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ERC funding: How to deliver gene therapies to a specific target site?

No. 52 | 21/09/2022 | by Koh

With its "Proof of Concept" grants, the European Research Council ERC supports scientists in further developing the commercial potential of their research results. Nina Papavasiliou from the German Cancer Research Center (DKFZ) is now receiving the prestigious grant for the second time: she wants to advance the development of a "molecular delivery service" that ensures that therapeutic genes reach the right address in the body in a targeted manner. One of the aims of this project is to develop more targeted cancer vaccines.

Nina Papavasiliou
© Jutta Jung / DKFZ

What is special about the ERC's "Proof of Concept" grants is that only those who already receive ERC funding can apply for it. The Research Council thus wants to enable scientists to test and develop possible areas of application for their research results obtained in an ERC-funded project.

On the basis of her ERC Consolidator Grant, which was approved in 2016, Nina Papavasiliou has now already been able to acquire the second ‚Proof of Concept' grant. Her current idea: to channel therapeutic genes as precisely as possible into target cells, she relies on a type of artificial exosomes. Biologists use this term to describe tiny membrane vesicles that are secreted by the cell and can carry a molecular cargo inside them.

Papavasiliou wants to equip the membrane of these vesicles with molecules that dock precisely to certain surface proteins of the target cells. The docking maneuver causes the vesicle and the target cell to fuse, and the molecular cargo finds its way into the interior of the target cells.

The DKFZ researcher uses the membranes of the blood parasite Trypanosoma brucei, the pathogen that causes sleeping sickness, for the artificial exosomes. These membranes are covered in extremely high density with a surface protein of the pathogen. Using an enzymatic reaction Papavasiliou can bind any molecules to this surface protein that enables precise targeting of all types of target cells.

For gene therapies, the vesicles could be loaded with therapeutic genes and thus represent an alternative to the viral gene ferries that have been mostly used up to now but are often problematic. For RNA vaccinations against cancer cells, it would also be possible to transport mRNA molecules inside the vesicles. By binding the vesicles to a cancer-specific protein, the mRNA molecules would be specifically delivered only into the tumor cells.

Biologist Nina Papavasiliou received her PhD from Rockefeller University in New York in 1998, where she headed an immunology research department until 2015. Since 2016, the U.S. American has headed the Division Immune Diversity at the German Cancer Research Center. For her outstanding research achievements, Nina Papavasiliou was accepted as a member of the European Molecular Biology Organization (EMBO) in 2020. In 2021, she was awarded the first-ever DKFZ Innovation Award.

A picture of Nina Papavasiliou is available for download: 

Note on use of images related to press releases
Use is free of charge. The German Cancer Research Center (Deutsches Krebsforschungszentrum, DKFZ) permits one-time use in the context of reporting about the topic covered in the press release. Images have to be cited as follows: "Source: Jutta Jung / DKFZ ".
Distribution of images to third parties is not permitted unless prior consent has been obtained from DKFZ's Press Office (phone: ++49-(0)6221 42 2854, E-mail: Any commercial use is prohibited.

With more than 3,000 employees, the German Cancer Research Center (Deutsches Krebsforschungszentrum, DKFZ) is Germany’s largest biomedical research institute. DKFZ scientists identify cancer risk factors, investigate how cancer progresses and develop new cancer prevention strategies. They are also developing new methods to diagnose tumors more precisely and treat cancer patients more successfully. The DKFZ's Cancer Information Service (KID) provides patients, interested citizens and experts with individual answers to questions relating to cancer.

To transfer promising approaches from cancer research to the clinic and thus improve the prognosis of cancer patients, the DKFZ cooperates with excellent research institutions and university hospitals throughout Germany:

  • National Center for Tumor Diseases (NCT, 6 sites)
  • German Cancer Consortium (DKTK, 8 sites)
  • Hopp Children's Cancer Center (KiTZ) Heidelberg
  • Helmholtz Institute for Translational Oncology (HI-TRON Mainz) - A Helmholtz Institute of the DKFZ
  • DKFZ-Hector Cancer Institute at the University Medical Center Mannheim
  • National Cancer Prevention Center (jointly with German Cancer Aid)
The DKFZ is 90 percent financed by the Federal Ministry of Education and Research and 10 percent by the state of Baden-Württemberg. The DKFZ is a member of the Helmholtz Association of German Research Centers.


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