A partial down-regulation of WASP is sufficient to inhibit podosome formation in dendritic cells

Mol Ther. 2006 Apr;13(4):729-37. doi: 10.1016/j.ymthe.2005.11.003. Epub 2005 Dec 15.

Abstract

The Wiskott Aldrich syndrome protein (WASP) is a hematopoietic-specific cytoskeletal regulator that is necessary for induction of normal immunity. In the context of effective gene therapy for WAS, cellular models of human WASP deficiency are important for definition of the threshold of protein expression required for optimal activity. Using lentiviral vector-mediated RNA interference (RNAi), we were able to down-regulate the levels of human WASP in cell lines and primary cells. In dendritic cells (DC), RNAi-induced WASP deficiency did not impair phenotypic maturation but perturbed cytoskeletal organization. As a result, podosomes, which are actin-rich structures present in immature adherent DC, were formed less efficiently and motility was disturbed. Overall, treatment of cells with RNAi recapitulated the phenotype of cells derived from patients or animals with inactivating mutations of the WAS gene. Interestingly, reduction of the levels of WASP to about 60% of normal was sufficient to inhibit the formation of podosomes in DC, implying that this cell type requires near-normal levels of WASP to sustain physiological cytoskeleton-dependent activities.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Cell Movement / drug effects
  • Cell Movement / physiology*
  • Cells, Cultured
  • Cytoskeleton / drug effects
  • Cytoskeleton / metabolism
  • Dendritic Cells / metabolism*
  • Dose-Response Relationship, Drug
  • Down-Regulation*
  • Gene Expression
  • Genetic Therapy
  • Genetic Vectors / therapeutic use
  • Humans
  • Jurkat Cells
  • Lentivirus / genetics
  • Mutation
  • RNA, Small Interfering / metabolism
  • RNA, Small Interfering / pharmacology*
  • Transduction, Genetic
  • Wiskott-Aldrich Syndrome / genetics
  • Wiskott-Aldrich Syndrome Protein / deficiency
  • Wiskott-Aldrich Syndrome Protein / genetics
  • Wiskott-Aldrich Syndrome Protein / metabolism
  • Wiskott-Aldrich Syndrome Protein / physiology*

Substances

  • RNA, Small Interfering
  • Wiskott-Aldrich Syndrome Protein