Granulocyte-macrophage colony-stimulating factor mRNA stabilization enhances transgenic expression in normal cells and tissues

Blood. 1995 Oct 1;86(7):2551-8.

Abstract

To increase transgenic production of granulocyte-macrophage colony-stimulating factor (GM-CSF), we mutated the mRNA's 3'-untranslated region, AUUUA instability elements. Expression vectors containing human or murine GM-CSF cDNAs coding for wild-type (GM-AUUUA) or mutant versions with reiterated AUGUA repeats (GM-AUGUA) were transfected into cells in culture or animals using particle-mediated gene-transfer technology. Normal peripheral blood mononuclear cells accumulated 20-fold greater levels of GM-CSF mRNA and secreted comparably greater amounts of cytokine after transfection with hGM-AUGUA expression vectors versus hGM-AUUUA. hGM-AUGUA mRNA was fivefold more stable (t 1/2 = 95 minutes) than hGM-AUUUA mRNA (t 1/2 = 20 minutes), accounting for elevated steady-state levels. Transfection site extracts and serum samples obtained 24 hours after gene transfer of hGM-AUGUA cDNA into mouse skin contained greater than 32 ng/mL and 650 pg/mL of GM-CSF protein, respectively, compared with 0.33 ng/mL and less than 8 pg/mL for hGM-AUUUA cDNA. GM-CSF produced from mGM-AUGUA cDNA transfected into rat abdominal epidermis induced a profound neutrophil infiltrate. These data suggest a novel strategy for enhanced production of biologically active cytokines by normal cells after in vivo gene transfer.

Publication types

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

MeSH terms

  • Animals
  • Base Sequence
  • Cells, Cultured
  • DNA, Complementary / genetics
  • Drug Stability
  • Epidermis / metabolism
  • Gene Expression*
  • Genetic Vectors*
  • Granulocyte-Macrophage Colony-Stimulating Factor / genetics*
  • Humans
  • Leukocytes, Mononuclear
  • Mice
  • Molecular Sequence Data
  • Mutagenesis
  • RNA, Messenger / metabolism*
  • Rats
  • Recombinant Proteins
  • Repetitive Sequences, Nucleic Acid
  • Skin / metabolism
  • Transfection

Substances

  • DNA, Complementary
  • RNA, Messenger
  • Recombinant Proteins
  • Granulocyte-Macrophage Colony-Stimulating Factor