Therapy; D005921:Glomerulonephritis / Q000628:therapy; D006801:Humans; D007108:Immune Tolerance; D007674:Kidney Diseases / Q000235:genetics / Q000628:therapy*; D016030:Kidney Transplantation / Q000276:immunology; D009394:Nephritis, Hereditary / Q000235:genetics / Q000628:therapy
C478159:COL4A5 protein, human; D024141:Collagen Type IV