Identification of PDGFR as a receptor for AAV-5 transduction

Nat Med. 2003 Oct;9(10):1306-12. doi: 10.1038/nm929. Epub 2003 Sep 14.


Understanding the process of vector transduction has important implications for the application and optimal use of a vector system for human gene therapy. Recent studies with vectors based on adeno-associated virus type 5 (AAV-5) have shown utility of this vector system in the lung, central nervous system, muscle and eye. To understand the natural tropism of this virus and to identify proteins necessary for AAV-5 transduction, we characterized 43 cell lines as permissive or nonpermissive for AAV-5 transduction and compared the gene expression profiles derived from cDNA microarray analyses of those cell lines. A statistically significant correlation was observed between expression of the platelet-derived growth factor receptor (PDGFR-alpha-polypeptide) and AAV-5 transduction. Subsequent experiments confirmed the role of PDGFR-alpha and PDGFR-beta as receptors for AAV-5. The tropism of AAV-5 in vivo also correlated with the expression pattern of PDGFR-alpha.

MeSH terms

  • Animals
  • Cell Line
  • Dependovirus / genetics*
  • Dependovirus / metabolism*
  • ErbB Receptors / genetics
  • ErbB Receptors / metabolism
  • Gene Expression Profiling
  • Genetic Therapy / methods
  • Genetic Vectors / genetics
  • Genetic Vectors / metabolism
  • Hippocampus / cytology
  • Hippocampus / physiology
  • Humans
  • Oligonucleotide Array Sequence Analysis
  • Protein Isoforms / genetics
  • Protein Isoforms / metabolism
  • Receptors, Platelet-Derived Growth Factor / genetics
  • Receptors, Platelet-Derived Growth Factor / metabolism*
  • Statistics as Topic
  • Transduction, Genetic*


  • Protein Isoforms
  • ErbB Receptors
  • Receptors, Platelet-Derived Growth Factor