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
Behrens, G. ; Edelmann, S.L. ; Raj, T. ; Kronbeck, N. ; Monecke, T. ; Davydova, E.-O. ; Wong, E.H. ; Kifinger, L. ; Giesert, F. ; Kirmaier, M.E. ; Hohn, C. ; de Jonge, L.S. ; Pisfil, M.G. ; Fu, M. ; Theurich, S. ; Feske, S. ; Kawakami, N. ; Wurst, W. ; Niessing, D. ; Heissmeyer, V.
Disrupting Roquin-1 interaction with Regnase-1 induces autoimmunity and enhances antitumor responses.O'Neill, T.J. ; Seeholzer, T. ; Gewies, A. ; Gehring, T. ; Giesert, F. ; Hamp, I. ; Grass, C. ; Schmidt, H. ; Kriegsmann, K. ; Tofaute, M.J. ; Demski, K. ; Poth, T. ; Rosenbaum, M. ; Schnalzger, T. ; Ruland, J. ; Göttlicher, M. ; Kriegsmann, M. ; Naumann, R. ; Heissmeyer, V. ; Wurst, W. ; Krappmann, D.
TRAF6 prevents fatal inflammation by homeostatic suppression of MALT1 protease.Truong, D.J.J. ; Phlairaharn, T. ; Eßwein, B. ; Gruber, C. ; Tümen, D. ; Baligács, E. ; Armbrust, N. ; Vaccaro, F.L. ; Lederer, E.-M. ; Beck, E.M. ; Geilenkeuser, J. ; Göppert, S. ; Krumwiede, L. ; Grätz, C. ; Raffl, G. ; Schwarz, D. ; Zirngibl, M. ; Živanić, M. ; Beyer, M. ; Körner, J.D. ; Santl, T. ; Evsyukov, V. ; Strauß, T. ; Schwarz, S.C. ; Höglinger, G.U. ; Heutink, P. ; Doll, S. ; Conrad, M. ; Giesert, F. ; Wurst, W. ; Westmeyer, G.G.
Non-invasive and high-throughput interrogation of exon-specific isoform expression.Eberherr, A.C. ; Maaske, A. ; Wolf, C. ; Giesert, F. ; Berutti, R. ; Rusha, E. ; Pertek, A. ; Kastlmeier, M.T. ; Voss, C. ; Plummer, M. ; Sayed, A. ; Graf, E. ; Effner, R. ; Volz, T. ; Drukker, M. ; Strom, T.M. ; Meitinger, T. ; Stöger, T. ; Buyx, A.M. ; Hagl, B. ; Renner, E.D.
Rescue of STAT3 function in hyper-IgE syndrome using adenine base editing.Frey, L. ; Ziętara, N. ; Lyszkiewicz, M. ; Marquardt, B. ; Mizoguchi, Y. ; Linder, M.I. ; Liu, Y. ; Giesert, F. ; Wurst, W. ; Dahlhoff, M. ; Schneider, M. ; Wolf, E. ; Somech, R. ; Klein, C.
Mammalian VPS45 orchestrates trafficking through the endosomal system.Swan, A.L. ; Schütt, C. ; Rozman, J. ; Del Mar Muñiz Moreno, M. ; Brandmaier, S. ; Simon, M. ; Leuchtenberger, S. ; Griffiths, M. ; Brommage, R. ; Keskivali-Bond, P. ; Grallert, H. ; Werner, T. ; Teperino, R. ; Becker, L. ; Miller, G. ; Moshiri, A. ; Seavitt, J.R. ; Cissell, D.D. ; Meehan, T.F. ; Acar, E.F. ; Lelliott, C.J. ; Flenniken, A.M. ; Champy, M.F. ; Sorg, T. ; Ayadi, A. ; Braun, R.E. ; Cater, H. ; Dickinson, M.E. ; Flicek, P. ; Gallegos, J. ; Ghirardello, E.J. ; Heaney, J.D. ; Jacquot, S. ; Lally, C. ; Logan, J.G. ; Teboul, L. ; Mason, J. ; Spielmann, N. ; McKerlie, C. ; Murray, S.A. ; Nutter, L.M.J. ; Odfalk, K.F. ; Parkinson, H. ; Prochazka, J. ; Reynolds, C.L. ; Selloum, M. ; Spoutil, F. ; Svenson, K.L. ; Vales, T.S. ; Wells, S.E. ; White, J.K. ; Sedlacek, R. ; Wurst, W. ; Lloyd, K.K.C. ; Croucher, P.I. ; Fuchs, H. ; Williams, G.R. ; Bassett, D. ; Gailus-Durner, V. ; Herault, Y. ; Mallon, A.M. ; Brown, S.D.M. ; Mayer-Kuckuk, P. ; Hrabě de Angelis, M. ; IMPC Consortium (Aguilar-Pimentel, J.A. ; Amarie, O.V. ; Bürger, A. ; Calzada-Wack, J. ; Cho, Y.-L. ; Giesert, F. ; Garrett, L. ; Graw, J. ; Hörlein, A. ; Hölter, S.M. ; Klein-Rodewald, T. ; Kühn, R. ; Lengger, C. ; Marschall, S. ; Rathkolb, B. ; Sanz-Moreno, A. ; Seisenberger, C. ; Steinkamp, R. ; Stoeger, C. ; Treise, I. ; Zimprich, A.) ; Beckers, J.
Mouse mutant phenotyping at scale reveals novel genes controlling bone mineral density.Russo, G.L. ; Sonsalla, G. ; Natarajan, P. ; Breunig, C. ; Bulli, G. ; Merl-Pham, J. ; Schmitt, S. ; Giehrl-Schwab, J. ; Giesert, F. ; Jastroch, M. ; Zischka, H. ; Wurst, W. ; Stricker, S.H. ; Hauck, S.M. ; Masserdotti, G. ; Götz, M.
CRISPR-mediated induction of neuron-enriched mitochondrial proteins boosts direct glia-to-neuron conversion.