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Refinado por: assunto: Gene Therapy remover
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1
Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells
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Detailed comparison of retroviral vectors and promoter configurations for stable and high transgene expression in human induced pluripotent stem cells

Hoffmann, D ; Schott, J W ; Geis, F K ; Lange, L ; Müller, F-J ; Lenz, D ; Zychlinski, D ; Steinemann, D ; Morgan, M ; Moritz, T ; Schambach, A

Gene therapy, 2017-05, Vol.24 (5), p.298-307 [Periódico revisado por pares]

England: Nature Publishing Group

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2
A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency
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A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency

Hacein-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 J

The New England journal of medicine, 2014-10, Vol.371 (15), p.1407-1417 [Periódico revisado por pares]

Waltham, MA: Massachusetts Medical Society

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3
Lentiviral Gene Therapy Using Cellular Promoters Cures Type 1 Gaucher Disease in Mice
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Lentiviral Gene Therapy Using Cellular Promoters Cures Type 1 Gaucher Disease in Mice

Dahl, 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, Stefan

Molecular therapy, 2015-05, Vol.23 (5), p.835-844 [Periódico revisado por pares]

United States: Elsevier Inc

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4
Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer
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Artigo
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Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer

PIKE-OVERZET, K ; RODIJK, M ; BREDIUS, R. G. M ; DRIESSEN, G. J. A ; THRASHER, A. J ; BAUM, C ; CAVAZZANA-CALVO, M ; VAN DONGEN, J. J. M ; STAAL, F. J. T ; NG, Y.-Y ; BAERT, M. R. M ; LAGRESLE-PEYROU, C ; SCHAMBACH, A ; ZHANG, F ; HOEBEN, R. C ; HACEIN-BEY-ABINA, S ; LANKESTER, A. C

Leukemia, 2011-09, Vol.25 (9), p.1471-1483 [Periódico revisado por pares]

Avenel, NJ: Nature Publishing Group

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5
Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression
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Artigo
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Woodchuck hepatitis virus post-transcriptional regulatory element deleted from X protein and promoter sequences enhances retroviral vector titer and expression

SCHAMBACH, A ; BOHNE, J ; BAUM, C ; HERMANN, F. G ; EGERER, L ; VON LAER, D ; GIROGLOU, T

Gene therapy, 2006-04, Vol.13 (7), p.641-645 [Periódico revisado por pares]

Basingstoke: Nature Publishing Group

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6
Improved retroviral episome transfer of transcription factors enables sustained cell fate modification
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Improved retroviral episome transfer of transcription factors enables sustained cell fate modification

Schott, J W ; Hoffmann, D ; Maetzig, T ; Müller, F-J ; Steinemann, D ; Zychlinski, D ; Cantz, T ; Baum, C ; Schambach, A

Gene therapy, 2014-11, Vol.21 (11), p.938-949 [Periódico revisado por pares]

England: Nature Publishing Group

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7
Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease
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Artigo
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Preclinical Optimization and Safety Studies of a New Lentiviral Gene Therapy for p47phox-Deficient Chronic Granulomatous Disease

Schejtman, Andrea ; Vetharoy, Winston ; Choi, Uimook ; Rivat, Christine ; Theobald, Narda ; Piras, Giuseppa ; Leon-Rico, Diego ; Buckland, Karen ; Armenteros-Monterroso, Elena ; Benedetti, Sara ; Ashworth, Michael T. ; Rothe, Michael ; Schambach, Axel ; Gaspar, Hubert Bobby ; Kang, Elizabeth M. ; Malech, Harry L. ; Thrasher, Adrian J. ; Santilli, Giorgia

Human gene therapy, 2021-09, Vol.32 (17-18), p.949-958 [Periódico revisado por pares]

New Rochelle: Mary Ann Liebert, Inc

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8
Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID
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Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID

Charrier, Sabine ; Lagresle-Peyrou, Chantal ; Poletti, Valentina ; Rothe, Michael ; Cédrone, Grégory ; Gjata, Bernard ; Mavilio, Fulvio ; Fischer, Alain ; Schambach, Axel ; de Villartay, Jean-Pierre ; Cavazzana, Marina ; Hacein-Bey-Abina, Salima ; Galy, Anne

Mol Ther Methods Clin Dev, 2019-12, Vol.15, p.232-245 [Periódico revisado por pares]

United States: Elsevier Inc

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9
Correction of B-cell development in Btk-deficient mice using lentiviral vectors with codon-optimized human BTK
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Artigo
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Correction of B-cell development in Btk-deficient mice using lentiviral vectors with codon-optimized human BTK

NG, Y. Y ; BAERT, M. R. M ; PIKE-OVERZET, K ; RODIJK, M ; BRUGMAN, M. H ; SCHAMBACH, A ; BAUM, C ; HENDRIKS, R. W ; VAN DONGEN, J. J. M ; STAAL, F. J. T

Leukemia, 2010-09, Vol.24 (9), p.1617-1630 [Periódico revisado por pares]

Avenel, NJ: Nature Publishing Group

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10
Transcriptional Enhancers Induce Insertional Gene Deregulation Independently From the Vector Type and Design
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Transcriptional Enhancers Induce Insertional Gene Deregulation Independently From the Vector Type and Design

Maruggi, Giulietta ; Porcellini, Simona ; Facchini, Giulia ; Perna, Serena K ; Cattoglio, Claudia ; Sartori, Daniela ; Ambrosi, Alessandro ; Schambach, Axel ; Baum, Christopher ; Bonini, Chiara ; Bovolenta, Chiara ; Mavilio, Fulvio ; Recchia, Alessandra

Molecular therapy, 2009-05, Vol.17 (5), p.851-856 [Periódico revisado por pares]

United States: Elsevier Inc

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