Human Gene Therapy: Risks and Safety Considerations


Gene therapy has long been perceived as a once‐in‐a‐lifetime treatment that can give life‐long benefits. Some potential risks have been underestimated or have not received sufficient attention.

Keywords: gene; disease; viral; therapy; safety; oncogenesis; virogenesis

Further Reading

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Bushman FD (2003) Targeting survival: integration site selection by retroviruses and LTR‐retrotransposons. Cell 115: 135–138 (Review).

Cavazzana‐Calvo M, Hacein‐Bey S, de Saint Basile G et al. (2000) Gene therapy of human severe combined immunodeficiency (SCID)‐X1 disease. Science 288: 669–672.

Fox JL (2000) Gene‐therapy death prompts broad civil lawsuit. Nature Biotechnology 18: 1136.

Friedmann T (1997) Overcoming the obstacles to gene therapy. Scientific American 276: 96–101.

Hertig C, Coupar BE, Gould AR and Boyle DB (1997) Field and vaccine strains of fowlpox virus carry integrated sequences from the avian retrovirus, reticuloendotheliosis virus. Virology 235: 367–376.

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Thomas CE, Ehrhardt A and Kay MA (2003) Progress and problems with the use of viral vectors for gene therapy. Nature Review Genetics 4: 346–358 (Review).

Vadolas J, Williamson R and Ioannou PA (2002) Gene therapy for inherited lung disorders: an insight into pulmonary defence. Pulmonary Pharmacology and Therapeutics 15: 61–72 (Review).

Wu X, Li Y, Crise B and Burgess SM (2003) Transcription start regions in the human genome are favored targets for MLV integration. Science 300: 1749–1751.

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Ioannou, Panos A, and Vadolas, Jim(Jan 2006) Human Gene Therapy: Risks and Safety Considerations. In: eLS. John Wiley & Sons Ltd, Chichester. [doi: 10.1038/npg.els.0003946]