|
pubmed:abstractText |
Primary hyperoxaluria-type 1 (PH-1) is a rare autosomal recessive disorder of glyoxalate metabolism caused by deficiency in the liver-specific peroxisomal enzyme alanine-glyoxalate transaminase 1 (AGT) resulting in the increased oxidation of glyoxalate to oxalate. Accumulation of oxalate in the kidney and other soft tissues results in loss of renal function and significant morbidity. The present treatment options offer some relief in the short term, but they are not completely successful. In the present study, we tested the feasibility of corrective gene therapy for this metabolic disorder.
|
|
pubmed:affiliation |
Signal Transduction Laboratory, Program in Urosciences, Division of Urology, Department of Surgery, University of Colorado School of Medicine, Denver, Colo. 80262, USA. kari.koul@uchsc.edu
|
