"Novel Treatment Approaches Unveiled for Polycystic Kidney Disease"

Scientists have made significant progress in understanding and potentially treating polycystic kidney disease (PKD) by using gene editing and 3-D human cell models to study the genetics of the disease. They discovered that a single normal gene copy can prevent the formation of cysts in PKD, offering potential for tailored gene therapy. Additionally, a type of drug called glycoside was found to counteract the effects of the defective gene. These findings could lead to new therapeutic approaches for treating PKD, which affects millions worldwide. The study was partially funded by the National Institutes of Health (NIH) and is published in Cell Stem Cell.