2024
Effective genome editing with an enhanced ISDra2 TnpB system and deep learning-predicted ωRNAs
K F Marquart, N Mathis*, A Mollaysa*, S Müller, L Kissling, T Rothgangl, L Schmidheini, P I Kulcsár, A Allam, M M Kaufmann, M Matsushita, T Haenggi, T Cathomen, M Kopf, M Krauthammer, G Schwank
Nature Methods, 2024
*These authors contributed equally
Machine learning prediction of prime editing efficiency across diverse chromatin contexts
N Mathis, A Allam, A Tálas, LKissling, E Benvenuto, L Schmidheini, R Schep, T Damodharan, Z Balázs, S Janjuha, E I Ioannidi, D Böck, B van Steensel, M Krauthammer & G Schwank
Nature Biotechnology, 2024
Base editing of Ptbp1 in neurons alleviates symptoms in a mouse model for Parkinson’s disease
D Böck, M Wilhelm, J Mumenthaler, D F Carpanese, S d’Aquin, A Cremonesi, A Rassi, J Häberle, T Patriarchi, G Schwank
eLife preprint, 2024
Enhancing prime editor activity by directed protein evolution in yeast
Y Weber, D Böck, A Ivașcu, N Mathis, T Rothgangl, E I Ioannidi, A C Blaudt, L Tidecks, M Vadovics, H Muramatsu, A Reichmuth, K F Marquart, L Kissling, N Pardi, M Jinek, G Schwank
Nature Communications, 2024
In vivo base editing of a pathogenic Eif2b5 variant improves vanishing white matter phenotypes in mice
D Böck*, I M Revers*, A S J Bomhof, A E J Hillen, C Boeijink, L Kissling, S Egli, M A Moreno-Mateos, M S van der Knaap, N P van Til, G Schwank
Molecular Therapy, 2024
*These authors contributed equally
Treatment of a genetic liver disease in mice through transient prime editor expression
T Rothgangl, E I Ioannidi, Y Weber, A Tálas, D Böck, M Matsushita, E A Villiger, L Schmidheini, J Moon, P J C Lin, S H Y Fan, K F Marquart, C Schwerdel, N Rimann, E Faccin, L Villiger, H Muramatsu, M Vadovics, A Cremonesi, B Thöny, M Kopf, J Häberle, N Pardi, Y K Tam, G Schwank
BioRxiv, 2024
Targeting the IDH1R132H mutation in gliomas by CRISPR/Cas precision base editing
Remi Weber, Flavio Vasella, Artsiom Klimko, Manuela Silginer, Martine Lamfers, Marian Christoph Neidert, Luca Regli, Gerald Schwank, Michael Weller
Neuro-Oncology Advances, 2024
Targeted knock-in of NCF1 cDNA into the NCF2 locus leads to myeloid phenotypic correction of p47phox-deficient chronic granulomatous disease
K M Siow, M Güngör, D Wrona, F Raimondi, O Pastukhov, P Tsapogas, T Menzi, M Schmitz, P I Kulcsár, G Schwank, A Schulz, M Jinek, Ute Modlich, U Siler, J Reichenbach
Molecular Therapy Nucleic Acids, 2024
Therapeutic liver cell transplantation to treat murine PKU
Melanie Willimann, Hiu Man Grisch-Chan, Nicole Rimann, Tanja Rothgangl, Martina Hruzova, Gerald Schwank, Beat Thöny
Journal of Inherited Metabolic Disease, 2024
2023
Mutant SF3B1 promotes malignancy in PDAC
P Simmler, E I Ioannidi, T Mengis, K F Marquart, S Asawa, K Lehmann, A Khales, T Thomas, C Schwerdel, N Aceto, G Rätsch, M Stoffel, G Schwank
Elife, 2023
Predicting prime editing efficiency across diverse edit types and chromatin contexts with machine learning
N Mathis, A Allam, A Tálas, E Benvenuto, R Schep, T Damodharan, Z Balázs, S Janjuha, L Schmidheini, D Böck, B van Steensel, M Krauthammer, G Schwank
BioRxiv, 2023
Continuous directed evolution of a compact CjCas9 variant with broad PAM compatibility
L Schmidheini, N Mathis, K F Marquart, T Rothgangl, L Kissling, D Böck, C Chanez, J P Wang, M Jinek, G Schwank
