Chance or necessity? Insertional mutagenesis in gene therapy and its consequences
Baum, C ; von Kalle, C ; Staal, F J T ; Li, Z X ; Fehse, B ; Schmidt, M ; Weerkamp, F ; Karlsson, Stefan LU
; Wagemaker, G
and Williams, D A
(2004)
In Molecular Therapy
9(1).
p.5-13
- Abstract
- Recently, unusual forms of leukemias have developed as complications following retroviral transfer of potentially therapeutic genes into hematopoietic cells. A crucial component in the pathogenesis of these complications was the upregulation of a cellular proto-oncogene by random insertion of the retroviral gene transfer vector. These findings have great implications for the genetic manipulation of somatic stem cells in medicine. This review discusses the extent to which the random oncogene activation may have required disease-specific stimuli of the transgene and the hematopoietic milieu to become leukemogenic. Based on these considerations, we propose approaches to risk prediction and prevention.
Please use this url to cite or link to this publication:
https://lup.lub.lu.se/record/288600
- author
- Baum, C
; von Kalle, C
; Staal, F J T
; Li, Z X
; Fehse, B
; Schmidt, M
; Weerkamp, F
; Karlsson, Stefan
LU
; Wagemaker, G
and Williams, D A
- organization
- publishing date
- 2004
- type
- Contribution to journal
- publication status
- published
- subject
- in
- Molecular Therapy
- volume
- 9
- issue
- 1
- pages
- 5 - 13
- publisher
- Nature Publishing Group
- external identifiers
-
- wos:000188659300007
- pmid:14741772
- scopus:10744233606
- ISSN
- 1525-0024
- DOI
- 10.1016/j.ymthe.2003.10.013
- language
- English
- LU publication?
- yes
- id
- be94cc16-1003-4393-afe5-84f0bd2e7eda (old id 288600)
- date added to LUP
- 2016-04-01 12:17:10
- date last changed
- 2022-03-13 07:50:33
@article{be94cc16-1003-4393-afe5-84f0bd2e7eda,
abstract = {{Recently, unusual forms of leukemias have developed as complications following retroviral transfer of potentially therapeutic genes into hematopoietic cells. A crucial component in the pathogenesis of these complications was the upregulation of a cellular proto-oncogene by random insertion of the retroviral gene transfer vector. These findings have great implications for the genetic manipulation of somatic stem cells in medicine. This review discusses the extent to which the random oncogene activation may have required disease-specific stimuli of the transgene and the hematopoietic milieu to become leukemogenic. Based on these considerations, we propose approaches to risk prediction and prevention.}},
author = {{Baum, C and von Kalle, C and Staal, F J T and Li, Z X and Fehse, B and Schmidt, M and Weerkamp, F and Karlsson, Stefan and Wagemaker, G and Williams, D A}},
issn = {{1525-0024}},
language = {{eng}},
number = {{1}},
pages = {{5--13}},
publisher = {{Nature Publishing Group}},
series = {{Molecular Therapy}},
title = {{Chance or necessity? Insertional mutagenesis in gene therapy and its consequences}},
url = {{http://dx.doi.org/10.1016/j.ymthe.2003.10.013}},
doi = {{10.1016/j.ymthe.2003.10.013}},
volume = {{9}},
year = {{2004}},
}