Research suggests avenues toward gene therapies for polycystic kidney disease
New Haven, Conn. — Autosomal dominant polycystic kidney disease (ADPKD) is the most common potentially lethal genetic disease—about a half million people in the United States alone suffer from the condition. There is no cure, but new research could open the door to new gene therapies for treating most cases of the disease.
For several decades, researchers have known that mutations in the PKD1 gene, which encodes the polycystin-1 (PC1) protein, can cause the disease in about 80% of cases. However, the protein is too big to be modified through gene therapy strategies. Now, a research ...













