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Departments

Centre for the Technologies of Gene and Cell Therapy

Our goal is to develop diverse biomedical tools aimed at preparing therapeutic solutions for genetic diseases and difficult-to-treat cancers. We will develop new technologies for gene and cell therapies and immunotherapies.

The main focus of our work is the comprehensive translational process, which systematically links basic science with actual clinical application. Development follows the principle of "from the lab bench to the hospital bed" – from the identification and validation of therapeutic targets, the development and optimization of delivery systems, and testing in advanced preclinical models, to the preparation of candidates for clinical trials. We pay special attention to safety, reproducibility, and compliance with regulatory requirements, thereby ensuring realistic possibilities for transferring therapies into the clinical environment.

A key part of translation is collaboration with leading physicians who help shape research priorities based on the actual needs of patients. Regular expert meetings, joint research planning, and the inclusion of clinical data ensure that the solutions developed are clinically relevant and feasible.

At the same time, we actively collaborate with patient associations, which represent the indispensable voice of healthcare system users. This ensures a better understanding of the burden of disease, ethical considerations, and the expectations of patients and their families. This integration of science, clinical practice, and patients creates a solid translational platform for the development of advanced, safe, and accessible therapeutic solutions.

More information on our webpage: https://ctgct.si/en/

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Representative Publications

  1. Ivanovski, F., Jazbec, V., Varda, N., Jerala, R., & Benčina, M. (2025). Ultrasound-responsive mammalian cell synthetic biology. IEEE Transactions on Ultrasonics, Ferroelectrics, and Frequency Control, 1–1. https://doi.org/10.1109/TUFFC.2025.3570813
  2. Frank, Julia Katharina, Kampleitner, Carina, Heimel, Patrick, Leinfellner, Gabriele, Hanetseder, Dominik, Sperger, Simon, Frischer, Amelie, Schädl, Barbara, Marolt  Presen, Darja, et al (2025). Circulating miRNAs are associated with successful bone regeneration. Frontiers in bioengineering and biotechnology. 2025, vol. 13, str. 1-15. https://www.frontiersin.org/journals/bioengineering-and-biotechnology/articles/10.3389/fbioe.2025.1527493/full.
  3. LAPANJA, Tjaša, MICHELI, Pietro, GONZALEZ-GUERRA, Andres, RADOMSKYI, Oleksandr, DE FRANCESCHI, Gioia, MURAVEVA, Anna, ATTINGER, Alexander, ROTH, Chiara Nina, TRIPODI, Matteo, BOISSONNET, Tom, et al. Pupil size modulation drives retinal activity in mice and shapes human perception. Nature communications. 2025, vol. 16, issue 1, article no. 7334, str. 1-19, ilustr. ISSN 2041-1723. https://www.nature.com/articles/s41467-025-62736-4.
  4. LEVSTEK, Tina, BREZNIK, Nika, BALANT MARIN, Kaja, PODKRAJŠEK, Tisa, VUJKOVAC, Bojan, NOWAK, Albina, OLIVEIRA, João-Paulo, DOSTÁLOVÁ, Gabriela, LINHART, Aleš, ŠAFAŘÍKOVÁ, Marketa, ALTARESCU, Gheona, TREBUŠAK PODKRAJŠEK, Katarina. A non-coding signature in SHROOM3 is associated with kidney disease progression in Fabry disease. Molecular genetics and metabolism. [Online izd.]. Jan. 2026, vol. 147, iss. 1, [article no.] 109710, str. 1-9, ilustr. ISSN 1096-7206. https://www.sciencedirect.com/science/article/pii/S1096719225007024?via%3Dihub,
