Correction of IVS I-110(G>A) β-thalassemia by CRISPR/Cas-and TALEN-mediated disruption of aberrant regulatory elements in human hematopoietic stem and progenitor cells
Patsali, Petros, Turchiano, Giandomenico, Papasavva, Panayiota, Romito, Marianna, Loucari, Constantinos C, Stephanou, Coralea, Christou, Soteroulla, Sitarou, Maria, Mussolino, Claudio, Cornu, Tatjana I, Antoniou, Michael N, Lederer, Carsten W, Cathomen, Toni, Kleanthous, Marina
Published in Haematologica (Roma) (01.11.2019)
Published in Haematologica (Roma) (01.11.2019)
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Unexpected failure rates for modular assembly of engineered zinc fingers
Joung, J Keith, Ramirez, Cherie L, Foley, Jonathan E, Wright, David A, Müller-Lerch, Felix, Rahman, Shamim H, Cornu, Tatjana I, Winfrey, Ronnie J, Sander, Jeffry D, Fu, Fengli, Townsend, Jeffrey A, Cathomen, Toni, Voytas, Daniel F
Published in Nature methods (01.05.2008)
Published in Nature methods (01.05.2008)
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On- and off-target effects of paired CRISPR-Cas nickase in primary human cells
Klermund, Julia, Rhiel, Manuel, Kocher, Thomas, Chmielewski, Kay Ole, Bischof, Johannes, Andrieux, Geoffroy, el Gaz, Melina, Hainzl, Stefan, Boerries, Melanie, Cornu, Tatjana I., Koller, Ulrich, Cathomen, Toni
Published in Molecular therapy (01.05.2024)
Published in Molecular therapy (01.05.2024)
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HIV Gene Therapy: An Update
Cornu, Tatjana I, Mussolino, Claudio, Müller, Matthias C, Wehr, Claudia, Kern, Winfried V, Cathomen, Toni
Published in Human gene therapy (01.01.2021)
Published in Human gene therapy (01.01.2021)
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Gene editing of hematopoietic stem cells restores T-cell response in familial hemophagocytic lymphohistiocytosis
Dettmer-Monaco, Viviane, Weißert, Kristoffer, Ammann, Sandra, Monaco, Gianni, Lei, Lei, Gräßel, Linda, Rhiel, Manuel, Rositzka, Julia, Kaufmann, Masako M, Geiger, Kerstin, Andrieux, Geoffroy, Lao, Jessica, Thoulass, Gudrun, Schell, Christoph, Boerries, Melanie, Illert, Anna L, Cornu, Tatjana I, Ehl, Stephan, Aichele, Peter, Cathomen, Toni
Published in Journal of allergy and clinical immunology (01.01.2024)
Published in Journal of allergy and clinical immunology (01.01.2024)
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Epitope-engineered human hematopoietic stem cells are shielded from CD123-targeted immunotherapy
Marone, Romina, Landmann, Emmanuelle, Devaux, Anna, Lepore, Rosalba, Seyres, Denis, Zuin, Jessica, Burgold, Thomas, Engdahl, Corinne, Capoferri, Giuseppina, Dell'Aglio, Alessandro, Larrue, Clément, Simonetta, Federico, Rositzka, Julia, Rhiel, Manuel, Andrieux, Geoffroy, Gallagher, Danielle N, Schröder, Markus S, Wiederkehr, Amélie, Sinopoli, Alessandro, Do Sacramento, Valentin, Haydn, Anna, Garcia-Prat, Laura, Divsalar, Christopher, Camus, Anna, Xu, Liwen, Bordoli, Lorenza, Schwede, Torsten, Porteus, Matthew, Tamburini, Jérôme, Corn, Jacob E, Cathomen, Toni, Cornu, Tatjana I, Urlinger, Stefanie, Jeker, Lukas T
Published in The Journal of experimental medicine (04.12.2023)
Published in The Journal of experimental medicine (04.12.2023)
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Disruption of HIV-1 co-receptors CCR5 and CXCR4 in primary human T cells and hematopoietic stem and progenitor cells using base editing
Knipping, Friederike, Newby, Gregory A., Eide, Cindy R., McElroy, Amber N., Nielsen, Sarah C., Smith, Kyle, Fang, Yongxing, Cornu, Tatjana I., Costa, Caroline, Gutierrez-Guerrero, Alejandra, Bingea, Samuel P., Feser, Colby J., Steinbeck, Benjamin, Hippen, Keli L., Blazar, Bruce R., McCaffrey, Anton, Mussolino, Claudio, Verhoeyen, Els, Tolar, Jakub, Liu, David R., Osborn, Mark J.
