Discovery of Risdiplam, a Selective Survival of Motor Neuron‑2 (SMN2) Gene Splicing Modifier for the Treatment of Spinal Muscular Atrophy (SMA)
Ratni, Hasane, Ebeling, Martin, Baird, John, Bendels, Stefanie, Bylund, Johan, Chen, Karen S, Denk, Nora, Feng, Zhihua, Green, Luke, Guerard, Melanie, Jablonski, Philippe, Jacobsen, Bjoern, Khwaja, Omar, Kletzl, Heidemarie, Ko, Chien-Ping, Kustermann, Stefan, Marquet, Anne, Metzger, Friedrich, Mueller, Barbara, Naryshkin, Nikolai A, Paushkin, Sergey V, Pinard, Emmanuel, Poirier, Agnès, Reutlinger, Michael, Weetall, Marla, Zeller, Andreas, Zhao, Xin, Mueller, Lutz
Published in Journal of medicinal chemistry (09.08.2018)
Published in Journal of medicinal chemistry (09.08.2018)
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Journal Article
Binding to SMN2 pre-mRNA-protein complex elicits specificity for small molecule splicing modifiers
Sivaramakrishnan, Manaswini, McCarthy, Kathleen D., Campagne, Sébastien, Huber, Sylwia, Meier, Sonja, Augustin, Angélique, Heckel, Tobias, Meistermann, Hélène, Hug, Melanie N., Birrer, Pascale, Moursy, Ahmed, Khawaja, Sarah, Schmucki, Roland, Berntenis, Nikos, Giroud, Nicolas, Golling, Sabrina, Tzouros, Manuel, Banfai, Balazs, Duran-Pacheco, Gonzalo, Lamerz, Jens, Hsiu Liu, Ying, Luebbers, Thomas, Ratni, Hasane, Ebeling, Martin, Cléry, Antoine, Paushkin, Sergey, Krainer, Adrian R., Allain, Frédéric H.-T., Metzger, Friedrich
Published in Nature communications (14.11.2017)
Published in Nature communications (14.11.2017)
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Journal Article
The small interferon-induced transmembrane genes and proteins
Siegrist, Fredy, Ebeling, Martin, Certa, Ulrich
Published in Journal of interferon & cytokine research (01.01.2011)
Published in Journal of interferon & cytokine research (01.01.2011)
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Journal Article
The Immunogenicity of Antibody Aggregates in a Novel Transgenic Mouse Model
Bessa, Juliana, Boeckle, Sabine, Beck, Hermann, Buckel, Thomas, Schlicht, Sonja, Ebeling, Martin, Kiialainen, Anna, Koulov, Atanas, Boll, Björn, Weiser, Thomas, Singer, Thomas, Rolink, Antonius G., Iglesias, Antonio
Published in Pharmaceutical research (01.07.2015)
Published in Pharmaceutical research (01.07.2015)
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Journal Article
White-to-brown metabolic conversion of human adipocytes by JAK inhibition
Moisan, Annie, Lee, Youn-Kyoung, Zhang, Jitao David, Hudak, Carolyn S., Meyer, Claas A., Prummer, Michael, Zoffmann, Sannah, Truong, Hoa Hue, Ebeling, Martin, Kiialainen, Anna, Gérard, Régine, Xia, Fang, Schinzel, Robert T., Amrein, Kurt E., Cowan, Chad A.
