Prolonged expression of therapeutic levels of human granulocyte colony-stimulating factor in rats following gene transfer to skeletal muscle

Hum Gene Ther. 1996 Aug 1;7(12):1423-9. doi: 10.1089/hum.1996.7.12-1423.


Gene transfer to skeletal muscle was examined as a means of gene therapy for neutropenia. A recombinant retrovirus containing a human granulocyte colony-stimulating factor (G-CSF) gene was introduced into primary human or rat myoblasts, which were then shown to produce biologically active G-CSF. Transplantation of G-CSF-producing rat myoblasts into the muscle of syngeneic rats resulted in a 15-fold increase in absolute neutrophil counts. This increase correlated with detection of circulating human G-CSF protein throughout the 6-month duration of the experiment. These results clearly demonstrate long-term production of therapeutically relevant amounts of a human protein by normal cells in vivo.

Publication types

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

MeSH terms

  • Animals
  • Cells, Cultured
  • Child, Preschool
  • Gene Transfer Techniques*
  • Genetic Therapy
  • Granulocyte Colony-Stimulating Factor / biosynthesis*
  • Granulocyte Colony-Stimulating Factor / genetics
  • Hematopoiesis / drug effects
  • Humans
  • Leukocyte Count / drug effects*
  • Male
  • Muscle, Skeletal / metabolism*
  • Neutropenia / therapy
  • Neutrophils / drug effects*
  • Rats
  • Rats, Inbred F344
  • Recombinant Fusion Proteins / biosynthesis*
  • Time Factors


  • Recombinant Fusion Proteins
  • Granulocyte Colony-Stimulating Factor