Gene Therapy of Infectious Liver Diseases
The principal goal of our laboratory is to develop new treatments for chronic infection by the hepatitis B virus (HBV) and the hepatitis Delta virus (HDV), using therapeutic strategies based on gene transfer or gene therapy.
Although there is a vaccine capable of effectively preventing infection, more than 350 million people worldwide are currently chronic carriers of the hepatitis B virus, of whom 15 million are infected by HDV. These patients have a high risk of developing diseases that, in many cases, will cause death, such as cirrhosis and liver cancer. Current treatment for this disease, based on long-term administration of IFN-ɑ and antiviral drugs, is far from adequate. Many patients do not respond to the treatment and may experience major side effects, and many develop treatment-resistant viruses. For these reasons, the need to develop new therapies for this disease is evident.
The work we carry out in our laboratory can be divided into two areas of research:
Development of new animal models of infection with both viruses, which provide us with greater knowledge of the biology of the pathogen at the molecular level and its interaction with the host cell.
Analysis of the antiviral activity of viral vectors carrying therapeutic genes.
Collaborations with other groups
In collaboration with groups inside and outside of CIMA, we are working on the development of viral vectors for the transport of genes to other organs/tissues including brain, heart, eye and hematopoietic tissue.
"Our laboratory is working on developing new treatments for liver diseases, based on gene transfer using one of the safest and most effective viral vectors currently in existence: the adeno-associated virus. In the next few years, we hope to obtain a clear clinical benefit in patients with rare diseases such as Wilson's disease and hyperoxaluria", Dra. Gloria González, Program Director.