Published in Nature cell biology (01.01.2015)
Published in Nature cell biology (01.01.2015)
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Journal Article
CD22 Blockage Restores Age-Related Impairments of Microglia Surveillance Capacity
Aires, Vanessa, Coulon-Bainier, Claire, Pavlovic, Anto, Ebeling, Martin, Schmucki, Roland, Schweitzer, Christophe, Kueng, Erich, Gutbier, Simon, Harde, Eva
Published in Frontiers in immunology (01.06.2021)
Published in Frontiers in immunology (01.06.2021)
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Journal Article
Brain Shuttle Antibody for Alzheimer’s Disease with Attenuated Peripheral Effector Function due to an Inverted Binding Mode
Weber, Felix, Bohrmann, Bernd, Niewoehner, Jens, Fischer, Jens A.A., Rueger, Petra, Tiefenthaler, Georg, Moelleken, Joerg, Bujotzek, Alexander, Brady, Kevin, Singer, Thomas, Ebeling, Martin, Iglesias, Antonio, Freskgård, Per-Ola
Published in Cell reports (Cambridge) (02.01.2018)
Published in Cell reports (Cambridge) (02.01.2018)
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Journal Article
Expression profiling of human immune cell subsets identifies miRNA-mRNA regulatory relationships correlated with cell type specific expression
Allantaz, Florence, Cheng, Donavan T, Bergauer, Tobias, Ravindran, Palanikumar, Rossier, Michel F, Ebeling, Martin, Badi, Laura, Reis, Bernhard, Bitter, Hans, D'Asaro, Matilde, Chiappe, Alberto, Sridhar, Sriram, Pacheco, Gonzalo Duran, Burczynski, Michael E, Hochstrasser, Denis, Vonderscher, Jacky, Matthes, Thomas
Published in PloS one (20.01.2012)
Published in PloS one (20.01.2012)
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Journal Article
Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes
Garcia-Lopez, Amparo, Tessaro, Francesca, Jonker, Hendrik R. A., Wacker, Anna, Richter, Christian, Comte, Arnaud, Berntenis, Nikolaos, Schmucki, Roland, Hatje, Klas, Petermann, Olivier, Chiriano, Gianpaolo, Perozzo, Remo, Sciarra, Daniel, Konieczny, Piotr, Faustino, Ignacio, Fournet, Guy, Orozco, Modesto, Artero, Ruben, Metzger, Friedrich, Ebeling, Martin, Goekjian, Peter, Joseph, Benoît, Schwalbe, Harald, Scapozza, Leonardo
Published in Nature communications (23.05.2018)
Published in Nature communications (23.05.2018)
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Journal Article
Genome-based analysis of the nonhuman primate Macaca fascicularis as a model for drug safety assessment
Ebeling, Martin, Küng, Erich, See, Angela, Broger, Clemens, Steiner, Guido, Berrera, Marco, Heckel, Tobias, Iniguez, Leonardo, Albert, Thomas, Schmucki, Roland, Biller, Hermann, Singer, Thomas, Certa, Ulrich
Published in Genome research (01.10.2011)
Published in Genome research (01.10.2011)
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Journal Article
Targeting neuronal lysosomal dysfunction caused by β-glucocerebrosidase deficiency with an enzyme-based brain shuttle construct
Gehrlein, Alexandra, Udayar, Vinod, Anastasi, Nadia, Morella, Martino L., Ruf, Iris, Brugger, Doris, von der Mark, Sophia, Thoma, Ralf, Rufer, Arne, Heer, Dominik, Pfahler, Nina, Jochner, Anton, Niewoehner, Jens, Wolf, Luise, Fueth, Matthias, Ebeling, Martin, Villaseñor, Roberto, Zhu, Yanping, Deen, Matthew C., Shan, Xiaoyang, Ehsaei, Zahra, Taylor, Verdon, Sidransky, Ellen, Vocadlo, David J., Freskgård, Per-Ola, Jagasia, Ravi
Published in Nature communications (12.04.2023)
Published in Nature communications (12.04.2023)
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Journal Article
Alzheimer's Risk Gene TREM2 Determines Functional Properties of New Type of Human iPSC-Derived Microglia
