Title:Biosafety Considerations Using Gamma-Retroviral Vectors in Gene Therapy
Volume: 13
Issue: 6
Author(s): Annette Deichmann and Manfred Schmidt
Affiliation:
Keywords:
Gamma-retroviral vector, safety, biosafety, gene therapy.
Abstract: Gene therapy has become a feasible and efficient strategy for the treatment of human genetic diseases. The
main principle of a gene therapeutic regimen relies on the delivery of a corrected gene of interest in human cells. In about
one fifth of the clinical trials, gamma-retroviral vectors are used as gene-transfer vehicle. However, previous successful
gene therapy trials revealed gamma-retroviral vector-mediated severe adverse events: Upregulation of proto-oncogenes
led to malignant transformation of the affected cells and tumor progression. These severe adverse events enhanced the development
of new ‘safer’ gamma-retroviral vectors and comprehensive biosafety studies. This review highlights all possible
safety and biosafety risks of gamma-retroviral vectors.