Nature Chemical Biology, 2023
Prime editing of the beta-1 adrenoceptor in the brain reprograms mouse behavior
D Boeck*, L Tidecks*, M Wilhelm*, Y Weber, E Ioannidi, J Mumenthaler, T Rothgangl,
L Schmidheini, S Janjuha, T Patriarchi, G Schwank
BioRxiv, 2023
*These authors contributed equally
Safe delivery of AAV vectors to the liver of small weaned pigs by ultrasound guided percutaneous transhepatic portal vein injection
T Rothgangl*, M Hruzova*, R Gnannt, N Aeberhard, L Kissling, H M Grisch-Chan, S Klassa, N Rimann, K F Marquart, E Ioannidi, A Wolf, C Kupatt, X Sidler, J Häberle, G Schwank, B Thöny
BioRxiv, 2023
*These authors contributed equally
Predicting prime editing efficiency and product purity by deep learning
N Mathis*, A Allam*, L Kissling, K F Marquart, L Schmidheini, C Solari, Z Balázs, M Krauthammer, G Schwank
Nature Biotechnology, 2023
*These authors contributed equally
State-of-the-art 2023 on gene therapy for phenylketonuria
M Martinez, C O Harding, G Schwank, B Thöny
Journal of Inherited Metabolic Disease, 2023
2022
SF3B1 promotes tumor malignancy through splicing-independent co-activation of HIF1α
P T Simmler*, C Cortijo*, L M Koch, P Galliker, S Angori, H A Bolck, C Mueller, A Vukolic, P Mirtschink, Y Christinat, N R Davidson, K Lehmann, G Pellegrini, C Pauli, D Lenggenhager, I Guccini, T Ringel, C Hirt, K F Marquart, M Schaefer, G Rätsch, M Peter, H Moch, M Stoffel, G Schwank
Cell Reports, 2022
*These authors contributed equally
Mutant SF3B1 promotes PDAC malignancy through TGF-β resistance
P T Simmler, T Mengis, K Lehmann, A Kahles, T Thomas, G Rätsch, M Stoffel, G Schwank
BioRxiv, 2022
Loss of Rnf31 and Vps4b sensitizes pancreatic cancer to T-cell-mediated killing
N Frey, L Tortola, D Egli, S Janjuha, T Rothgangl, K F Marquart, F Ampenberger, M Kopf, G Schwank
Nature Communications, 2022
CRISPR-Based Screening in Three-Dimensional Organoid Cultures to Identify TGF-β Pathway Regulators
N Frey, G Schwank
TGF-Beta Signaling – Methods and Protocols, 2022
In vivo prime editing of a metabolic liver disease in mice
D Boeck*, T Rothgangl*, L Villiger*, L Schmidheini, M Matsushita, N Mathis, E Ioannidi, N Rimann, H M Grisch-Chan, S Kreutzer, Z Kontarakis, M Kopf, B Thöny, G Schwank
Science Translational Medicine, 2022
*These authors contributed equally
Drug screening and genome editing in human pancreatic cancer organoids identifies drug-gene interactions and candidates for off-label therapy
C K Hirt, T H Booji, L Grob, P Simmler, N C Toussaint, D Keller, D Taube, V Ludwig, A Goryachkin, C Pauli, D Lenggenhager, D J Stekhoven, C U Stirnimann, K Endhardt, F Ringnalda, L Villiger, A Siebenhüner, S Karkampouna, M De Menna, J Beshay, H Klett, M Kruithof-de Julio, J Schüler, G Schwank
Cell Genomics, 2022
In vivo targeting of a variant causing vanishing white matter using CRISPR/Cas9
A E J Hillen, M Hruzova, T Rothgangl, M Breur, M Bugiani, M S van der Knaap, G Schwank, V M Heine
Molecular Therapy – Methods & Clinical Development, 2022
2021
Replacing the SpCas9 HNH domain by deaminases generates compact base editors with an alternative targeting scope
L Villiger*, L Schmidheini*, N Mathis, T Rothgangl, K F Marquart, G Schwank
Molecular Therapy – Nucleic Acids, 2021
*These authors contributed equally
