Genome editing helps decipher a congenital liver disease

Congenital hepatic fibrosis (CHF) is a rare genetic disease that can lead to severe liver impairment. The symptoms of CHF are distinct compared to other liver ailments, and while the PKHD1 gene has been implicated, the exact molecular basis for the unique disease pathology is unknown. Using a CRISPR-generated model, researchers at Tokyo Medical and Dental University (TMDU) identified molecular targets contributing to CHF. These targets may offer new therapeutic approaches to the disease.
Source: EurekaAlert,