Novel gene scalpels repair defective genetic material

„Hundreds of teams around the world are working flat out to repair genetic defects in the genome or DNA using molecular scissors“, explains molecular biologist Felix Lansing. This method makes it possible to make targeted changes to the genome. The technology became known worldwide in 2020 when the Nobel Prize for Chemistry went to the developers of the gene scissors „CRISPR/Cas9“. „Despite all the progress, however, most of these scissors are far from perfect, as they often only cut the DNA but do not repair it. The repair must then be carried out by the cell itself, which is often flawed," Lansing explains the problem with CRISPR/Cas9, for example. In addition, gene scissors could only edit smaller sections of the genome and would therefore only offer a chance of a cure for some patients with genetic diseases.

In his dissertation, Lansing has now developed the scissors into a scalpel. The result: a new method for treating haemophilia A (bleeding disorder). Those affected lack a certain protein in their genetic make-up, which is why their blood does not clot on its own - a life-threatening danger in the event of injuries or operations. An ideal correction would cure a patient of the severe form of haemophilia A. Additional protein replacement therapy would only be necessary, if at all, in the case of an imminent operation or the most severe injuries.

Lansing's gene scalpel cuts precisely and repairs flawlessly at the same time. „The work is done by enzymes or recombinases. For example, the so-called Cre recombinase can cut, insert, rotate or invert DNA sections and even replace them," he explains.

Among the 8,000 genetic diseases, it is primarily haemophilia, autoimmune diseases and retinitis pigmentosa - a group of retinal diseases in which the photoreceptors gradually die - that have a chance of being cured by gene scalpels. Since 2020, Lansing has been transferring the results of his doctorate into the development and implementation of preclinical testing of recombinases for the treatment of Hämophilia A in the GO-Bio RecTech project in Dresden. His work paves the way for the broad application of the technology – for the beginning of a new era of genome surgery.

Felix Lansing (32) studied molecular biology at the University of Bielefeld and the Technical University of Dresden from 2011 to 2016, where he also completed his doctorate from 2017 to 2021. The recombinases he has developed for the treatment of haemophilia A are currently being tested in preclinical studies before they are approved for clinical trials.

Source: Press release Körber Stiftung from 19 December 2022