TargetPOLG: Mechanisms of POLG-disease and approaches to therapy

POLG diseases are exceptionally variable in clinical manifestations most often affecting the nervous system, liver and the muscle. The TargetPOLG project will utilize novel mouse models and human materials to explore and identify molecular mechanisms that provoke manifestation of the POLG-disease manifestation and affect the progression. Our special focus is the brain and liver manifestations and modifying factors. We will use the molecular data to search for intervention targets, to be tested in our models, and when justified by the preclinical data, human pilot trials.