Current Gene Therapy

Ignacio Anegon
Director INSERM UMR 1064-Center for Research in Transplantation and Immunology
CHU de Nantes. 30, boulevard
Nantes
France

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Biosafety Considerations Using Gamma-Retroviral Vectors in Gene Therapy

Author(s): Annette Deichmann, Manfred Schmidt.

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.

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Article Details

VOLUME: 13
ISSUE: 6
Year: 2013
Page: [469 - 477]
Pages: 9
DOI: 10.2174/15665232113136660004
Price: $58