Massimiliano Paganelli did his medical studies at the University of Rome “La Sapienza”. He started caring for children with liver diseases in 2005 during his first fellowship in pediatric hepatology at the University of Naples “Federico II”. During his training in pediatrics at “La Sapienza” university, Dr Paganelli cultivated such an interest in gastrointestinal and liver diseases of children with an intense clinical and research activity in one of the leading specialized department in Europe. He then moved to Brussels for 18 months of subspecialty training in hepatology and liver transplantation at Cliniques Saint-Luc in Brussels (a pioneer center for pediatric liver transplantation). In Brussels, Dr Paganelli started focusing on liver cell transplantation and cell therapy for liver diseases. He did a PhD in pediatric hepatology and cell therapy at the Université catholique de Louvain, working on in vitro differentiation of stem cells to hepatocytes, in vitro disease modeling with stem cells-derived hepatocytes, cell therapy for inborn errors of liver metabolism. His researches led to several prizes, presentations at international meetings and scientific publications. Subsequently Dr Paganelli refined his clinical training with a fellowship in pediatric gastroenterology and nutrition at CHU Sainte-Justine, where he was recruited to continue his clinical activity as pediatric gastroenterologist/hepatologist (with a special focus on liver transplantation) and carry on his research on the use of stem cells for the treatment of liver diseases. Research Interests Dr Paganelli’s research aims at treating liver diseases by the means of cellular therapy. His lab generates induced pluripotent stem (iPS) cells from the blood of patients with liver diseases in order to achieve the following objectives: In vitro differentiation to hepatocyte-like cells and establishment of cellular models of the different diseases. Such models are used to study the pathophysiology of the disease, try new treatments, and assess the effect of known drugs in order to predict in vivo response (personalized medicine). Genome editing by CRISPR/Cas9 technology to correct the disease-causing mutation (for inborn errors of liver metabolism), with subsequent expansion of the corrected population, differentiation to hepatocyte-like cells and transplantation. Such an approach aims at restoring the deficient function eliminating the need for liver transplantation. Generation of liver organoids with tridimensional structure and function comparable to the human liver. Such liver buds will be created from corrected iPS obtained from patients with metabolic liver disease. Once their creation protocol and culture conditions improved and their safety proven, liver organoids might be transplanted to patients to replace their diseased liver. The projects currently ongoing in the lab imply the study of iPS cells-to-hepatocyte differentiation process, the creation and validation of an in vitro model of hereditary tyrosinemia type I, the correction of the disease-causing mutation in such a model, the assessment of the efficacy and safety of the transplantation of hepatocytes derived form corrected iPS cells in a tyrosinemia mouse model. Dr Paganelli takes part in several clinical studies on liver diseases he is confronted with in the daily practice, with a special interest for liver transplantation, neonatal cholestasis and chronic hepatitis B.