New insights and therapeutic approaches of Polycystic Kidney and Liver Disease

Resumo

Polycystic kidney disease (PKD) is a group of monogenic disorders that result in renal cyst development. The principal extrarenal manifestation of PKD is the Polycystic Liver Disease (PLD). Since the genes and their encoded proteins were described, several in vitro and animal models were developed for the study of PKD and PLD and multiple molecular pathway were discovered and characterized. However, the central and key mechanism that trigger cyst formation remains unclear. In the same way, there is currently no fully efficient therapy for PKD and PLD. In this context, the present work has as principal aim find novel molecular pathways involved in the cystogenesis process, as well as the search of new therapeutic targets and its tested in orthologous animal models. In conclusion, the present thesis expand the knowledge about new insights and two new therapeutic approaches in the fields of PKD and PLD.