Gammaretrovirus-mediated correction of SCID-X1 is associated with skewed vector integration site distribution in vivo

We treated 10 children with X-linked SCID (SCID-X1) using gammaretrovirus-mediated gene transfer. Those with sufficient follow-up were found to have recovered substantial immunity in the absence of any serious adverse events up to 5 years after treatment. To determine the influence of vector integra...

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Veröffentlicht in:	The Journal of clinical investigation 2007-08, Vol.117 (8), p.2241-2249
Hauptverfasser:	Schwarzwaelder, Kerstin, Howe, Steven J, Schmidt, Manfred, Brugman, Martijn H, Deichmann, Annette, Glimm, Hanno, Schmidt, Sonja, Prinz, Claudia, Wissler, Manuela, King, Douglas J S, Zhang, Fang, Parsley, Kathryn L, Gilmour, Kimberly C, Sinclair, Joanna, Bayford, Jinhua, Peraj, Rachel, Pike-Overzet, Karin, Staal, Frank J T, de Ridder, Dick, Kinnon, Christine, Abel, Ulrich, Wagemaker, Gerard, Gaspar, H Bobby, Thrasher, Adrian J, von Kalle, Christof
Format:	Artikel
Sprache:	eng
Schlagworte:	Adult Biomedical research Care and treatment CD3 Complex Cell Proliferation Cell Survival - genetics Cell Survival - immunology Child Child, Preschool Female Follow-Up Studies Gammaretrovirus Genetic aspects Genetic Vectors Graft Survival - genetics Graft Survival - immunology Hematopoietic Stem Cell Transplantation Hematopoietic Stem Cells - immunology Humans Infant Lymphocytes Male Severe combined immunodeficiency T-Lymphocytes - immunology Transduction, Genetic Transplantation, Autologous Virus Integration X-Linked Combined Immunodeficiency Diseases - genetics X-Linked Combined Immunodeficiency Diseases - immunology X-Linked Combined Immunodeficiency Diseases - therapy
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