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vector therapy expression number analysis gene therapy insertion blood figure level study lentiviral b cells patient leukemia spleen il 2rg mice scid-x transduced integration t-cell transplantation sffv.corag sample γ cpr-co γc reconstitution order antibody group promoter rag 1 expression wild-type 1-/- region t cells spleen cells american society transgene hematopoietic table result development sequence cpr-co b-cell protein murine sffv.corag 1-treated mice fl ow cytometry genomic pretransplant conditioning rag 1à/à mice month ficiency function receptor conditioning notch virus correction trial scid-x 1 rag 1 ucoe.corag chromosome immunodeficiency mutation rearrangement response clone animal population transfer lmo 2 1-treated cancer therapy vol wild-type cells insertion sites flow cytometry analysis
4 Most Recent Publications
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Insertion sites in engrafted cells cluster within a limited repertoire of genomic areas after gammaretroviral vector gene therapy
(Article)
Deichmann, A. Brugman, M.H. Grez, M. Hacein-Bey-Abina, S. Cavazzana-Calvo, M. Fischer, A. Paruzynski, A. Gabriel, R. Abel, U. Glimm, H. Cattoglio, C. Mavilio, F. Bartholomae, C. Cassani, B. Aiuti, A. Dunbar, C.E. Baum, C. Gaspar, H.B. Thrasher, A.J. Kalle, C. von Schmidt, M. Wagemaker, G. Schwarzwaelder, K. Verstegen, M.M.A. Howe, S.J. Arens, A. Ott, M.G. Hoelzer, D. Seger, R. |
2011-11-01
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Correction of murine SCID-X1 by lentiviral gene therapy using a codon-optimized IL2RG gene and minimal pretransplant conditioning
(Article)
Huston, M.W. Til, N.P. van Baum, J.A.C. Kalle, C. von Mavilio, F. Zhang, F. Blundell, M.P. Thrasher, A.J. Verstegen, M.M.A. Wagemaker, G. Visser, T.P. Arshad, S.H. Brugman, M.H. Cattoglio, C. Nowrouzi, A. Li, Y.J. Schambach, A. Schmidt, M.K. |
2011-10-01
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Correction of murine Rag1 deficiency by self-inactivating lentiviral vector-mediated gene transfer
(Article)
Pike-Overzet, K. Rodijk, M. Bredius, R.G.M. Driessen, G.J.A. Thrasher, A.J. Baum, J.A.C. Cavazzana-Calvo, M. Dongen, J.J.M. van Staal, F.J.T. Ng, Y.Y. Baert, M.R. Lagresle-Peyrou, C. Schambach, A. Zhang, F. Hoeben, R.C. Hacein-Bey-Abina, S. Lankester, A.C. |
2011-09-01
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Insertional mutagenesis combined with acquired somatic mutations causes leukemogenesis following gene therapy of SCID-X1 patients
(Article)
Howe, S.J. Mansour, M.R. Gilmour, K.C. Adams, S.A. Thornhill, S.I. Parsley, K.L. Staal, F.J.T. Gale, R.E. Linch, D.C. Bayford, J. Brown, L. Quaye, M. Schwarzwaelder, K. Kinnon, C. Ancliff, P. Webb, D. Schmidt, M. Kalle, C. von Gaspar, H.B. Thrasher, A.J. Bartholomae, C. Hubank, M. Kempski, H. Brugman, M.H. Pike-Overzet, K. Chatters, S.J. Ridder, D. de |
2008-09-02
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