Gentherapie von hereditären Lebererkrankungen

Gene therapy has been explored for the treatment of hereditary monogenic liver diseases. It includes gene repair, gene replacement as well as hepatocyte, stem cell or liver transplantation. These strategies have been successfully explored in preclinical in vivo models of hereditary monogenic liver diseases as well as in individual patients. While their principle and efficacy has been demonstrated, in principle, numerous problems need to be solved before gene therapy will complement or replace existing therapeutic options. Furthermore, recent developments in the field of stem cell biology with the generation of individual stem cells from skin fibroblasts will certainly also have an impact on further developments in gene therapy in general and stem cell transplantation in particular.