Gene Editing
The Gene Editing Group is dedicated to discovering, developing, and applying innovative genetic tools that drive translational advances in cell and gene therapies. We work with promising systems such as CRISPR/Cas9, CRISPRi, and Cas13d, which require specific adaptations for each unique application. Our research addresses critical challenges in areas like gene therapy (Moretti et al., 2020), cell replacement therapies (Giehrl-Schwab et al., 2022), and novel RNA modulation and imaging systems (Gruber et al., 2024). For therapies that target specific cell types such as stem cells, or cells in challenging environments like the CNS, precise, targeted delivery is essential. To meet this need, we are developing a novel delivery platform inspired by nature, enhanced by artificial intelligence, and built with synthetic proteins (Schuhmacher et al., submitted). In addition, our transgenic unit supports both in-house scientists and external collaborators by creating customized model systems, including iPSCs and advanced mouse models (Giusti et al., 2024; Behrens et al., 2021). Overall, our vision is to empower the next generation of scientific and translational breakthroughs by providing and applying tailored genetic tools.
Team Members
Publications
Vetrivel, S. ; Graw, J. ; Giesert, F.
Identification of ocular regulatory functions of core histone variant H3.2 in mouse.Vetrivel, S. ; Tiso, N. ; Kügler, A. ; Irmler, M. ; Horsch, M. ; Beckers, J. ; Hladik, D. ; Giesert, F. ; Gailus-Durner, V. ; Fuchs, H. ; Sabrautzki, S. ; Adler, T. ; Treise, I. ; Busch, D.H. ; Aguilar-Pimentel, J.A. ; Ollert, M. ; Götz, A. ; Amarie, O.V. ; Stöger, T. ; Schulz, H. ; Becker, L. ; Klopstock, T. ; Schrewe, A. ; Spielmann, N. ; Bekeredjian, R. ; Garrett, L. ; Hölter, S.M. ; Zimprich, A. ; Wurst, W. ; Mayer-Kuckuk, P. ; Hans, W. ; Rozman, J. ; Klingenspor, M. ; Neff, F. ; da Silva Buttkus, P. ; Calzada-Wack, J. ; Rácz, I. ; Zimmer, A. ; Rathkolb, B. ; Wolf, E. ; Prehn, C. ; Adamski, J. ; Östereicher, M.A. ; Miller, G. ; Steinkamp, R. ; Lengger, C. ; Maier, H. ; Stoeger, C. ; Leuchtenberger, S. ; Hrabě de Angelis, M. ; Graw, J.
Mutation in the mouse histone gene Hist2h3c1 leads to degeneration of the lens vesicle and severe microphthalmia.Funk, N. ; Munz, M. ; Ott, T. ; Brockmann, K. ; Wenninger-Weinzierl, A. ; Kühn, R. ; Vogt Weisenhorn, D.M. ; Giesert, F. ; Wurst, W. ; Gasser, T. ; Biskup, S.
The Parkinson's disease-linked Leucine-rich repeat kinase 2 (LRRK2) is required for insulin-stimulated translocation of GLUT4.