Receptor targeting of adeno-associated virus vectors

Gene Ther. 2003 Jul;10(14):1142-51. doi: 10.1038/sj.gt.3301976.

Abstract

Adeno-associated virus (AAV) is a promising vector for human somatic gene therapy. However, its broad host range is a disadvantage for in vivo gene therapy, because it does not allow the selective tissue- or organ-restricted transduction required to enhance the safety and efficiency of the gene transfer. Therefore, increasing efforts are being made to target AAV-2-based vectors to specific receptors. The studies summarized in this review show that it is possible to target AAV-2 to a specific cell. So far, the most promising approach is the genetic modification of the viral capsid. However, the currently available AAV-2 targeting vectors need to be improved with regard to the elimination of the wild-type AAV-2 tropism and the improvement of infectious titers. The creation of highly efficient AAV-2 targeting vectors will also require a better understanding of the transmembrane and intracellular processing of this virus.

Publication types

  • Research Support, Non-U.S. Gov't
  • Review

MeSH terms

  • Animals
  • Antibodies / genetics
  • Capsid Proteins / genetics
  • Dependovirus / genetics*
  • Gene Targeting / methods*
  • Genetic Engineering
  • Genetic Therapy / methods*
  • Genetic Vectors / administration & dosage*
  • Genetic Vectors / genetics
  • Genome, Viral
  • Humans
  • Ligands
  • Receptors, Cell Surface / metabolism*
  • Transduction, Genetic / methods

Substances

  • Antibodies
  • Capsid Proteins
  • Ligands
  • Receptors, Cell Surface