Breakthrough in Disease Modeling and Cell-Based Therapy

By creating diseased liver cells from a small sample of human skin, scientists have for the first time shown that stem cells can be used to model a diverse range of inherited disorders. The University of Cambridge (United Kingdom) researchers’ findings may soon lead to new treatments for those suffering from liver diseases.  For their research, the scientists took skin biopsies from seven patients who suffered from a variety of inherited liver diseases and three healthy individuals (the control group). They then reprogrammed cells from the skin samples back into stem cells. These stem cells were then used to generate liver cells which mimicked a broad range of liver diseases – the first time patient-specific liver diseases have been modelled using stem cells – and to create ‘healthy’ liver cells from the control group. Importantly, the three diseases the scientists modelled covered a diverse range of pathological mechanisms, thereby demonstrating the potential application of their research on a wide variety of disorders. Writing that: “We report a simple and effective platform for hepatocyte generation from patient-specific human [induced pluripotent stem] cells,” the team concludes that: “These patient-derived hepatocytes demonstrate that it is possible to model diseases whose phenotypes are caused by pathological dysregulation of key processes within adult cells.”