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Titel:

Clonal evidence for the transduction of CD34+ cells with lymphomyeloid differentiation potential and self-renewal capacity in the SCID-X1 gene therapy trial.

Dokumenttyp:
Clinical Trial; Journal Article
Autor(en):
Schmidt, M; Hacein-Bey-Abina, S; Wissler, M; Carlier, F; Lim, A; Prinz, C; Glimm, H; Andre-Schmutz, I; Hue, C; Garrigue, A; Le Deist, F; Lagresle, C; Fischer, A; Cavazzana-Calvo, M; von Kalle, C
Abstract:
Immune function has been restored in 9 of 10 children with X-linked severe combined immunodeficiency by gamma c gene transfer in CD34+ cells. The distribution of both T-cell receptor (TCR) V beta family usage and TCR V beta complementarity-determining region 3 (CDR3) length revealed a broadly diversified T-cell repertoire. Retroviral integration site analysis in T cells demonstrated a high number of distinct insertion sites, indicating polyclonality of genetically corrected cell clones, in all p...     »
Zeitschriftentitel:
Blood
Jahr:
2005
Band / Volume:
105
Heft / Issue:
7
Seitenangaben Beitrag:
2699-706
Sprache:
eng
Volltext / DOI:
doi:10.1182/blood-2004-07-2648
PubMed:
http://view.ncbi.nlm.nih.gov/pubmed/15585650
Print-ISSN:
0006-4971
TUM Einrichtung:
II. Medizinische Klinik und Poliklinik (Gastroenterologie)
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