Project presentation 2019

The animation or cover image shows the innovative "prime editing" process for precisely correcting DNA changes in cells. In this process, the CRISPR/Cas9 system is guided precisely to the DNA sequence to be modified by means of a specially extended guide RNA, a so-called prime editing guide RNA (pegRNA), where the opposite strand of the double helix is cut by the Cas9 nickase. By linking the system with a reverse transcriptase, a correction sequence can be specified in the extended part of the pegRNA, which then serves as a blueprint during DNA repair.

To investigate the safety of the process, we use a reporter system that indicates successful repair with red fluorescence and undesired incorrect repair with green fluorescence. This method is currently being used in research projects to correct disease-specific mutations in stem cells or differentiated (liver) cells derived from them. However, the aim of the work is also to investigate future forms of application directly in the organism.

The animation was created by Prof. Dr. med. Tobias Cantz (Translational Hepatology and Stem Cell Biology working group) from the Clinical Department of Gastroenterology, Hepatology and Endocrinology. The work of the research group is part of the REBIRTH Center for Translational Regenerative Medicine and aims to gain a better understanding of the mechanisms of liver development and regeneration as well as pathophysiological changes in hepatic diseases and make them the target of innovative treatment strategies.

Until the Research Information System (FIS) is introduced, research reports are not possible due to technical challenges. However, we would like to take the opportunity here to explicitly present a representative project.