Potential New Therapies for Untreatable Kidney Disease
The best hope for people with an inherited form of kidney disease that causes kidney failure is dialysis or a kidney transplant. But a study led by Yale researchers reveals a potential strategy for developing new drug therapies for these patients.
They used mouse models and human tissue samples to study one of the two mutated genes that lead to autosomal dominant polycystic kidney disease (ADPKD). This form of kidney disease is the most commonly inherited type and difficult to treat. The researchers focused their investigation on measuring the production of energy in kidney cells affected by the disease.
They discovered that when the gene for the protein called Polycystin 2 is turned off or missing, cellular energy ramps up, leading to the formation of cysts that damage the kidneys.