  5. LEVSTEK, Tina, BAHČIČ, Erazem, VUJKOVAC, Bojan, COKAN VUJKOVAC, Andreja, TESOVNIK, Tine, REMEC, Žiga Iztok, ČUK, Vanja, TREBUŠAK PODKRAJŠEK, Katarina. Telomere length, oxidative stress, and kidney damage biomarkers in Fabry nephropathy. Cells. 2025, vol. 14, no. 3, str. 1-15, ilustr. ISSN 2073-4409. https://www.mdpi.com/2073-4409/14/3/218.
  6. LEVSTEK, Tina, VUJKOVAC, Bojan, NOWAK, Albina, OLIVEIRA, João-Paulo, DOSTÁLOVÁ, Gabriela, LINHART, Aleš, ŠAFAŘÍKOVÁ, Marketa, ALTARESCU, Gheona, TREBUŠAK PODKRAJŠEK, Katarina. Identification of genetic variants associated with Fabry nephropathy progression using whole-exome sequencing. Molecular genetics and metabolism. 2025, vol. 145, iss. 4, [article no.] 109191, str. 1-5. https://www.sciencedirect.com/science/article/abs/pii/S1096719225001829?via%3Dihub.
  7. Presen, D. M., Lainšček, D., Kinghorn, J., Sebestyen, Z., Kuball, J., Amini, L., Reinke, P., Fuchs, A., Jerala, R., & Benčina, M. (2024). CTGCT, Centre of Excellence for the Technologies of Gene and Cell Therapy: Collaborative Translation of Scientific Discoveries into Advanced Treatments for Neurological Rare Genetic Diseases and Cancer. Computational and Structural Biotechnology Journal. https://doi.org/10.1016/j.csbj.2024.11.051
  8. KIELER, Markus, PRAMMER, Leona Sophia, HELLER, Gerwin, HOFMANN, Melanie, SPERGER, Simon, HANETSEDER, Dominik, NIEDERREITER, Birgit, KOMLJENOVIC, Andrea, MAROLT PRESEN, Darja, et al. Itaconate is a metabolic regulator of bone formation in homeostasis and arthritis. eARD. Elektronska izd. 2024, vol. 83, iss. 11, str. 1465-1479. https://ard.bmj.com/content/early/2024/07/10/ard-2023-224898, https://pubmed.ncbi.nlm.nih.gov/38986577/.
  9. MAROLT PRESEN, Darja, GOESCHL, Vanessa, HANETSEDER, Dominik, OGRIN, Laura, STETCO, Alexandra-Larissa, TANŠEK, Anja, POŽENEL, Laura, BRUSZEL, Bella, MITULOVIĆ, Goran, OESTERREICHER, Johannes, ZIPPERLE, Johannes, et al. Prolonged cultivation enhances the stimulatory activity of hiPSC mesenchymal progenitor-derived conditioned medium. Stem cell research & therapy. 2024, vol. 15, str. 1-22. https://stemcellres.biomedcentral.com/articles/10.1186/s13287-024-03960-5.
  10. Ivanovski, F., Meško, M., Lebar, T., Rupnik, M., Lainšček, D., Gradišek, M., Jerala, R., & Benčina, M. (2024). Ultrasound-mediated spatial and temporal control of engineered cells in vivo. Nature Communications, 15(1), 7369. https://doi.org/10.1038/s41467-024-51620-2
  11. Mezgec, K., Snoj, J., Ulčakar, L., Ljubetič, A., Tušek Žnidarič, M., Škarabot, M., & Jerala, R. (2024). Coupling of Spectrin Repeat Modules for the Assembly of Nanorods and Presentation of Protein Domains. ACS Nano. https://doi.org/10.1021/acsnano.4c07701
  12. Jazbec, V., Varda, N., Šprager, E., Meško, M., Vidmar, S., Romih, R., Podobnik, M., Kežar, A., Jerala, R., & Benčina, M. (2024). Protein Gas Vesicles of Bacillus megaterium as Enhancers of Ultrasound-Induced Transcriptional Regulation. ACS Nano, 18(26), 16692–16700. https://doi.org/10.1021/acsnano.4c01498
  13. Esih, H., Mezgec, K., Billmeier, M., Malenšek, Š., Benčina, M., Grilc, B., Vidmar, S., Gašperlin, M., Bele, M., Zidarn, M., Zupanc, T. L., Morgan, T., Jordan, I., Sandig, V., Schrödel, S., Thirion, C., Protzer, U., Wagner, R., Lainšček, D., & Jerala, R. (2024). Mucoadhesive film for oral delivery of vaccines for protection of the respiratory tract. Journal of Controlled Release, 371, 179–192. https://doi.org/10.1016/j.jconrel.2024.05.041
  14. Plaper, T., Rihtar, E., Železnik Ramuta, T., Forstnerič, V., Jazbec, V., Ivanovski, F., Benčina, M., & Jerala, R. (2024). The art of designed coiled-coils for the regulation of mammalian cells. Cell Chemical Biology. https://doi.org/10.1016/j.chembiol.2024.06.001
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