2025
Gene therapy of rare diseases as a milestone in medicine - overview of the field and report on initial experiences in Slovenia.
Grošelj, U., Kavčič, M., Drole Torkar, A., Kafol, J., Lainšček, D., Jerala, R., Sever, M., Zver, S., Serša, G., Čemažar, M., Strojan, P., Grošelj, A., Žerjav Tanšek, M., Miroševič, Š., Ivančan, S., Prelog, T., Gosar, D., Oražem Mrak, J., Mlinarič, M., … Osredkar, D. (2025). In
Orphanet Journal of Rare Diseases (Vol. 20, Issue 1). BioMed Central Ltd.
https://doi.org/10.1186/s13023-025-03828-8
RNA trans-splicing to rescue β-catenin: A novel approach for treating CTNNB1-Haploinsufficiency disorder.
Maruna, M., Sušjan-Leite, P., Meško, M., Miroševič, Š., & Jerala, R. (2025).
Molecular Therapy Nucleic Acids,
36(3).
https://doi.org/10.1016/j.omtn.2025.102680
Check the blogpost
Genotypic, functional, and phenotypic characterization in CTNNB1 neurodevelopmental syndrome.
Žakelj, N., Gosar, D., Miroševič, Š., Sanders, S. J., Ljungdahl, A., Kohani, S., Huang, S., Leong, L. I., An, Y., Teo, M. J., Moultrie, F., Jerala, R., Lainšček, D., Forstnerič, V., Sušjan, P., Lisowski, L., Perez-Iturralde, A., Mrak, J. O., Chan, H. Y. E., & Osredkar, D. (2025).
Human Genetics and Genomics Advances,
6(4).
https://doi.org/10.1016/j.xhgg.2025.100483
Clustering of NLRP3 induced by membrane or protein scaffolds promotes inflammasome assembly.
Boršić, E., Ramuta, T. Ž., Orehek, S., Kreft, M. E., Geyer, M., Jerala, R., & Hafner-Bratkovič, I. (2025).
Nature Communications ,
16(1).
https://doi.org/10.1038/s41467-025-60277-4
Check the blogpost
Synthetic Biology for Designing Allostery and its Potential Biomedical Applications.
Ultrasound-responsive mammalian cell synthetic biology.
Ivanovski, F., Jazbec, V., Varda, N., Jerala, R., & Benčina, M. (2025).
IEEE Transactions on Ultrasonics, Ferroelectrics, and Frequency Control, 1–1.
https://doi.org/10.1109/TUFFC.2025.3570813
Check the blogpost
Paving the way toward treatment solutions for CTNNB1 syndrome: a patient organization perspective
Miroševič, Š., Khandelwal, S., Amerson, E., Parks, E., Parks, M., Cochran, L., González Hernández, A., Ferraro, M., Lisowski, L., Perez-Iturralde, A., Chung, W., Jacob, M. H., Žakelj, N., Lainšček, D., Forstnerič, V., Sušjan, P., Maruna, M., Jerala, R., & Osredkar, D. (2025).
Therapeutic Advances in Rare Disease,
6.
doi.org/10.1177/26330040251318355
Check the blogpost
Ligand-induced assembly of antibody variable fragments for the chemical regulation of biological processes
Rihtar, E., Fink, T., Lebar, T., Lainšček, D., Kolenc, Ž., Polajnar, L. K., & Jerala, R. (2025).
Cell Chemical Biology.
doi.org/10.1016/j.chembiol.2025.01.007
Check the blogpost
2024
Cytokine-armed pyroptosis induces antitumor immunity against diverse types of tumors
Orehek, S., Ramuta, T. Ž., Lainšček, D., Malenšek, Š., Šala, M., Benčina, M., Jerala, R., & Hafner-Bratkovič, I. (2024).
doi.org/10.1038/s41467-024-55083-3
Check the blogpost
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
Presen, D. M., Lainšček, D., Kinghorn, J., Sebestyen, Z., Kuball, J., Amini, L., Reinke, P., Fuchs, A., Jerala, R., & Benčina, M. (2024).
Computational and Structural Biotechnology Journal.
doi.org/10.1016/j.csbj.2024.11.051
Check the blogpost
MyD88 protein destabilization mitigates NF-κB-dependent protection against macrophage apoptosis
Lainšček, D., Horvat, S., Dolinar, K., Ivanovski, F., Romih, R., Pirkmajer, S., Jerala, R., & Manček-Keber, M. (2024). C
ell Communication and Signaling,
22(1), 549.
doi.org/10.1186/s12964-024-01930-1
Check the blogpost
Ultrasound-mediated spatial and temporal control of engineered cells in vivo
Ivanovski, F., Meško, M., Lebar, T., Rupnik, M., Lainšček, D., Gradišek, M., Jerala, R., & Benčina, M. (2024).
doi.org/10.1038/s41467-024-51620-2
Check the blogpost
Coupling of Spectrin Repeat Modules for the Assembly of Nanorods and Presentation of Protein Domains
Mezgec, K., Snoj, J., Ulčakar, L., Ljubetič, A., Tušek Žnidarič, M., Škarabot, M., & Jerala, R. (2024).
ACS Nano.
doi.org/10.1021/acsnano.4c07701
The art of designed coiled-coils for the regulation of mammalian cells
Plaper, T., Rihtar, E., Železnik Ramuta, T., Forstnerič, V., Jazbec, V., Ivanovski, F., Benčina, M., & Jerala, R. (2024). Elsevier Ltd.
doi.org/10.1016/j.chembiol.2024.06.001
Protein Gas Vesicles of Bacillus megaterium as Enhancers of Ultrasound-Induced Transcriptional Regulation
Jazbec, V., Varda, N., Šprager, E., Meško, M., Vidmar, S., Romih, R., Podobnik, M., Kežar, A., Jerala, R., & Benčina, M. (2024).
ACS Nano,
18(26), 16692–16700.
doi.org/10.1021/acsnano.4c01498
Check the blogpost
Mucoadhesive film for oral delivery of vaccines for protection of the respiratory tract
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).
Journal of Controlled Release,
371, 179–192.
doi.org/10.1016/j.jconrel.2024.05.041
Designed allosteric protein logic
Plaper, T., Merljak, E., Fink, T., Satler, T., Ljubetič, A., Lainšček, D., Jazbec, V., Benčina, M., Stevanoska, S., Džeroski, S., & Jerala, R. (2024).
Cell Discovery,
10(1).
doi.org/10.1038/s41421-023-00635-y
