Cell and Gene Therapy

Cell and Gene Therapy

The liver, the largest internal organ of the body, consists of exceptional anatomical structures in both biliary system and vasculature and can react to injury with a remarkable regenerative capacity. The biliary system, an exocrine system in the liver, connects the apical surface of every single hepatocyte to the duodenum through bile canaliculi, which drain into the canals of Hering and finally into bile ducts. Hepatocytes compose the parenchyma of the liver with their many functions for the body including protein carbohydrate and lipid metabolism as well as hormonal activities. It is thus not surprising that most of the hereditary liver diseases relate to genes expressed mainly in hepatocytes. For hereditary liver diseases novel therapeutic strategies are being developed, which are based on cell therapies, gene therapies and in vivo correction of the underlying gene defect. Additionally, the group participates in international clinical studies and tests cell- and gene therapy approaches as a translational effort to improve the life of patients with these often deadly metabolic diseases. 

Loss of hepatocytes and their respective hepatocyte functions is considered a hallmark of acute liver failure (ALF). Although the regenerative capacity of the liver is only temporary insufficient, hepatic parenchymal functions below a certain threshold can result in systemic organ failure and death of the patient. Our group aims to identify novel drugs, which can block the cell death in ALF and lead to increased survival of patients with acute liver failure.

Persistent injury of the liver initially induces regenerative tissue responses but eventually results in scarring and excess deposition of extracellular matrix components including collagen. Fibrosis and cirrhosis of the liver are the result of chronic injuries including metabolic disorders, persistent infections, autoimmune reactions, allergic responses, chemical insults or radiation. Chronic liver failure is also the most prominent risk factor for hepatocellular carcinoma, which ranks third amongst the leading causes of cancer-related deaths worldwide. In our group we apply technologies such as cellular reprogramming, gene transfer/gene editing, stem cell based endogenous tissue engineering and microRNAs to develop novel therapies for end stage chronic liver diseases. 


Reinhart Koselleck DFG project for Michael Ott

Research funding for forward-looking ideas regarding liver-transplantation - Jährlich sterben etwa 70.000 Menschen allein in Europa an Leberversagen.…