Successful RAG1-SCID gene therapy depends on the level of RAG1 expression

[...]in mice reconstituted with low Rag1 expression, a block in development can be seen at the immature single positive to DP transition. [...]the level of RAG1 expression in transduced stem cells and after transplantation and reconstitution in lymphocytes is a crucial determinant of outcome.

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Bibliographische Detailangaben
Veröffentlicht in:	Journal of allergy and clinical immunology 2014-07, Vol.134 (1), p.242-243
Hauptverfasser:	Pike-Overzet, Karin, PhD, Baum, Christopher, MD, Bredius, Robbert G.M., MD, PhD, Cavazzana, Marina, MD, PhD, Driessen, Gert-Jan, MD, PhD, Fibbe, Willem E., MD, PhD, Gaspar, H. Bobby, MD, PhD, Hoeben, Rob C., PhD, Lagresle-Peyrou, Chantal, PhD, Lankester, Arjan, MD, PhD, Meij, Pauline, PhD, Schambach, Axel, MD, PhD, Thrasher, Adrian, MD, PhD, Van Dongen, Jacques J.M., MD, PhD, Zwaginga, Jaap-Jan, MD, PhD, Staal, Frank J.T., PhD
Format:	Artikel
Sprache:	eng
Schlagworte:	Allergy and Immunology Animals Female Gene expression Gene therapy Genetic Therapy Genetic Vectors - genetics Homeodomain Proteins - genetics Humans Immune system Lentivirus - genetics Male Severe Combined Immunodeficiency - genetics Severe Combined Immunodeficiency - therapy
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Beschreibung
Zusammenfassung:	[...]in mice reconstituted with low Rag1 expression, a block in development can be seen at the immature single positive to DP transition. [...]the level of RAG1 expression in transduced stem cells and after transplantation and reconstitution in lymphocytes is a crucial determinant of outcome.
ISSN:	0091-6749 1097-6825
DOI:	10.1016/j.jaci.2014.04.033