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 |
|---|---|
| Pages (from-to) | 1031-1038 |
| Number of pages | 8 |
| Journal | Molecular Therapy |
| Volume | 17 |
| Issue number | 6 |
| DOIs | |
| Publication status | Published - 1 Mar 2009 |
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Keywords
- gene therapy
- leukemia
- cytokine receptors
Cite this
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A novel model of SCID-X1 reconstitution reveals predisposition to retrovirus-induced lymphoma but no evidence of ¿C gene oncogenicity. / Scobie, Linda; Hector, R.; Grant, Louise; Bell, Margaret; Neilsen, Anne A.; Meikle, Sharon; Philbey, Adrain; Thrasher, Adrain J.
In: Molecular Therapy, Vol. 17, No. 6, 01.03.2009, p. 1031-1038.Research output: Contribution to journal › Article
TY - JOUR
T1 - A novel model of SCID-X1 reconstitution reveals predisposition to retrovirus-induced lymphoma but no evidence of ¿C gene oncogenicity
AU - Scobie, Linda
AU - Hector, R.
AU - Grant, Louise
AU - Bell, Margaret
AU - Neilsen, Anne A.
AU - Meikle, Sharon
AU - Philbey, Adrain
AU - Thrasher, Adrain J.
N1 - Originally published in: Molecular Therapy (2009), 17 (2), pp.1031-1038.
PY - 2009/3/1
Y1 - 2009/3/1
N2 - 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).
AB - 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).
KW - gene therapy
KW - leukemia
KW - cytokine receptors
UR - http://www.scopus.com/inward/record.url?eid=2-s2.0-67349162619&partnerID=8YFLogxK
U2 - 10.1038/mt.2009.59
DO - 10.1038/mt.2009.59
M3 - Article
VL - 17
SP - 1031
EP - 1038
JO - Molecular Therapy
JF - Molecular Therapy
SN - 1525-0016
IS - 6
ER -