Polycystic liver diseases: advanced insights into the molecular mechanisms

摘要: Polycystic liver diseases are genetic disorders characterized by progressive bile duct dilatation and/or cyst development. The large volume of hepatic cysts causes different symptoms and complications such as abdominal distension, local pressure with back pain, hypertension, gastro-oesophageal reflux dyspnea well bleeding, infection rupture the cysts. Current therapeutic strategies based on surgical procedures pharmacological management, which partially prevent or ameliorate disease. However, these treatments only show short-term modest beneficial effects, transplantation is definitive therapy. Therefore, interest in understanding molecular mechanisms involved disease pathogenesis increasing so that new targets for therapy can be identified. In this Review, underlying polycystic most relevant pathways cystogenesis discussed. Moreover, main clinical preclinical studies highlighted future directions basic research indicated.