Progress and problems with the use of viral vectors for gene therapy

Nat Rev Genet. 2003 May;4(5):346-58. doi: 10.1038/nrg1066.


Gene therapy has a history of controversy. Encouraging results are starting to emerge from the clinic, but questions are still being asked about the safety of this new molecular medicine. With the development of a leukaemia-like syndrome in two of the small number of patients that have been cured of a disease by gene therapy, it is timely to contemplate how far this technology has come, and how far it still has to go.

Publication types

  • Research Support, U.S. Gov't, P.H.S.
  • Review

MeSH terms

  • Adenoviridae / genetics
  • Defective Viruses / genetics
  • Dependovirus / genetics
  • Genetic Therapy / trends*
  • Genetic Vectors*
  • Humans
  • Lentivirus / genetics
  • Neoplasms / therapy*
  • Retroviridae / genetics
  • Simplexvirus / genetics
  • Viruses / genetics*