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. The terminal branches of the hepatic portal vein and hepatic artery enter the liver sinusoids, which are characterized by fenestrated and discontinuous endothelium. 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. Novel therapeutic strategies involve cell therapies, gene therapies and, most recently, also in vivo correction of the underlying gene defect.

The Guest Research Group Cell- and Gene Therapy is headed by Professor Michael Ott and develops novel therapeutic approaches for acute and hereditary liver diseases as well as chronic liver failure including hepatocellular carcinoma. 

Loss of hepatocytes and their respective hepatocyte functions is considered as the 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. 

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 final result of chronic injuries, which can be caused by a variety of stimuli 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/editing, stem cell based endogenous tissue engineering and therapeutic microRNAs to develop novel therapies for liver diseases. 


02. March 2016

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. In den meisten Fällen sind Infektionskrankheiten - wie Hepatitis C...