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Health researchers to launch new technique for correcting genetic defects

Congenital genetic defects can lead to failures of the immune system, so it cannot defend the body against the microorganisms that we are constantly exposed to. Backed by a new grant, Professor Trine Mogensen and her team of researchers from AU and AUH are working to develop a new technology to correct these genetic defects.

A properly functioning immune system is vital to our survival, and this is particularly true in the early years of life. Immunodeficiency disorders contribute to many of the most severe congenital diseases, but now knowledge about the genetic basis for these diseases is so extensive that the exact genetic errors can be identified.

With a grant of DKK 25 million from the Innovation Fund Denmark, researchers from Aarhus University and Aarhus University Hospital will now further develop the new CRISPR/Cas technology, which has over the past five years made precise and effective editing of the human genome possible.

In the project, researchers will use what are known as a gene-scissors. This is a special enzyme that can cut the defective gene out of the patient's stem cells and replace it with the correct genetic information. In this way, the patient's defective immune system is replaced by immune cells which are formed from the corrected stem cells and therefore have the normal protective effect against infections.

The researchers behind the project hope that they will already be able to apply for and obtain permission to treat the first patients with the new technology during the first five years of the project.

With the grant from the Innovation Fund Denmark, a collaboration will be established between Aarhus University, where Rasmus Bak and Jacob Giehm Mikkelsen work with the molecular-genetic methods for correcting the genetic errors, and Aarhus University Hospital, where Trine H. Mogensen, Peter Hokland and Bjarne K. Møller work with immunodeficient patients and transplantation of hematopoietic stem cells.

Together, this research group could bring the CRISPR/Cas technology all the way from the laboratory to the individual patient.


Professor, Specialty Registrar Trine H. Mogensen
Aarhus University, Department of Biomedicine and Department of Clinical Medicine
Aarhus University Hospital, Department of Infectious Diseases
Email: trine.mogensen@biomed.au.dk
Mobile: (+45) 2012 5280