Primary hyperoxaluria type I is the most common type of hyperoxaluria. It can begin at any age.
It is a rare liver disease caused by mutation in a gene, AGXT, which causes overproduction of a substance called oxalate (oxalic acid).
The excessive presence of this chemical compound, when bound to calcium, causes kidney and bladder stones. Patients present severe renal damage and the only curative treatment is liver transplantation, which in most cases must be accompanied by renal transplantation.
Increased levels of oxalate in the blood can lead to deposits of this substance in other tissues of the body (systemic oxalosis) and cause additional health problems.
At the Cima Universidad de Navarra we are working on the development of gene correction strategies to treat the disease.
We dream of curing hyperoxaluria
Treatment options have increased significantly in recent years, but much remains to be discovered.
We deepen our knowledge of the disease and develop gene therapy strategies to correct it.
We seek to optimize gene therapy vectors to enhance gene transfer efficiency.
We strive to bring our promising results to the patient as soon as possible.
REFERENCES IN TRANSLATIONAL RESEARCH
Hepatic Diseases Gene Therapy Research Group
Reference team in the development of strategies based on gene therapy for the treatment of rare liver diseases of genetic origin and the study and design of new treatments for chronic viral hepatitis.
It is part of the Gene Therapy and Regulation of Gene Expression Program.