Abstract
he emergence of leukemia following gene transfer to restore common cytokine receptor ¿ chain (¿C) function in X-linked severe combined immunodeficiency (SCID-X1) has raised important questions with respect to gene therapy safety. To explore the risk factors involved, we tested the oncogenic potential of human ¿C in new strains of transgenic mice expressing the gene under the control of the CD2 promoter and locus control region (LCR).
Original language | English |
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Pages (from-to) | 1031-1038 |
Number of pages | 8 |
Journal | Molecular Therapy |
Volume | 17 |
Issue number | 6 |
DOIs | |
Publication status | Published - 1 Mar 2009 |
Keywords
- gene therapy
- leukemia
- cytokine receptors