Markus Grompe is a Senior Professor at Oregon Health & Science University, where his research integrates cell biology, hepatology, genetics, and molecular biology to address liver disease and gene therapy. His recent work focuses on advancing in vivo gene editing and delivery platforms, including lipid nanoparticle-mediated base editing in the pancreas and AAV capsid optimization in steatotic human livers, as well as lentiviral vector potency for hepatocyte-targeted therapy. He also investigates disease-specific constraints in hepatocyte gene editing and develops large-animal models, such as a porcine model of Fanconi anemia, to assess therapeutic toxicity and efficacy. His contributions have been recognized with major funding, including a 2016 award for metformin therapy in Fanconi anemia, and his international collaborations span translational gene therapy and liver physiology.
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