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Gene therapy : Therapeutic gene causing lymphoma

Woods, Niels Bjarne LU ; Bottero, Virginie ; Schmidt, Manfred ; Von Kalle, Christof and Verma, Inder M. (2006) In Nature 440(7088). p.1123-1123
Abstract

The development of T-cell leukaemia following the otherwise successful treatment of three patients with X-linked severe combined immune deficiency (X-SCID) in gene-therapy trials using haematopoietic stem cells has led to a re-evaluation of this approach. Using a mouse model for gene therapy of X-SCID, we find that the corrective therapeutic gene IL2RG itself can act as a contributor to the genesis of T-cell lymphomas, with one-third of animals being affected. Gene-therapy trials for X-SCID, which have been based on the assumption that IL2RG is minimally oncogenic, may therefore pose some risk to patients.

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author
publishing date
type
Contribution to journal
publication status
published
keywords
Animals, Cell Transformation, Neoplastic/genetics, Clinical Trials as Topic/adverse effects, Disease Models, Animal, Dogs, Genetic Therapy/adverse effects, Hematopoietic Stem Cell Transplantation, Humans, Lymphoma, T-Cell/etiology, Mice, Mice, SCID, Oncogenes/genetics, Receptors, Interleukin-2/genetics, Severe Combined Immunodeficiency/genetics, Time Factors
in
Nature
volume
440
issue
7088
pages
1 pages
publisher
Nature Publishing Group
external identifiers
  • scopus:33646361846
  • pmid:16641981
ISSN
0028-0836
DOI
10.1038/4401123a
language
English
LU publication?
no
id
71dd568d-95de-4a30-845e-d9356a4b12a1
date added to LUP
2017-01-26 08:30:16
date last changed
2020-04-07 04:06:55
@article{71dd568d-95de-4a30-845e-d9356a4b12a1,
  abstract     = {<p>The development of T-cell leukaemia following the otherwise successful treatment of three patients with X-linked severe combined immune deficiency (X-SCID) in gene-therapy trials using haematopoietic stem cells has led to a re-evaluation of this approach. Using a mouse model for gene therapy of X-SCID, we find that the corrective therapeutic gene IL2RG itself can act as a contributor to the genesis of T-cell lymphomas, with one-third of animals being affected. Gene-therapy trials for X-SCID, which have been based on the assumption that IL2RG is minimally oncogenic, may therefore pose some risk to patients.</p>},
  author       = {Woods, Niels Bjarne and Bottero, Virginie and Schmidt, Manfred and Von Kalle, Christof and Verma, Inder M.},
  issn         = {0028-0836},
  language     = {eng},
  month        = {04},
  number       = {7088},
  pages        = {1123--1123},
  publisher    = {Nature Publishing Group},
  series       = {Nature},
  title        = {Gene therapy : Therapeutic gene causing lymphoma},
  url          = {http://dx.doi.org/10.1038/4401123a},
  doi          = {10.1038/4401123a},
  volume       = {440},
  year         = {2006},
}