Reich, Marvin, Paris, Iñaki, Ebeling, Martin, Dahm, Nadine, Schweitzer, Christophe, Reinhardt, Dieter, Schmucki, Roland, Prasad, Megana, Köchl, Fabian, Leist, Marcel, Cowley, Sally A, Zhang, Jitao David, Patsch, Christoph, Gutbier, Simon, Britschgi, Markus
Published in Frontiers in immunology (03.02.2021)
Published in Frontiers in immunology (03.02.2021)
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Journal Article
A Sensitive In Vitro Approach to Assess the Hybridization-Dependent Toxic Potential of High Affinity Gapmer Oligonucleotides
Dieckmann, Andreas, Hagedorn, Peter H., Burki, Yvonne, Brügmann, Christine, Berrera, Marco, Ebeling, Martin, Singer, Thomas, Schuler, Franz
Published in Molecular therapy. Nucleic acids (02.03.2018)
Published in Molecular therapy. Nucleic acids (02.03.2018)
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Journal Article
Detect tissue heterogeneity in gene expression data with BioQC
Zhang, Jitao David, Hatje, Klas, Sturm, Gregor, Broger, Clemens, Ebeling, Martin, Burtin, Martine, Terzi, Fabiola, Pomposiello, Silvia Ines, Badi, Laura
Published in BMC genomics (04.04.2017)
Published in BMC genomics (04.04.2017)
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Journal Article
Selective oxytocin receptor activation prevents prefrontal circuit dysfunction and social behavioral alterations in response to chronic prefrontal cortex activation in male rats
Janz, Philipp, Knoflach, Frederic, Bleicher, Konrad, Belli, Sara, Biemans, Barbara, Schnider, Patrick, Ebeling, Martin, Grundschober, Christophe, Benekareddy, Madhurima
Published in Frontiers in cellular neuroscience (07.12.2023)
Published in Frontiers in cellular neuroscience (07.12.2023)
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Journal Article
mTORC1 Inhibition Corrects Neurodevelopmental and Synaptic Alterations in a Human Stem Cell Model of Tuberous Sclerosis
Costa, Veronica, Aigner, Stefan, Vukcevic, Mirko, Sauter, Evelyn, Behr, Katharina, Ebeling, Martin, Dunkley, Tom, Friedlein, Arno, Zoffmann, Sannah, Meyer, Claas A., Knoflach, Frédéric, Lugert, Sebastian, Patsch, Christoph, Fjeldskaar, Fatiha, Chicha-Gaudimier, Laurie, Kiialainen, Anna, Piraino, Paolo, Bedoucha, Marc, Graf, Martin, Jessberger, Sebastian, Ghosh, Anirvan, Bischofberger, Josef, Jagasia, Ravi
Published in Cell reports (Cambridge) (05.04.2016)
Published in Cell reports (Cambridge) (05.04.2016)
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Journal Article
Impaired myogenic development, differentiation and function in hESC-derived SMA myoblasts and myotubes
Hellbach, Nicole, Peterson, Suzanne, Haehnke, Daniel, Shankar, Aditi, LaBarge, Samuel, Pivaroff, Cullen, Saenger, Stefanie, Thomas, Carolin, McCarthy, Kathleen, Ebeling, Martin, Hayhurst Bennett, Monica, Schmidt, Uli, Metzger, Friedrich
Published in PloS one (10.10.2018)
Published in PloS one (10.10.2018)
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Journal Article
Epithelial cells as active player in fibrosis: findings from an in vitro model
Moll, Solange, Ebeling, Martin, Weibel, Franziska, Farina, Annarita, Araujo Del Rosario, Andrea, Hoflack, Jean Christophe, Pomposiello, Silvia, Prunotto, Marco
Published in PloS one (14.02.2013)
Published in PloS one (14.02.2013)
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Journal Article
Development of a 5-plex SILAC Method Tuned for the Quantitation of Tyrosine Phosphorylation Dynamics
Tzouros, Manuel, Golling, Sabrina, Avila, David, Lamerz, Jens, Berrera, Marco, Ebeling, Martin, Langen, Hanno, Augustin, Angélique
Published in Molecular & cellular proteomics (01.11.2013)
Published in Molecular & cellular proteomics (01.11.2013)
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