Crucial Step Towards Future Gene Medicine – University of Copenhagen

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10 May 2017

Crucial Step Towards Future Gene Medicine

Gene medicine

The leading pharmaceutical companies are listening, as new research conducted by the University of Copenhagen and Roskilde University, among others, may bring the development of gene medicine a crucial step forwards.

Researchers are constantly looking for new medicine and new ways of fighting diseases like e.g. cancer. Gene medicine constitutes a promising area of research, and researchers from the University of Copenhagen and Roskilde University, among others, have now for the first time ever managed to remedy the unwanted effects associated with the regulation of modified gene activity.

The new method has solved the problems previously facing researchers trying to develop this type of gene medicine. The researchers have previously been unable to fully control modified genes, e.g. preventing gene activity. So-called leaks occur when the researchers try to turn gene activity on or off, and that has limited the clinical application of the method. 

These and other problems can be remedied using the new ground-breaking method ’precise integration of inducible transcriptional elements’, also called PRIITE.

Expect Great Interest in the New Method
Professor Jesper Troelsen, who is head of the Gastro research group at the Department of Science and Environment at Roskilde University recognises the great potential of the new method.

‘We have discovered a method that is reliable – nothing unintentional happens in this process. We are able to activate and control genes in the intestine cells. Now we know exactly where in the genome to insert the controllable cells to prevent them from causing harm, and we are able to regulate the entire process’, says Jesper Troelsen.

Jesper Troelsen believes the method will be crucial to the development of future gene medicine, precisely because it is so reliable. And it can be used in the development of a range of products –  from the production of hormones to therapeutic antibodies fighting various forms of cancer. Cancer researchers in particular focus on the development of gene medicine, where the treatment is adjusted to the individual patient to a far greater extent, and here the new methods for precise regulation represent a crucial step forwards.

‘This affects both the application in academic contexts, but also in what we call translational clinical contexts, where such a crucial breakthrough in basic research will have an immediate effect through our cooperation with hospitals and the pharmaceutical industry’, Jesper Troelsen explains. ‘And it is a method that is very illustrative in teaching contexts and may even be demonstrated to upper secondary school students visiting the department’.

The research was conducted at the University of Copenhagen and Roskilde University, but has also involved collaborators at Herlev Hospital and in Portugal. The results have been published in the acclaimed journal Nucleic Acid Research’

Test Conducted on Intestine Cells
The method was developed by Associate Professor Eric Paul Bennett’s research group at the Copenhagen Center for Glycomics at the University of Copenhagen. It is based on the application of gene editing techniques, though which the researchers make changes to the genes. CRISPR is probably the most well-known of these techniques, but in this project gene editing was conducted in a far more efficient way using the similar ZFN method.

‘It was important for us to use our own gene editing expertise on a clinically relevant biological problem. We therefore contacted Jesper Troelsen’s group at Roskilde University, which works on gene editing in connection with intestine diseases’, Eric Paul Bennett explains.

The study is ground-breaking in its approach to solving previous problems connected to controlled regulation of gene activity. The validation of the PRIITE method has required extensive genome analyses conducted by the Gastro group at Roskilde University.

The Gastro group at the Department of Science and Environment is especially interested in the CDX2 gene, which was chosen as a gene model for the PRIITE platform. The method can easily be adjusted to other genes. CDX2 is the protein that controls the maturation of ordinary intestine cells by activating the production of enzymes and proteins that are necessary for digestion. In connection with colon cancer our immune system causes the amount of CDX2 proteins in the intestine cells to decrease, but this may cause the cancer cells to move into other parts of the body. This can make the cancer spread, and it is therefore important to be able to regulate the amount of CDX2 in the intestine cells.

In addition to Professor Jesper Troelsen, PhD Student Sylvester Larsen, PhD Student Johanne Davidsen and Associate Professor Cathy Mitchelmore from the Gastro group at Roskilde University also participated in the research project. The study has been published in the journal Nucleic Acid Research.


Contact
Associate Professor Eric Paul Bennett, email: epb@sund.ku.dk, phone: +45 35 32 66 30
Professor Jesper Thorvald Troelsen, email: troelsen@ruc.dk, phone: +45 46 74 27 28