Result Number | Material Type | Add to My Shelf Action | Record Details and Options |
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1 |
Material Type: Artigo
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Insertional Transformation of Hematopoietic Cells by Self-inactivating Lentiviral and Gammaretroviral VectorsModlich, Ute ; Navarro, Susana ; Zychlinski, Daniela ; Maetzig, Tobias ; Knoess, Sabine ; Brugman, Martijn H. ; Schambach, Axel ; Charrier, Sabine ; Galy, Anne ; Thrasher, Adrian J. ; Bueren, Juan ; Baum, ChristopherMolecular therapy, 2009-11, Vol.17 (11), p.1919-1928 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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2 |
Material Type: Artigo
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Lentiviral Gene Therapy Using Cellular Promoters Cures Type 1 Gaucher Disease in MiceDahl, Maria ; Doyle, Alexander ; Olsson, Karin ; Månsson, Jan-Eric ; Marques, André R A ; Mirzaian, Mina ; Aerts, Johannes M ; Ehinger, Mats ; Rothe, Michael ; Modlich, Ute ; Schambach, Axel ; Karlsson, StefanMolecular therapy, 2015-05, Vol.23 (5), p.835-844 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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3 |
Material Type: Artigo
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Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cellsHoffmann, D ; Schott, J W ; Geis, F K ; Lange, L ; Müller, F-J ; Lenz, D ; Zychlinski, D ; Steinemann, D ; Morgan, M ; Moritz, T ; Schambach, AGene therapy, 2017-05, Vol.24 (5), p.298-307 [Periódico revisado por pares]England: Nature Publishing GroupTexto completo disponível |
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4 |
Material Type: Artigo
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Betibeglogene Autotemcel Gene Therapy for Non–β0/β0 Genotype β-ThalassemiaLocatelli, Franco ; Thompson, Alexis A ; Kwiatkowski, Janet L ; Porter, John B ; Thrasher, Adrian J ; Hongeng, Suradej ; Sauer, Martin G ; Thuret, Isabelle ; Lal, Ashutosh ; Algeri, Mattia ; Schneiderman, Jennifer ; Olson, Timothy S ; Carpenter, Ben ; Amrolia, Persis J ; Anurathapan, Usanarat ; Schambach, Axel ; Chabannon, Christian ; Schmidt, Manfred ; Labik, Ivan ; Elliot, Heidi ; Guo, Ruiting ; Asmal, Mohammed ; Colvin, Richard A ; Walters, Mark CThe New England journal of medicine, 2022-02, Vol.386 (5), p.415-427 [Periódico revisado por pares]Boston: Massachusetts Medical SocietyTexto completo disponível |
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5 |
Material Type: Artigo
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Capsid Engineering Overcomes Barriers Toward Adeno-Associated Virus Vector-Mediated Transduction of Endothelial CellsZhang, L ; Rossi, A ; Lange, L ; Meumann, N ; Koitzsch, U ; Christie, K ; Nesbit, M A ; Moore, C B T ; Hacker, U T ; Morgan, M ; Hoffmann, D ; Zengel, J ; Carette, J E ; Schambach, A ; Salvetti, A ; Odenthal, M ; Büning, HHuman gene therapy, 2019-10, Vol.30 (10), p.1284-1296 [Periódico revisado por pares]United States: Mary Ann Liebert, IncTexto completo disponível |
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6 |
Material Type: Artigo
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A new age of precision gene therapySchambach, Axel ; Buchholz, Christian J ; Torres-Ruiz, Raul ; Cichutek, Klaus ; Morgan, Michael ; Trapani, Ivana ; Büning, HildegardThe Lancet (British edition), 2024-02, Vol.403 (10426), p.568-582 [Periódico revisado por pares]England: Elsevier LtdTexto completo disponível |
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7 |
Material Type: Artigo
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Lentivirus Mediated Correction of Artemis-Deficient Severe Combined ImmunodeficiencyPunwani, Divya ; Kawahara, Misako ; Yu, Jason ; Sanford, Ukina ; Roy, Sushmita ; Patel, Kiran ; Carbonaro, Denise A ; Karlen, Andrea D ; Khan, Sara ; Cornetta, Kenneth ; Rothe, Michael ; Schambach, Axel ; Kohn, Donald B ; Malech, Harry L ; McIvor, R Scott ; Puck, Jennifer M ; Cowan, Morton JHuman gene therapy, 2017-01, Vol.28 (1), p.112-124 [Periódico revisado por pares]United States: Mary Ann Liebert, IncTexto completo disponível |
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8 |
Material Type: Artigo
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Lentiviral vectors containing an enhancer-less ubiquitously acting chromatin opening element (UCOE) provide highly reproducible and stable transgene expression in hematopoietic cellsZhang, Fang ; Thornhill, Susannah I. ; Howe, Steven J. ; Ulaganathan, Meera ; Schambach, Axel ; Sinclair, Joanna ; Kinnon, Christine ; Gaspar, H. Bobby ; Antoniou, Michael ; Thrasher, Adrian J.Blood, 2007-09, Vol.110 (5), p.1448-1457 [Periódico revisado por pares]Washington, DC: Elsevier IncTexto completo disponível |
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9 |
Material Type: Artigo
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Self-inactivating Gammaretroviral Vectors for Gene Therapy of X-linked Severe Combined ImmunodeficiencyThornhill, Susannah I ; Schambach, Axel ; Howe, Steven J ; Ulaganathan, Meera ; Grassman, Elke ; Williams, David ; Schiedlmeier, Bernhard ; Sebire, Neil J ; Gaspar, H Bobby ; Kinnon, Christine ; Baum, Christopher ; Thrasher, Adrian JMolecular therapy, 2008-03, Vol.16 (3), p.590-598 [Periódico revisado por pares]United States: Elsevier IncTexto completo disponível |
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10 |
Material Type: Artigo
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A Modified γ-Retrovirus Vector for X-Linked Severe Combined ImmunodeficiencyHacein-Bey-Abina, Salima ; Pai, Sung-Yun ; Gaspar, H. Bobby ; Armant, Myriam ; Berry, Charles C ; Blanche, Stephane ; Bleesing, Jack ; Blondeau, Johanna ; de Boer, Helen ; Buckland, Karen F ; Caccavelli, Laure ; Cros, Guilhem ; De Oliveira, Satiro ; Fernández, Karen S ; Guo, Dongjing ; Harris, Chad E ; Hopkins, Gregory ; Lehmann, Leslie E ; Lim, Annick ; London, Wendy B ; van der Loo, Johannes C.M ; Malani, Nirav ; Male, Frances ; Malik, Punam ; Marinovic, M. Angélica ; McNicol, Anne-Marie ; Moshous, Despina ; Neven, Benedicte ; Oleastro, Matías ; Picard, Capucine ; Ritz, Jerome ; Rivat, Christine ; Schambach, Axel ; Shaw, Kit L ; Sherman, Eric A ; Silberstein, Leslie E ; Six, Emmanuelle ; Touzot, Fabien ; Tsytsykova, Alla ; Xu-Bayford, Jinhua ; Baum, Christopher ; Bushman, Frederic D ; Fischer, Alain ; Kohn, Donald B ; Filipovich, Alexandra H ; Notarangelo, Luigi D ; Cavazzana, Marina ; Williams, David A ; Thrasher, Adrian JThe New England journal of medicine, 2014-10, Vol.371 (15), p.1407-1417 [Periódico revisado por pares]Waltham, MA: Massachusetts Medical SocietyTexto completo disponível |