Production of recombinant adeno-associated virus vectors

Hum Gene Ther. 2005 May;16(5):551-7. doi: 10.1089/hum.2005.16.551.


Recombinant adeno-associated virus (rAAV) is a prototypical gene therapy vector characterized by excellent safety profiles, wide host range, and the ability to transduce differentiated cells. Numerous rAAV-based vectors providing efficient and sustained expression of transgenes in target tissues have been developed for preclinical studies. Interest in rAAV has been driven by advances in production methods originally developed for rAAV serotype 2 vectors and expanded to include alternative serotypes. The transition to clinical trials is dependent on the development of scalable production methods of Good Manufacturing Practice-grade vectors described in this review.

Publication types

  • Review

MeSH terms

  • Animals
  • Baculoviridae / genetics
  • Cell Line
  • Dependovirus / genetics*
  • Dependovirus / isolation & purification*
  • Genetic Vectors / genetics*
  • Genetic Vectors / isolation & purification*
  • Helper Viruses / genetics
  • Helper Viruses / physiology
  • Humans
  • Plasmids
  • Recombination, Genetic*
  • Serotyping
  • Transduction, Genetic
  • Transfection