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Identification of PDGFR as a receptor for AAV-5 transduction

  1. Author:
    Di Pasquale, G.
    Davidson, B. L.
    Stein, C. S.
    Martins, I. S.
    Scudiero, D.
    Monks, A.
    Chiorini, J. A.

  2. Author Address

    Natl Inst Dent & Craniofacial Res, Gene Therapy & Therapeut Branch, NIH, Bethesda, MD 20892 USA Natl Inst Dent & Craniofacial Res, Gene Therapy & Therapeut Branch, NIH, Bethesda, MD 20892 USA Univ Iowa, Coll Med, Dept Internal Med, Iowa City, IA 52242 USA Univ Iowa, Coll Med, Dept Neurol, Iowa City, IA 52242 USA Univ Iowa, Coll Med, Dept Physiol & Biophys, Iowa City, IA 52242 USA SAIC Frederick, Frederick, MD 21702 USA Chiorini JA Natl Inst Dent & Craniofacial Res, Gene Therapy & Therapeut Branch, NIH, Bethesda, MD 20892 USA
    1. Year: 2003
  2. Journal: Nature Medicine
    1. 9
    2. 10
    3. Pages: 1306-1312
  4. Type of Article: Article
  1. Abstract:

    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.

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