Gene therapy progress and prospects: development of improved lentiviral and retroviral vectors--design, biosafety, and production

Gene Ther. 2005 Jul;12(14):1089-98. doi: 10.1038/sj.gt.3302570.

Abstract

Replication defective vectors derived from simple retroviruses or the more complex genomes of lentiviruses continue to offer the advantages of long-term expression, cell and tissue specific tropism, and large packaging capacity for the delivery of therapeutic genes. The occurrence of adverse events caused by insertional mutagenesis in three patients in a gene therapy trial for X-linked SCID emphasizes the potential for problems in translating this approach to the clinic. Several genome-wide studies of retroviral integration are now providing novel insights into the integration site preferences of different vector classes. We review recent developments in vector design, integration, biosafety, and production.

Publication types

  • Review

MeSH terms

  • Gene Transfer Techniques
  • Genetic Therapy / adverse effects
  • Genetic Therapy / methods
  • Genetic Therapy / trends*
  • Genetic Vectors / genetics*
  • Humans
  • Lentivirus / genetics*
  • Mutagenesis, Insertional
  • Retroviridae / genetics*