Published in Molecular therapy (05.01.2022)
Published in Molecular therapy (05.01.2022)
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Correction: Patients and mice with deficiency in the SNARE protein SYNTAXIN-11 have a secondary B cell defect
Kögl, Tamara, Chang, Hsin-Fang, Staniek, Julian, Chiang, Samuel C C, Thoulass, Gudrun, Lao, Jessica, Weißert, Kristoffer, Dettmer-Monaco, Viviane, Geiger, Kerstin, Manna, Paul T, Beziat, Vivien, Momenilandi, Mana, Tu, Szu-Min, Keppler, Selina J, Pattu, Varsha, Wolf, Philipp, Kupferschmid, Laurence, Tholen, Stefan, Covill, Laura E, Ebert, Karolina, Straub, Tobias, Groß, Miriam, Gather, Ruth, Engel, Helena, Salzer, Ulrich, Schell, Christoph, Maier, Sarah, Lehmberg, Kai, Cornu, Tatjana I, Pircher, Hanspeter, Shahrooei, Mohammad, Parvaneh, Nima, Elling, Roland, Rizzi, Marta, Bryceson, Yenan T, Ehl, Stephan, Aichele, Peter, Ammann, Sandra
Published in The Journal of experimental medicine (01.07.2024)
Published in The Journal of experimental medicine (01.07.2024)
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Journal Article
Patients and mice with deficiency in the SNARE protein SYNTAXIN-11 have a secondary B cell defect
Kögl, Tamara, Chang, Hsin-Fang, Staniek, Julian, Chiang, Samuel C C, Thoulass, Gudrun, Lao, Jessica, Weißert, Kristoffer, Dettmer-Monaco, Viviane, Geiger, Kerstin, Manna, Paul T, Beziat, Vivien, Momenilandi, Mana, Tu, Szu-Min, Keppler, Selina J, Pattu, Varsha, Wolf, Philipp, Kupferschmid, Laurence, Tholen, Stefan, Covill, Laura E, Ebert, Karolina, Straub, Tobias, Groß, Miriam, Gather, Ruth, Engel, Helena, Salzer, Ulrich, Schell, Christoph, Maier, Sarah, Lehmberg, Kai, Cornu, Tatjana I, Pircher, Hanspeter, Shahrooei, Mohammad, Parvaneh, Nima, Elling, Roland, Rizzi, Marta, Bryceson, Yenan T, Ehl, Stephan, Aichele, Peter, Ammann, Sandra
Published in The Journal of experimental medicine (01.07.2024)
Published in The Journal of experimental medicine (01.07.2024)
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Versatile and efficient genome editing in human cells by combining zinc-finger nucleases with adeno-associated viral vectors
Händel, Eva-Maria, Gellhaus, Katharina, Khan, Kafaitullah, Bednarski, Christien, Cornu, Tatjana I, Müller-Lerch, Felix, Kotin, Robert M, Heilbronn, Regine, Cathomen, Toni
Published in Human gene therapy (01.03.2012)
Published in Human gene therapy (01.03.2012)
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Editing CCR5: a novel approach to HIV gene therapy
Cornu, Tatjana I, Mussolino, Claudio, Bloom, Kristie, Cathomen, Toni
Published in Advances in experimental medicine and biology (01.01.2015)
Published in Advances in experimental medicine and biology (01.01.2015)
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Function-Preserving Single Amino Acid Substitutions Shield Hematopoietic Stem and Progenitor Cells from CD117 Targeted Immunotherapy In Vivo
Marone, Romina, Lepore, Rosalba, Rositzka, Julia, Dettmer-Monaco, Viviane, dell' Aglio, Alessandro, Capoferri, Giuseppina, Brault, Julie, Ten Buren, Emiel, Burgold, Thomas, Camus, Anna, Divsalar, Christopher, Garcia Prat, Laura, Haydn, Anna, Hermann, Felix, Heugel, Marcel, Knezevic, Andreja, Lehmann, Frank Michael, Do Sacramento, Valentin, Sinopoli, Alessandro, Wellinger, Lisa C, Wiederkehr, Amélie, Yumlu, Saniye, Cathomen, Toni, Winkler, Thomas, Cornu, Tatjana I, Urlinger, Stefanie, Jeker, Lukas T
Published in Blood (15.11.2022)
Published in Blood (15.11.2022)
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A Highly Efficient and GMP Compliant Protocol to Manufacture CCR5 Edited Cells to Treat HIV Infection
Romito, Marianna, Meyer, Emily, Poddar, Sushmita, Heinz, Helena, Rositzka, Julia, Hildenbeutel, Markus, Gautron, Anne-Sophie, Busser, Brian W, Kok, Yik Lim, Turchiano, Giandomenico, Haas, Simone, Schäfer, Richard, Smith, Julianne, Metzner, Karin J, Mussolino, Claudio, Duchateau, Philippe, Cathomen, Toni, Cornu, Tatjana I
Published in Blood (29.11.2018)
Published in Blood (29.11.2018)
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Gene Therapy Getting Personal: Mutation-Specific Editing and Gene Addition Strategies for β-Thalassaemia
Lederer, Carsten W., Patsali, Petros, Mussolino, Claudio, Turchiano, Giandomenico, Papasavva, Panayiota, Romito, Marianna, Stephanou, Coralea, Cornu, Tatjana I., Kolnagou, Annita, Christou, Soteroula, Sitarou, Maria, Antoniou, Mike N., Cathomen, Toni, Kleanthous, Marina
Published in Hemoglobin (02.11.2019)
Published in Hemoglobin (02.11.2019)
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