Predicting base editing outcomes with an attention-based deep learning algorithm trained on high-throughput target library screens
K F Marquart*, A Allam*, S Janjuha*, A Sintsova, L Villiger, N Frey, M Krauthammer, G Schwank
Nature Communications, 2021
*These authors contributed equally
In vivo adenine base editing of PCSK9 in macaques reduces LDL cholesterol levels
T Rothgangl, M K Dennis, P J C Lin, R Oka, D Witzigmann, L Villiger, W Qi, M Hruzova, L Kissling, D Lenggenhager, C Borrelli, S Egli, N Frey, N Bakker, J A Walker II, A P Kadina, D V Victorov, M Pacesa, S Kreutzer, Z Kontarakis, A Moor, M Jinek, D Weissman, M Stoffel, R v Boxtel, K Holden, N Pardi, B Thöny, J Häberle, Y K Tam, S C Semple & G Schwank
Nature Biotechnology, 2021
In vivo cytidine base editing of hepatocytes without detectable off-target mutations in RNA and DNA
L Villiger*, T Rothgangl*, D Witzigmann, R Oka, P J C Lin, W Qi, S Janjuha, C Berk, F Ringnalda, M B Beattie, M Stoffel, B Thöny, J Hall, H Rehrauer, R van Boxtel, Y K Tam and G Schwank
Nature Biomedical Engineering, 2021
*These authors contributed equally
miR-802 regulates Paneth cell function and enterocyte differentiation in the mouse small intestine
A Goga* , B Yagabasan*, K Herrmanns*, S Godbersen, P N Silva, R Denzler, M Zünd, M Furter, G Schwank, S Sunagawa, W-D Hardt, M Stoffel
Nature Communication, 2021
*These authors contributed equally
Identification of HIF-dependent alternative splicing in gastrointestinal cancers and characterization of a long, coding isoform of SLC35A3
P Markolin, N Davidson, C Hirt, C D Chabbert, N Zamboni, G Schwank, W Krek, G Rätsch
Genomics, 2021
2020
Genome-Scale CRISPR Screening in Human Intestinal Organoids Identifies Drivers of TGF-β Resistance
T Ringel, N Frey, F Ringnalda, S Janjuha, S Cherkaoui, S Butz, S Srivatsa, M Pirkl, G Russo, L Villiger, G Rogler, H Clevers, N Beerenwinkel, N Zamboni, T Baubec, G Schwank
Cell Stem Cell 26 (3), 431-440. e8, 2020
High-throughput automated organoid culture via stem-cell aggregation in microcavity arrays
N Brandenberg, S Hoehnel, F Kuttler, K Homicsko, C Ceroni, T Ringel, N Gjorevski, G Schwank, G Coukos, G Turcatti, M P Lutolf
Nature Biomedical Engineering, 2020
Germ‐Free and Microbiota‐Associated Mice yield Small Intestinal Epithelial Organoids with Equivalent and Robust Transcriptome/Proteome Expression Phenotypes
A Hausmann, G Russo, J Grossmann, M Zünd, G Schwank, R Aebersold, Y Liu, ME Sellin, W Hardt
Cellular Microbiology, e13191, 2020
2019
In vitro generation of CRISPR-Cas9 complexes with covalently bound repair templates for genome editing in mammalian cells
N Savić, F Ringnalda, C Berk, K Bargsten, J Hall, M Jinek, G Schwank
Bio-protocol, 9 (1), 2019
State-of-the-art 2019 on gene therapy for phenylketonuria
HM Grisch-Chan, G Schwank, CO Harding, B Thöny
Human Gene Therapy, 30 (10), 1274-1283, 2019
Characterization of HIF-dependent alternative isoforms in pancreatic cancer
P Markolin, N Davidson, C Hirt, C Chabbert, N Zamboni, G Schwank, W Krek, G Raetsch
bioRxiv, 826156, 2019
2018
Treatment of a metabolic liver disease by in vivo genome base editing in adult mice
L Villiger, HM Grisch-Chan, H Lindsay, F Ringnalda, CB Pogliano, G Allegri, R Fingerhut, J Häberle, J Matos, MD Robinson, B Thöny, G Schwank
Nature Medicine, 24 (10), 1519-1525, 2018
