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.
Keywords: Gamma-retroviral vector, safety, biosafety, gene therapy.
Current Gene Therapy
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.
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Cite this article as:
Deichmann Annette and Schmidt Manfred, Biosafety Considerations Using Gamma-Retroviral Vectors in Gene Therapy, Current Gene Therapy 2013; 13 (6) . https://dx.doi.org/10.2174/15665232113136660004
DOI https://dx.doi.org/10.2174/15665232113136660004 |
Print ISSN 1566-5232 |
Publisher Name Bentham Science Publisher |
Online ISSN 1875-5631 |
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