Efficient gene delivery and expression in mammalian cells using DNA coupled with perfringolysin O

Gene Ther. 1995 Sep;2(7):498-503.

Abstract

Current non-viral DNA vectors for gene therapy are limited by low cellular transfection efficiencies and low levels of gene expression due to inefficient endosomal DNA release. We have used perfringolysin O (PFO), a membrane active bacterial protein, to deliver DNA into cells. PFO belongs to the so-called sulphydryl-activated family of membrane active bacterial proteins, which have been used to deliver small molecules and proteins into cells. PFO was incorporated into DNA complexes through a biotin-streptavidin bridge and the DNA-PFO complexes were used to deliver the Escherichia coli beta-galactosidase gene into cells. High levels of gene expression were achieved in murine sol 8 myoblast cells using these DNA-PFO complexes. The level of gene expression correlated well with the content of PFO in the complexes. Under optimal conditions, 15-20% of the cells were stained blue with X-gal. Furthermore, the expression was independent of a receptor ligand. Thus, membrane active bacterial proteins may be an important tool for the future development of non-viral DNA delivery systems for gene therapy.

Publication types

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

MeSH terms

  • Animals
  • Bacterial Toxins / genetics
  • Bacterial Toxins / pharmacology*
  • Cell Line
  • DNA / genetics
  • DNA / pharmacology*
  • Drug Delivery Systems*
  • Gene Expression
  • Gene Transfer Techniques*
  • Hemolysin Proteins / genetics
  • Hemolysin Proteins / pharmacology*
  • Mice
  • beta-Galactosidase / genetics*
  • beta-Galactosidase / pharmacology

Substances

  • Bacterial Toxins
  • Hemolysin Proteins
  • Clostridium perfringens theta-toxin
  • DNA
  • beta-Galactosidase