Lentiviral vectors transcriptionally targeted to hematopoietic cells by WASP gene proximal promoter sequences

Gene Ther. 2005 Apr;12(8):715-23. doi: 10.1038/sj.gt.3302457.

Abstract

The development of vectors that express a therapeutic transgene efficiently and specifically in hematopoietic cells (HCs) is an important goal for gene therapy of hematological disorders. In order to achieve this, we used a 500 bp fragment from the proximal WASP gene promoter to drive the expression of the WASP cDNA in the context of a self-inactivating lentiviral vector. Single-round transduction of WASp-deficient herpesvirus saimiri (HVS)-immortalized cells as well as primary allospecific T cells from Wiskott-Aldrich syndrome (WAS) patients with this vector (WW) resulted in expression levels similar to those of control cells. Non-HCs were transduced with similar efficiency, but the levels of WASp were 135-350 times lower than those achieved in HCs. Additionally, transduction of WASp-deficient cells with WW conferred a selective growth advantage in vitro. Therefore, lentiviral vectors incorporating proximal promoter sequences from the WASP gene confer hematopoietic-specific, and physiological protein expression.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Blotting, Western / methods
  • Cell Line, Transformed
  • Cell Proliferation
  • Colony-Forming Units Assay
  • Gene Expression
  • Genetic Therapy / methods*
  • Genetic Vectors / administration & dosage
  • Genetic Vectors / genetics
  • Herpesvirus 2, Saimiriine / genetics
  • Humans
  • Mice
  • Mice, Inbred BALB C
  • Promoter Regions, Genetic*
  • Proteins / genetics*
  • Reverse Transcriptase Polymerase Chain Reaction
  • Stem Cells / metabolism
  • T-Lymphocytes / metabolism*
  • Transduction, Genetic / methods
  • Transgenes
  • Wiskott-Aldrich Syndrome / immunology
  • Wiskott-Aldrich Syndrome / therapy*
  • Wiskott-Aldrich Syndrome Protein

Substances

  • Proteins
  • WAS protein, human
  • Was protein, mouse
  • Wiskott-Aldrich Syndrome Protein