WFRF:(BAUM M)
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Chance or necessity? Insertional mutagenesis in gene therapy and its consequences
- Baum, C (author)
- von Kalle, C (author)
- Staal, F J T (author)
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- Li, Z X (author)
- Fehse, B (author)
- Schmidt, M (author)
- Weerkamp, F (author)
- Wagemaker, G (author)
- Williams, D A (author)
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- (creator_code:org_t)
- Elsevier BV, 2004
- 2004
- English.
- In: Molecular Therapy. - : Elsevier BV. - 1525-0024 .- 1525-0016. ; 9:1, s. 5-13
- Research review (peer-reviewed)
Abstract
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- 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.
Subject headings
- MEDICIN OCH HÄLSOVETENSKAP -- Medicinska och farmaceutiska grundvetenskaper -- Medicinsk genetik (hsv//swe)
- MEDICAL AND HEALTH SCIENCES -- Basic Medicine -- Medical Genetics (hsv//eng)
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- Baum, C
- von Kalle, C
- Staal, F J T
- Li, Z X
- Fehse, B
- Schmidt, M
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- Weerkamp, F
- Karlsson, Stefan
- Wagemaker, G
- Williams, D A
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- MEDICAL AND HEALTH SCIENCES
- MEDICAL AND HEAL ...
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- and Medical Genetics
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- Molecular Therap ...
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- Lund University
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