Inborn errors of liver metabolism are liver diseases caused by a gene mutation entailing multiple severe and often lethal consequences. A treatment is available for only few of such conditions, mostly to reduce symptoms and almost never to cure the disease. Our research aims at treating such disorders by the means of cell transplantation, thus curing the disease while freeing the patient from the need of liver transplantation, lifelong treatments and compelling diets.
We generate stem cells (called induced pluripotent stem cells, or iPSC) from the patients’ blood, and using them to generate liver cells (the hepatocytes). We use these hepatocytes to establish a model of patients’ liver disorders, in order to study the diseases and test new drugs. We work to correct the mutation causing the diseases in the patients’ iPSC (genome editing). Edited cells are used to generate hepatocytes, which are be identical to the patients’ liver cells except that they are “healthy”. The final aim is to transplant such hepatocytes back to the patients in order to cure their disease (“autologous” transplantation, as the patient receives its
own cells, not needing any immunosuppression). This approach could be a significant step towards translating autologous cell therapy to the clinical practice, with the potential to dramatically change the quality of life of people with inborn errors of liver metabolism.
We are working on hereditary tyrosinemia type I as a proof of concept of many other inborn errors of liver metabolism. In the meanwhile, we are focusing on understanding and improving the differentiation potential of iPSC, working on their epigenetic profile. We are collaborating with other groups in order to establish in vitro models of several liver diseases to be used for pathophysiologic studies and drug testing. Last but not least, we are specializing in liver and gut organoid development for both disease modeling and cell therapy.
We built a young, dynamic, international environment, where everybody is committed to this ambitious translational project. The lab is well founded and recently received the competitive Gilead Research Scholar in Liver Disease Award. Taking advantage of the stimulating scientific environment of the Université de Montréal, we work in close proximity with the patients and the clinical team of the CHU Sainte-Justine, and we established several highly fruitful collaborations in Canada and abroad.