Covalent linkage of the DNA repair template to the CRISPR-Cas9 nuclease enhances homology-directed repair
N Savic, FCAS Ringnalda, H Lindsay, C Berk, K Bargsten, Y Li, D Neri, MD Robinson, C Ciaudo, J Hall, M Jinek, G Schwank
Elife, 7, e33761, 2018
Growth of epithelial organoids in a defined hydrogel
N Broguiere, L Isenmann, C Hirt, T Ringel, S Placzek, E Cavalli, F Ringnalda, L Villiger, R Züllig, R Lehmann, G Rogler, MH Heim, J Schüler, M Zenobi-Wong, G Schwank
Advanced Materials, 30 (43), 1801621, 2018
Organoid models of human liver cancers derived from tumor needle biopsies
S Nuciforo, I Fofana, MS Matter, T Blumer, D Calabrese, T Boldanova, S Piscuoglio, S Wieland, F Ringnalda, G Schwank, LM Terracciano, CKY Ng, MH Heim
Cell Reports, 24 (5), 1363-1376, 2018
2016
Advances in therapeutic CRISPR/Cas9 genome editing
N Savić, G Schwank
Translational Research, 168, 15-21, 2016
CRISPR/Cas9-mediated genome editing of mouse small intestinal organoids
G Schwank, H Clevers
Gastrointestinal Physiology and Diseases, 3-11, 2016
Tissue-specific mutation accumulation in human adult stem cells during life
F Blokzijl, J De Ligt, M Jager, V Sasselli, S Roerink, N Sasaki, M Huch, S Boymans, E Kuijk, P Prins, I Nijman, I Martincorena, M Mokry, C Wiegerinck, S Middendorp, T Sato, G Schwank, E Nieuwenhuis, M Verstegen, L Van Der Laan, J De Jonge, J IJzermans, R Vries, M Van De Wetering, M Stratton, H Clevers, E Cuppen, R Van Boxtel
Nature, 538 (7624), 260-264, 2016
Until 2015
Sequential cancer mutations in cultured human intestinal stem cells
J Drost, RH Van Jaarsveld, B Ponsioen, C Zimberlin, R Van Boxtel, A Buijs, N Sachs, R Overmeer, G Offerhaus, H Begthel, J Korving, M Van De Wetering, G Schwank, M Logtenberg, E Cuppen, H Snippert, J Medema, G Kops, H Clevers
Nature, 521 (7550), 43-47, 2015
Paneth cell extrusion and release of antimicrobial products is directly controlled by immune cell–derived IFN-γ
HF Farin, WR Karthaus, P Kujala, M Rakhshandehroo, G Schwank, R Vries, E Kalkhoven, E Nieuwenhuis, H Clevers
Journal of Experimental Medicine, 211 (7), 1393-1405, 2014
Functional repair of CFTR by CRISPR/Cas9 in intestinal stem cell organoids of cystic fibrosis patients
G Schwank, BK Koo, V Sasselli, JF Dekkers, I Heo, T Demircan, N Sasaki, S Boymans, E Cuppen, C van der Ent, E Nieuwenhuis, J Beekman, H Clevers
Cell Stem Cell, 13 (6), 653-658, 2013
Generation of BAC transgenic epithelial organoids
G Schwank, A Andersson-Rolf, BK Koo, N Sasaki, H Clevers
PLOS ONE, 8 (10), 2013
Comment on “Dynamics of dpp signaling and proliferation control”
G Schwank, SF Yang, S Restrepo, K Basler
Science, 335 (6067), 401, 2012
Formation of the long range Dpp morphogen gradient
G Schwank, S Dalessi, SF Yang, R Yagi, AM de Lachapelle, M Affolter, S Bergmann, K Basler
PLOS BIOLOGY, 9 (7), 2011
Antagonistic growth regulation by Dpp and Fat drives uniform cell proliferation
G Schwank, G Tauriello, R Yagi, E Kranz, P Koumoutsakos, K Basler
Developmental Cell, 20 (1), 123-130, 2011
Regulation of organ growth by morphogen gradients
G Schwank, K Basler
Cold Spring Harbor Perspectives in Biology, 2 (1), 2010
Growth regulation by Dpp: an essential role for Brinker and a non-essential role for graded signaling levels
G Schwank, S Restrepo, K Basler
Development, 135 (24), 4003-4013, 2008