Hepatic Diseases Gene Therapy

The Gene Therapy of Liver Diseases Group at Cima focuses its research on two lines of research:

Gene therapy of rare liver diseases of genetic origin: study of diseases whose origin is in the liver and in particular those hereditary diseases that can be cured by liver transplantation. We work to understand the pathophysiology of some of these rare diseases and to develop new therapies through strategies based on gene transfer and gene editing techniques that can offer solutions to these pathologies.

Study and development of new treatments for chronic viral hepatitis: we seek to deepen the knowledge of the pathology associated with infection by hepatitis B virus (HBV) and hepatitis Delta virus (HDV) to develop new treatments for these chronic infections.
To carry out this research, our group strives to improve the gene transfer vehicles, thanks to a better knowledge of the vector at the molecular level and of different aspects that affect its efficacy. On the other hand, thanks to the use of adeno-associated viruses as vehicles to transfer genes to the liver, we have developed animal mouse models that reproduce some of the most important characteristics of these diseases in humans.