Article
Phenotypic correction of a mouse model for primary hyperoxaluria with adeno-associated virus gene transfer.
Molecular therapy : the journal of the American Society of Gene Therapy - 1 May 2011
Salido Eduardo, Rodriguez-Pena Marisol, Santana Alfredo, Beattie Stuart G, Petry Harald, Torres Armando
Abstract excerpt
Primary hyperoxaluria type I (PH1) is an inborn error of metabolism caused by deficiency of the hepatic enzyme alanine-glyoxylate aminotransferase (AGXT or AGT) which leads to overproduction of oxalate by the liver and subsequent urolithiasis and renal failure. The current therapy largely depends on liver transplantation, which is associated with significant morbidity and mortality. To explore an alternative...
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