Chance or necessity? Insertional mutagenesis in gene therapy and its consequences
Författare
Summary, in English
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.
Avdelning/ar
Publiceringsår
2004
Språk
Engelska
Sidor
5-13
Publikation/Tidskrift/Serie
Molecular Therapy
Volym
9
Issue
1
Dokumenttyp
Artikel i tidskrift
Förlag
Nature Publishing Group
Ämne
- Medical Genetics
Status
Published
ISBN/ISSN/Övrigt
- ISSN: 1525-0024